\u0645\u0627 \u0648\u0631\u0627\u0621 \u0623\u0628\u062d\u0627\u062b AAV: \u0627\u0644\u0628\u062d\u062b \u0639\u0646 \u0637\u0631\u0642 \u0623\u0643\u062b\u0631 \u0641\u0639\u0627\u0644\u064a\u0629 \u0644\u062a\u0648\u0635\u064a\u0644 \u0627\u0644\u0623\u062f\u0648\u064a\u0629<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n\u062a\u0627\u0631\u064a\u062e \u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a \u0627\u0644\u0642\u0627\u0626\u0645 \u0639\u0644\u0649 AAV<\/h2>\n\n\n\n
\u0646\u0638\u0631\u064b\u0627 \u0644\u0639\u062f\u0645 \u0627\u0645\u062a\u0644\u0627\u0643\u0647\u0627 \u0627\u0644\u063a\u0644\u0627\u0641 \u0627\u0644\u062e\u0627\u0631\u062c\u064a \u0627\u0644\u0645\u0639\u062a\u0627\u062f \u0644\u0644\u0641\u064a\u0631\u0648\u0633\u0627\u062a\u060c \u062a\u064f\u0633\u0645\u0649 \u0627\u0644\u0641\u064a\u0631\u0648\u0633\u0627\u062a \u0627\u0644\u063a\u062f\u064a\u0629 \u0627\u0644\u0645\u0631\u062a\u0628\u0637\u0629 \u0628\u0627\u0644\u063a\u062f\u0629 \u0627\u0644\u062f\u0631\u0642\u064a\u0629 \u0639\u0627\u062f\u0629\u064b \u0628\u0627\u0644\u0641\u064a\u0631\u0648\u0633\u0627\u062a \u0627\u0644\u0639\u0627\u0631\u064a\u0629 \u0623\u0648 \u063a\u064a\u0631 \u0627\u0644\u0645\u063a\u0644\u0641\u0629. \u0628\u0644 \u062a\u062d\u062a\u0648\u064a \u0639\u0644\u0649 \u063a\u0644\u0627\u0641 \u0628\u0631\u0648\u062a\u064a\u0646\u064a \u064a\u064f\u063a\u0644\u0641 \u0627\u0644\u062d\u0645\u0636 \u0627\u0644\u0646\u0648\u0648\u064a \u0644\u0644\u0641\u064a\u0631\u0648\u0633.<\/p>\n\n\n\n
\u0641\u064a \u0639\u0627\u0645 \u0661\u0669\u0666\u0665\u060c \u0623\u062b\u0646\u0627\u0621 \u062a\u062d\u0636\u064a\u0631 \u0645\u0633\u062a\u062d\u0636\u0631\u0627\u062a \u0627\u0644\u0641\u064a\u0631\u0648\u0633\u0627\u062a \u0627\u0644\u063a\u062f\u064a\u0629\u060c \u0627\u0643\u062a\u0634\u0641 \u0627\u0644\u0639\u0644\u0645\u0627\u0621 \u0647\u0630\u0647 \u0627\u0644\u0641\u064a\u0631\u0648\u0633\u0627\u062a \u0628\u0634\u0643\u0644\u064d \u063a\u064a\u0631 \u0645\u0642\u0635\u0648\u062f \u0646\u0633\u0628\u064a\u064b\u0627. \u0648\u0638\u0647\u0631 \u0645\u0641\u0647\u0648\u0645 \u0647\u0646\u062f\u0633\u0629 \u0627\u0644\u0641\u064a\u0631\u0648\u0633\u0627\u062a \u0627\u0644\u063a\u062f\u064a\u0629 \u0644\u062a\u0648\u0635\u064a\u0644 \u0627\u0644\u0623\u062f\u0648\u064a\u0629 \u0628\u0641\u0636\u0644 \u0627\u0644\u062d\u0645\u0636 \u0627\u0644\u0646\u0648\u0648\u064a \u0627\u0644\u0641\u064a\u0631\u0648\u0633\u064a \u0627\u0644\u0645\u064f\u063a\u0644\u0651\u0641. \u0627\u0633\u062a\u064f\u062e\u062f\u0645 \u0646\u0627\u0642\u0644 \u0641\u064a\u0631\u0648\u0633 \u0627\u0644\u063a\u062f\u064a\u0629 \u0641\u064a \u0627\u0644\u0628\u062f\u0627\u064a\u0629 \u0644\u062f\u0649 \u0645\u0631\u064a\u0636 \u0645\u0635\u0627\u0628 \u0628\u0627\u0644\u062a\u0644\u064a\u0641 \u0627\u0644\u0643\u064a\u0633\u064a\u060c \u0648\u0647\u0648 \u0645\u0631\u0636 \u0648\u0631\u0627\u062b\u064a \u064a\u0624\u062f\u064a \u0625\u0644\u0649 \u062a\u0631\u0627\u0643\u0645 \u0627\u0644\u0645\u062e\u0627\u0637 \u0641\u064a \u0627\u0644\u062c\u0633\u0645\u060c \u0628\u0639\u062f \u062b\u0644\u0627\u062b\u064a\u0646 \u0639\u0627\u0645\u064b\u0627 \u0645\u0646 \u0627\u0643\u062a\u0634\u0627\u0641\u0647.<\/p>\n\n\n\n
\u0641\u064a \u0639\u0627\u0645 \u0662\u0660\u0661\u0662\u060c \u0648\u0627\u0641\u0642 \u0627\u0644\u0627\u062a\u062d\u0627\u062f \u0627\u0644\u0623\u0648\u0631\u0648\u0628\u064a \u0639\u0644\u0649 \u062f\u0648\u0627\u0621 \u062c\u0644\u064a\u0628\u064a\u0631\u0627\u060c \u0648\u0647\u0648 \u0623\u0648\u0644 \u0639\u0644\u0627\u062c \u062c\u064a\u0646\u064a \u064a\u064f\u0639\u062a\u0645\u062f \u0639\u0644\u0649 \u0627\u0644\u0625\u0637\u0644\u0627\u0642. \u0641\u064a \u062c\u0644\u064a\u0628\u064a\u0631\u0627\u060c \u0646\u064f\u0642\u0644 \u0627\u0644\u062c\u064a\u0646 \u0639\u0628\u0631 \u0646\u0627\u0642\u0644 \u0641\u064a\u0631\u0648\u0633\u064a \u0645\u0631\u062a\u0628\u0637 \u0628\u0627\u0644\u063a\u062f\u0629 \u0627\u0644\u062f\u0631\u0642\u064a\u0629 \u0644\u0639\u0644\u0627\u062c \u0646\u0642\u0635 \u0644\u064a\u0628\u0627\u0632 \u0627\u0644\u0628\u0631\u0648\u062a\u064a\u0646 \u0627\u0644\u062f\u0647\u0646\u064a \u0627\u0644\u0648\u0631\u0627\u062b\u064a\u060c \u0648\u0647\u0648 \u0627\u0636\u0637\u0631\u0627\u0628 \u0648\u0631\u0627\u062b\u064a \u0646\u0627\u062f\u0631 \u064a\u0639\u0627\u0646\u064a \u0641\u064a\u0647 \u0627\u0644\u062c\u0633\u0645 \u0645\u0646 \u0646\u0642\u0635 \u0625\u0646\u0632\u064a\u0645 \u064a\u064f\u062d\u0644\u0644 \u062f\u0647\u0648\u0646 \u0627\u0644\u062f\u0645. \u0628\u0639\u062f \u062e\u0645\u0633 \u0633\u0646\u0648\u0627\u062a\u060c \u062e\u0641\u062a \u062d\u0645\u0627\u0633\u0629 \u0647\u0630\u0627 \u0627\u0644\u0639\u0644\u0627\u062c \u0639\u0646\u062f\u0645\u0627 \u0633\u062d\u0628\u062a\u0647 \u0634\u0631\u0643\u0629 \u064a\u0648\u0646\u064a\u0643\u064a\u0648\u0631\u060c \u0627\u0644\u0634\u0631\u0643\u0629 \u0627\u0644\u0645\u064f\u0628\u062a\u0643\u0631\u0629 \u0644\u0647\u060c \u0645\u0646 \u0627\u0644\u0633\u0648\u0642 \u0628\u0633\u0628\u0628 \u0627\u0631\u062a\u0641\u0627\u0639 \u062a\u0643\u0644\u0641\u062a\u0647 \u0648\u0627\u0646\u062e\u0641\u0627\u0636 \u0627\u0644\u0637\u0644\u0628 \u0639\u0644\u064a\u0647.<\/p>\n\n\n\n
\u0648\u0645\u0639 \u0630\u0644\u0643\u060c \u0623\u062a\u0627\u062d\u062a \u0627\u0644\u0645\u0648\u0627\u0641\u0642\u0629 \u0627\u0644\u062a\u0646\u0638\u064a\u0645\u064a\u0629 \u0637\u0631\u062d \u0627\u0644\u0645\u0632\u064a\u062f \u0645\u0646 \u0639\u0644\u0627\u062c\u0627\u062a \u062c\u064a\u0646\u0627\u062a AAV \u0641\u064a \u0627\u0644\u0633\u0648\u0642\u060c \u0648\u0627\u0644\u0639\u062f\u064a\u062f \u0645\u0646\u0647\u0627 \u0639\u0644\u0627\u062c\u064a. \u0648\u062a\u0634\u0645\u0644 \u0647\u0630\u0647 \u0627\u0644\u0639\u0644\u0627\u062c\u0627\u062a Elevidys \u0644\u0639\u0644\u0627\u062c \u0645\u0631\u0636 \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u062f\u0648\u0634\u064a\u0646 (DMD) \u0627\u0644\u0630\u064a \u064a\u064f\u0633\u0628\u0628 \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a\u060c \u0648Luxturna \u0644\u0639\u0644\u0627\u062c \u0645\u0631\u0636 \u0627\u0644\u0639\u064a\u0646 \u0627\u0644\u0648\u0631\u0627\u062b\u064a \u0627\u0644\u0646\u0627\u062f\u0631 \u0627\u0644\u0645\u0639\u0631\u0648\u0641 \u0628\u0627\u0633\u0645 \u0639\u0645\u0649 \u0644\u064a\u0628\u0631 \u0627\u0644\u062e\u0644\u0642\u064a\u060c \u0648Zolgensma \u0644\u0639\u0644\u0627\u062c \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0627\u0644\u0634\u0648\u0643\u064a\u060c \u0648\u0647\u0648 \u062d\u0627\u0644\u0629 \u062a\u0646\u0643\u0633\u064a\u0629 \u0639\u0635\u0628\u064a\u0629 \u062a\u064f\u0635\u064a\u0628 \u0627\u0644\u0646\u062e\u0627\u0639 \u0627\u0644\u0634\u0648\u0643\u064a\u060c \u0648Hemgenix \u0648Roctavian \u0644\u0639\u0644\u0627\u062c \u0627\u0636\u0637\u0631\u0627\u0628\u0627\u062a \u0627\u0644\u062f\u0645 \u0627\u0644\u0646\u0627\u062f\u0631\u0629 \u0627\u0644\u0647\u064a\u0645\u0648\u0641\u064a\u0644\u064a\u0627 B \u0648A\u060c \u0639\u0644\u0649 \u0627\u0644\u062a\u0648\u0627\u0644\u064a.<\/p>\n\n\n\n
\u0641\u064a\u0631\u062a\u0643\u0633 \u062a\u0646\u0647\u064a \u0634\u0631\u0627\u0643\u062a\u0647\u0627 \u0645\u0639 Affinia Therapeutics \u0648Tevard Biosciences<\/h2>\n\n\n\n
\u064a\u0628\u062f\u0648 \u0623\u0646 \u0639\u0644\u0627\u062c\u0627\u062a \u062c\u064a\u0646\u0627\u062a \u0627\u0644\u0641\u064a\u0631\u0648\u0633 \u0627\u0644\u063a\u062f\u064a \u0627\u0644\u0645\u0631\u062a\u0628\u0637 \u0628\u0627\u0644\u063a\u062f\u0629 \u0627\u0644\u062f\u0631\u0642\u064a\u0629 (AAV) \u0642\u062f \u062d\u0627\u0646 \u0648\u0642\u062a\u0647\u0627. \u0641\u0641\u064a \u0627\u062a\u0641\u0627\u0642\u064a\u0629 \u0628\u0642\u064a\u0645\u0629 1.6 \u0645\u0644\u064a\u0627\u0631 \u062f\u0648\u0644\u0627\u0631 \u0623\u0645\u0631\u064a\u0643\u064a \u0645\u0639 \u0634\u0631\u0643\u0629 \u0623\u0641\u064a\u0646\u064a\u0627 \u0639\u0627\u0645 2020\u060c \u0648\u0639\u062f\u062a \u0634\u0631\u0643\u0629 \u0641\u064a\u0631\u062a\u0643\u0633 \u0628\u0625\u064a\u062c\u0627\u062f \u0641\u064a\u0631\u0648\u0633\u0627\u062a \u063a\u062f\u064a\u0629 \u0645\u0631\u062a\u0628\u0637\u0629 \u0628\u0627\u0644\u063a\u062f\u0629 \u0627\u0644\u062f\u0631\u0642\u064a\u0629 (AAV) \u0644\u0639\u0644\u0627\u062c \u062f\u0648\u0634\u064a\u0646 \u0648\u0627\u0644\u062a\u0644\u064a\u0641 \u0627\u0644\u0643\u064a\u0633\u064a \u0628\u0634\u0643\u0644 \u0623\u0641\u0636\u0644. \u0648\u0645\u0639 \u0630\u0644\u0643\u060c \u0623\u0628\u0644\u063a\u062a \u0623\u0641\u064a\u0646\u064a\u0627 \u0634\u0631\u0643\u0629 \u0625\u0646\u062f\u0628\u0648\u064a\u0646\u062a\u0633 \u0627\u0644\u0623\u0633\u0628\u0648\u0639 \u0627\u0644\u0645\u0627\u0636\u064a \u0628\u0623\u0646\u0647\u0627 \u0627\u0633\u062a\u0639\u0627\u062f\u062a \u062d\u0642\u0648\u0642 \u0646\u0627\u0642\u0644\u0627\u062a \u062f\u0648\u0634\u064a\u0646. \u0641\u064a \u0639\u0627\u0645 2022\u060c \u0643\u0627\u0646\u062a \u0641\u064a\u0631\u062a\u0643\u0633 \u0642\u062f \u062a\u062e\u0644\u062a \u0628\u0627\u0644\u0641\u0639\u0644 \u0639\u0646 \u062d\u0642\u0648\u0642 \u0627\u0644\u0646\u0627\u0642\u0644\u0627\u062a \u0627\u0644\u062a\u064a \u062a\u0633\u062a\u0647\u062f\u0641 \u0627\u0644\u062a\u0644\u064a\u0641 \u0627\u0644\u0643\u064a\u0633\u064a.<\/p>\n\n\n\n
\u0645\u0646 \u0628\u064a\u0646 \u0623\u0645\u0648\u0631 \u0623\u062e\u0631\u0649\u060c \u0623\u064f\u0628\u0644\u063a\u062a \u0634\u0631\u0643\u0629 Tevard \u0623\u0648\u0627\u062e\u0631 \u0627\u0644\u0639\u0627\u0645 \u0627\u0644\u0645\u0627\u0636\u064a \u0628\u0627\u0646\u0633\u062d\u0627\u0628 \u0634\u0631\u0643\u0629 Vertex \u0645\u0646 \u0639\u0642\u062f DMD. \u0643\u0645\u0627 \u0627\u0633\u062a\u0639\u0627\u062f\u062a \u0627\u0644\u0634\u0631\u0643\u0629 \u062d\u0642\u0648\u0642 \u0628\u0631\u0646\u0627\u0645\u062c\u0647\u0627 \u0627\u0644\u062e\u0627\u0635 \u0628\u0627\u0644\u062d\u0645\u0636 \u0627\u0644\u0646\u0648\u0648\u064a \u0627\u0644\u0631\u064a\u0628\u0648\u0632\u064a \u0627\u0644\u0646\u0627\u0642\u0644 (tRNA) \u0647\u0630\u0627 \u0627\u0644\u0634\u0647\u0631. \u0648\u0628\u064a\u0646\u0645\u0627 \u062a\u0633\u062a\u0639\u062f \u0627\u0644\u0645\u062c\u0645\u0648\u0639\u0629 \u0644\u062a\u0642\u062f\u064a\u0645 \u0628\u064a\u0627\u0646\u0627\u062a\u0647\u0627 \u0644\u062f\u0639\u0645 \u0639\u0644\u0627\u062c\u0647\u0627 \u0628\u0627\u0644\u062d\u0645\u0636 \u0627\u0644\u0646\u0648\u0648\u064a \u0627\u0644\u0631\u064a\u0628\u0648\u0632\u064a \u0627\u0644\u0646\u0627\u0642\u0644 \u0641\u064a \u0645\u0624\u062a\u0645\u0631 \u0627\u0644\u062c\u0645\u0639\u064a\u0629 \u0627\u0644\u0623\u0645\u0631\u064a\u0643\u064a\u0629 \u0644\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a \u0648\u0627\u0644\u062e\u0644\u0648\u064a (ASGCT) \u0641\u064a \u0646\u064a\u0648 \u0623\u0648\u0631\u0644\u064a\u0646\u0632 \u0647\u0630\u0627 \u0627\u0644\u0623\u0633\u0628\u0648\u0639\u060c \u062a\u064f\u0628\u062f\u064a \u0634\u0631\u0643\u0629 Tevard \u062a\u0641\u0627\u0624\u0644\u0627\u064b \u0628\u0634\u0623\u0646 \u0627\u0644\u0628\u0631\u0646\u0627\u0645\u062c.<\/p>\n\n\n\n
\u0645\u062e\u0627\u0637\u0631 \u0627\u0644\u0639\u0644\u0627\u062c\u0627\u062a \u0627\u0644\u062c\u064a\u0646\u064a\u0629 \u0627\u0644\u0642\u0627\u0626\u0645\u0629 \u0639\u0644\u0649 \u0641\u064a\u0631\u0648\u0633 \u0627\u0644\u063a\u062f\u0629 \u0627\u0644\u062f\u0631\u0642\u064a\u0629 (AAV)<\/h2>\n\n\n\n
\u062a\u0646\u0633\u062d\u0628 \u0634\u0631\u0643\u0627\u062a \u0627\u0644\u0623\u062f\u0648\u064a\u0629 \u0627\u0644\u0643\u0628\u0631\u0649 \u0645\u0646 \u0627\u0644\u0628\u062d\u062b \u0648\u0627\u0644\u062a\u0637\u0648\u064a\u0631 \u0627\u0644\u0645\u062a\u0639\u0644\u0642 \u0628\u062c\u064a\u0646\u0627\u062a \u0641\u064a\u0631\u0648\u0633 \u0627\u0644\u063a\u062f\u0629 \u0627\u0644\u062f\u0631\u0642\u064a\u0629 (AAV)\u060c \u0628\u0645\u0627 \u0641\u064a \u0630\u0644\u0643 \u0627\u0646\u0633\u062d\u0627\u0628 \u0634\u0631\u0643\u0629 \u0641\u064a\u0631\u062a\u0643\u0633 \u0645\u0646 \u0627\u0644\u0645\u0634\u0647\u062f. \u0642\u0628\u0644 \u0639\u0627\u0645\u064a\u0646\u060c \u0623\u0646\u0647\u062a \u0634\u0631\u0643\u0629 \u0627\u0644\u0623\u062f\u0648\u064a\u0629 \u0627\u0644\u064a\u0627\u0628\u0627\u0646\u064a\u0629 \u0645\u062a\u0639\u062f\u062f\u0629 \u0627\u0644\u062c\u0646\u0633\u064a\u0627\u062a \u062a\u0627\u0643\u064a\u062f\u0627 \u0623\u0628\u062d\u0627\u062b\u0647\u0627 \u0645\u0627 \u0642\u0628\u0644 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0644\u0639\u0644\u0627\u062c\u0627\u062a \u062c\u064a\u0646\u0627\u062a \u0641\u064a\u0631\u0648\u0633 \u0627\u0644\u063a\u062f\u0629 \u0627\u0644\u062f\u0631\u0642\u064a\u0629. \u0648\u0631\u063a\u0645 \u0642\u0644\u0629 \u0627\u0644\u0645\u0639\u0644\u0648\u0645\u0627\u062a \u0622\u0646\u0630\u0627\u0643\u060c \u0643\u0627\u0646 \u0645\u0646 \u0627\u0644\u0645\u062a\u0648\u0642\u0639 \u0623\u0646 \u062a\u064f\u0635\u0628\u062d \u062a\u062d\u0627\u0644\u0641\u0627\u062a\u0647\u0627 \u0627\u0644\u062e\u0627\u0635\u0629 \u0628\u062c\u064a\u0646\u0627\u062a \u0641\u064a\u0631\u0648\u0633 \u0627\u0644\u063a\u062f\u0629 \u0627\u0644\u062f\u0631\u0642\u064a\u0629 \u0645\u0639 \u0634\u0631\u0643\u062a\u064a Codexis \u0641\u064a \u0643\u0627\u0644\u064a\u0641\u0648\u0631\u0646\u064a\u0627 \u0648Selecta Biosciences \u0641\u064a \u0645\u0627\u0633\u0627\u062a\u0634\u0648\u0633\u062a\u0633\u060c \u0648\u0647\u0645\u0627 \u0627\u062b\u0646\u062a\u0627\u0646 \u0645\u0646 \u0627\u062a\u0641\u0627\u0642\u064a\u0627\u062a \u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a \u0627\u0644\u0639\u0634\u0631 \u0627\u0644\u062a\u064a \u0648\u0642\u0639\u062a\u0647\u0627 \u0627\u0644\u0634\u0631\u0643\u0629 \u0628\u064a\u0646 \u0639\u0627\u0645\u064a 2020 \u06482022\u060c \u0645\u0648\u0636\u0639 \u0634\u0643.<\/p>\n\n\n\n
\u0648\u0645\u0646 \u0623\u062c\u0644 \u062a\u0648\u0641\u064a\u0631 \u0627\u0644\u0645\u0627\u0644 \u0641\u064a \u0645\u0648\u0627\u062c\u0647\u0629 \u0627\u0644\u0645\u062e\u0627\u0648\u0641 \u0627\u0644\u0645\u062a\u0632\u0627\u064a\u062f\u0629 \u0628\u0634\u0623\u0646 \u0633\u0644\u0627\u0645\u0629 \u0641\u064a\u0631\u0648\u0633 \u0627\u0644\u063a\u062f\u0629 \u0627\u0644\u062f\u0631\u0642\u064a\u0629 (AAV)\u060c \u0623\u0648\u0642\u0641\u062a \u0634\u0631\u0643\u0629 \u0627\u0644\u0623\u062f\u0648\u064a\u0629 \u0627\u0644\u0639\u0645\u0644\u0627\u0642\u0629 Biogen \u0623\u0628\u062d\u0627\u062b\u0647\u0627 \u0645\u0627 \u0642\u0628\u0644 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0644\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a \u0644\u0641\u064a\u0631\u0648\u0633 \u0627\u0644\u063a\u062f\u0629 \u0627\u0644\u062f\u0631\u0642\u064a\u0629 (AAV) \u0641\u064a \u0646\u0641\u0633 \u0627\u0644\u0639\u0627\u0645.<\/p>\n\n\n\n
\u064a\u0642\u0648\u0644 \u0641\u064a\u0644\u064a\u0628 \u0634\u0627\u0645\u0628\u0648\u0646\u060c \u0645\u0624\u0633\u0633 \u0648\u0631\u0626\u064a\u0633 \u062a\u0646\u0641\u064a\u0630\u064a \u0644\u0634\u0631\u0643\u0629 \u0627\u0644\u062a\u0643\u0646\u0648\u0644\u0648\u062c\u064a\u0627 \u0627\u0644\u062d\u064a\u0648\u064a\u0629 \u0627\u0644\u0641\u0631\u0646\u0633\u064a\u0629 EG 427\u060c \u0625\u0646 \u0645\u0644\u0641 \u0627\u0644\u0633\u0644\u0627\u0645\u0629 \u0627\u0644\u062e\u0627\u0635 \u0628\u0627\u0644\u0646\u0627\u0642\u0644\u0627\u062a \u0623\u0645\u0631 \u0628\u0627\u0644\u063a \u0627\u0644\u0623\u0647\u0645\u064a\u0629 \u0644\u062a\u0648\u0635\u064a\u0644 \u0627\u0644\u0623\u062f\u0648\u064a\u0629.<\/p>\n\n\n\n
\u0635\u0631\u062d \u0634\u0627\u0645\u0628\u0648\u0646 \u0642\u0627\u0626\u0644\u0627\u064b: "\u062a\u0641\u0642\u062f \u0627\u0644\u0639\u0644\u0627\u062c\u0627\u062a \u0627\u0644\u062c\u064a\u0646\u064a\u0629 \u0627\u0644\u0642\u0627\u0626\u0645\u0629 \u0639\u0644\u0649 \u0641\u064a\u0631\u0648\u0633 \u0627\u0644\u063a\u062f\u0629 \u0627\u0644\u062f\u0631\u0642\u064a\u0629 (AAV) \u0634\u0639\u0628\u064a\u062a\u0647\u0627 \u0644\u062f\u0649 \u0634\u0631\u0643\u0627\u062a \u0627\u0644\u0623\u062f\u0648\u064a\u0629 \u0628\u0633\u0628\u0628 \u0639\u062f\u062f \u0645\u0646 \u0627\u0644\u0645\u0634\u0643\u0644\u0627\u062a \u0627\u0644\u062a\u064a \u062a\u0639\u064a\u0642 \u062a\u0637\u0628\u064a\u0642\u0647\u0627 \u0627\u0644\u0641\u0639\u0627\u0644 \u0648\u0627\u0644\u0627\u0642\u062a\u0635\u0627\u062f\u064a \u062e\u0627\u0631\u062c \u0646\u0637\u0627\u0642 \u0627\u0644\u0623\u0645\u0631\u0627\u0636 \u0627\u0644\u0646\u0627\u062f\u0631\u0629. \u064a\u062c\u0628 \u0623\u0646 \u064a\u0643\u0648\u0646 \u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a \u0642\u0627\u062f\u0631\u064b\u0627 \u0639\u0644\u0649 \u0639\u0644\u0627\u062c \u0627\u0644\u0645\u0624\u0634\u0631\u0627\u062a \u0648\u0627\u0644\u0623\u0645\u0631\u0627\u0636 \u0627\u0644\u0645\u0632\u0645\u0646\u0629 \u062e\u0627\u0631\u062c \u0646\u0637\u0627\u0642 \u0627\u0644\u0623\u0645\u0631\u0627\u0636 \u0627\u0644\u0646\u0627\u062f\u0631\u0629 \u062d\u062a\u0649 \u064a\u062d\u0642\u0642 \u0625\u0645\u0643\u0627\u0646\u0627\u062a\u0647 \u0627\u0644\u0643\u0627\u0645\u0644\u0629 \u0643\u0639\u0644\u0627\u062c \u0641\u0639\u0627\u0644".<\/p>\n\n\n\n
\u0639\u0644\u0627\u0648\u0629 \u0639\u0644\u0649 \u0630\u0644\u0643\u060c \u064a\u0642\u0648\u0644 \u0634\u0627\u0645\u0628\u0648\u0646 \u0625\u0646 \u0627\u0644\u062a\u063a\u0644\u0628 \u0639\u0644\u0649 "\u0627\u0644\u0639\u0648\u0627\u0626\u0642 \u0627\u0644\u0631\u0626\u064a\u0633\u064a\u0629 \u0627\u0644\u062a\u064a \u062a\u0648\u0627\u062c\u0647 \u0627\u0644\u0639\u0644\u0627\u062c\u0627\u062a \u0627\u0644\u0642\u0627\u0626\u0645\u0629 \u0639\u0644\u0649 \u0641\u064a\u0631\u0648\u0633\u0627\u062a \u0627\u0644\u063a\u062f\u0629 \u0627\u0644\u062f\u0631\u0642\u064a\u0629" \u0647\u0648 \u0627\u0644\u0633\u0628\u064a\u0644 \u0627\u0644\u0648\u062d\u064a\u062f \u0644\u0639\u0644\u0627\u062c \u0627\u0644\u0623\u0645\u0631\u0627\u0636 \u0627\u0644\u0645\u0632\u0645\u0646\u0629. \u0648\u064a\u0634\u0645\u0644 \u0630\u0644\u0643 \u0627\u0633\u062a\u062e\u062f\u0627\u0645 \u0623\u0633\u0627\u0644\u064a\u0628 \u0645\u062d\u0644\u064a\u0629 \u0623\u0642\u0644 \u0645\u0646\u0627\u0639\u0629\u060c \u0643\u0644\u0645\u0627 \u0623\u0645\u0643\u0646\u060c \u0644\u062a\u062d\u0633\u064a\u0646 \u0645\u0633\u062a\u0648\u0649 \u0627\u0644\u0633\u0644\u0627\u0645\u0629. \u0648\u0644\u0625\u0646\u062a\u0627\u062c \u0623\u062f\u0648\u064a\u0629 \u0642\u0627\u0628\u0644\u0629 \u0644\u0625\u0639\u0627\u062f\u0629 \u0627\u0644\u062c\u0631\u0639\u0627\u062a \u0648\u062a\u062c\u0646\u0628 \u0646\u0645\u0648\u0630\u062c \u0627\u0644\u0639\u0644\u0627\u062c \u0644\u0645\u0631\u0629 \u0648\u0627\u062d\u062f\u0629\u060c \u0627\u0644\u0630\u064a \u064a\u0637\u0631\u062d \u062a\u062d\u062f\u064a\u0627\u062a \u0637\u0628\u064a\u0629 \u0648\u0627\u0642\u062a\u0635\u0627\u062f\u064a\u0629 \u0648\u0623\u062e\u0631\u0649 \u062a\u062a\u0639\u0644\u0642 \u0628\u0627\u0644\u0646\u0638\u0627\u0645 \u0627\u0644\u0639\u0644\u0627\u062c\u064a\u060c \u0641\u0625\u0646 \u062a\u062d\u0633\u064a\u0646 \u0627\u0644\u0642\u062f\u0631\u0629 \u0639\u0644\u0649 \u0627\u0644\u062a\u062d\u0645\u0644 \u0623\u0645\u0631\u064c \u0623\u0633\u0627\u0633\u064a.<\/p>\n\n\n\n
\u0631\u0648\u0634 \u062a\u062a\u062e\u0644\u0649 \u0639\u0646 \u0648\u062d\u062f\u0629 \u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a \u0648\u0641\u0627\u064a\u0632\u0631 \u062a\u0648\u0642\u0641 \u0625\u0646\u062a\u0627\u062c \u0628\u064a\u0643\u0641\u064a\u0632<\/h2>\n\n\n\n
\u0641\u064a \u0627\u0644\u0622\u0648\u0646\u0629 \u0627\u0644\u0623\u062e\u064a\u0631\u0629\u060c \u062a\u062e\u0644\u062a \u0634\u0631\u0643\u062a\u0627 \u0641\u0627\u064a\u0632\u0631 \u0648\u0631\u0648\u0634 \u0639\u0646 \u0627\u0644\u0628\u062d\u062b \u0648\u0627\u0644\u062a\u0637\u0648\u064a\u0631 \u0627\u0644\u0645\u062a\u0639\u0644\u0642\u064a\u0646 \u0628\u0641\u064a\u0631\u0648\u0633 \u0646\u0642\u0635 \u0627\u0644\u0645\u0646\u0627\u0639\u0629 \u0627\u0644\u0628\u0634\u0631\u064a\u0629 (AAV)\u060c \u0628\u0627\u0633\u062a\u062b\u0646\u0627\u0621 \u062a\u0627\u0643\u064a\u062f\u0627 \u0648\u0628\u064a\u0648\u062c\u064a\u0646 \u0641\u064a \u0639\u0627\u0645 \u0662\u0660\u0662\u0663. \u0623\u0648\u0642\u0641\u062a \u0641\u0627\u064a\u0632\u0631 \u0625\u0646\u062a\u0627\u062c \u0628\u064a\u0643\u0641\u064a\u0632\u060c \u0648\u0647\u0648 \u0639\u0644\u0627\u062c \u062c\u064a\u0646\u064a \u0642\u0627\u0626\u0645 \u0639\u0644\u0649 \u0641\u064a\u0631\u0648\u0633 \u0646\u0642\u0635 \u0627\u0644\u0645\u0646\u0627\u0639\u0629 \u0627\u0644\u0628\u0634\u0631\u064a\u0629\u060c \u0648\u0627\u0644\u0630\u064a \u0648\u0627\u0641\u0642\u062a \u0639\u0644\u064a\u0647 \u0625\u062f\u0627\u0631\u0629 \u0627\u0644\u063a\u0630\u0627\u0621 \u0648\u0627\u0644\u062f\u0648\u0627\u0621 \u0627\u0644\u0623\u0645\u0631\u064a\u0643\u064a\u0629 (FDA) \u0644\u0639\u0644\u0627\u062c \u0627\u0644\u0647\u064a\u0645\u0648\u0641\u064a\u0644\u064a\u0627 \u0628\u060c \u0642\u0628\u0644 \u0639\u0627\u0645. \u0648\u0641\u064a \u0641\u0628\u0631\u0627\u064a\u0631\u060c \u0623\u0634\u0627\u0631\u062a \u0627\u0644\u0634\u0631\u0643\u0629 \u0625\u0644\u0649 \u0639\u062f\u0645 \u0627\u0647\u062a\u0645\u0627\u0645\u0647\u0627 \u0628\u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a \u0644\u062d\u0627\u0644\u0629 \u0627\u0644\u0646\u0632\u064a\u0641 \u0647\u0630\u0647.<\/p>\n\n\n\n
\u062e\u0636\u0639\u062a \u0634\u0631\u0643\u0629 \u0627\u0644\u0623\u062f\u0648\u064a\u0629 \u0627\u0644\u0633\u0648\u064a\u0633\u0631\u064a\u0629 \u0627\u0644\u0639\u0645\u0644\u0627\u0642\u0629 "\u0631\u0648\u0634" \u0644\u0625\u0639\u0627\u062f\u0629 \u0647\u064a\u0643\u0644\u0629 \u0634\u0627\u0645\u0644\u0629 \u0641\u064a \u0642\u0633\u0645 \u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a Spark Therapeutics\u060c \u0648\u0645\u0642\u0631\u0647 \u0641\u064a\u0644\u0627\u062f\u0644\u0641\u064a\u0627\u060c \u0648\u0627\u0644\u0645\u064f\u0631\u0643\u0632 \u0639\u0644\u0649 \u0641\u064a\u0631\u0648\u0633 \u0646\u0642\u0635 \u0627\u0644\u0645\u0646\u0627\u0639\u0629 \u0627\u0644\u0628\u0634\u0631\u064a\u0629 (AAV). \u062a\u0645 \u062a\u0633\u0631\u064a\u062d \u0623\u0643\u062b\u0631 \u0645\u0646 \u0646\u0635\u0641 \u0627\u0644\u0645\u0648\u0638\u0641\u064a\u0646\u060c \u0644\u064a\u064f\u0639\u0627\u062f \u062f\u0645\u062c 310 \u0645\u0646\u0647\u0645 \u0641\u064a "\u0631\u0648\u0634". \u0645\u0646\u0630 \u0623\u0646 \u0623\u0639\u0644\u0646\u062a \u0627\u0644\u0634\u0631\u0643\u0629 \u0641\u064a \u0645\u0627\u0631\u0633\/\u0622\u0630\u0627\u0631 \u0639\u062f\u0645 \u0648\u062c\u0648\u062f "\u0641\u0648\u0627\u0626\u062f \u062a\u0622\u0632\u0631\u064a\u0629 \u0645\u0633\u062a\u0642\u0628\u0644\u064a\u0629 \u0643\u0628\u064a\u0631\u0629" \u0645\u0646 \u0648\u062c\u0648\u062f "\u0633\u0628\u0627\u0631\u0643"\u060c \u064a\u064f\u0639\u062a\u0642\u062f \u0623\u0646 \u0647\u0630\u0627 \u0627\u0644\u0625\u062c\u0631\u0627\u0621 \u062a\u0645 \u0627\u062a\u062e\u0627\u0630\u0647 \u0644\u062a\u062c\u0646\u0628 \u0623\u064a \u062e\u0633\u0627\u0626\u0631.<\/p>\n\n\n\n
\u0627\u0644\u0645\u0631\u0643\u0628\u0627\u062a \u0627\u0644\u062c\u0648\u064a\u0629 \u0630\u0627\u062a\u064a\u0629 \u0627\u0644\u0642\u064a\u0627\u062f\u0629 \u0645\u0642\u064a\u062f\u0629 \u0628\u062a\u0643\u0627\u0644\u064a\u0641 \u0627\u0644\u062a\u0635\u0646\u064a\u0639 \u0648\u062d\u062c\u0645 \u0627\u0644\u062d\u0645\u0648\u0644\u0629<\/h2>\n\n\n\n
\u0648\u0641\u0642\u064b\u0627 \u0644\u0634\u0627\u0645\u0628\u0648\u0646\u060c \u064a\u064f\u0639\u062f\u0651 \u0627\u0646\u062e\u0641\u0627\u0636 \u0627\u0644\u0637\u0644\u0628 \u0639\u0644\u0649 \u0646\u0627\u0642\u0644\u0627\u062a \u0627\u0644\u0623\u0645\u0631\u0627\u0636 \u0627\u0644\u0641\u064a\u0631\u0648\u0633\u064a\u0629 \u0627\u0644\u0645\u0631\u062a\u0628\u0637\u0629 \u0628\u0627\u0644\u0646\u0627\u0642\u0644\u0627\u062a (AAVs) \u0628\u0633\u0628\u0628 \u0635\u063a\u0631 \u062d\u062c\u0645 \u062d\u0645\u0648\u0644\u062a\u0647\u0627 \u0623\u062d\u062f \u0623\u0633\u0628\u0627\u0628 \u0627\u0646\u062e\u0641\u0627\u0636 \u0627\u0644\u0637\u0644\u0628 \u0639\u0644\u064a\u0647\u0627. \u0641\u064a \u0627\u0644\u0648\u0627\u0642\u0639\u060c \u062a\u0633\u062a\u0637\u064a\u0639 \u0646\u0627\u0642\u0644\u0627\u062a \u0627\u0644\u0623\u0645\u0631\u0627\u0636 \u0627\u0644\u0641\u064a\u0631\u0648\u0633\u064a\u0629 \u0627\u0644\u0645\u0631\u062a\u0628\u0637\u0629 \u0628\u0627\u0644\u0646\u0627\u0642\u0644\u0627\u062a (AAVs) \u062d\u0645\u0644 \u062d\u0645\u0648\u0644\u0627\u062a \u0623\u0643\u0628\u0631 \u0628\u0643\u062b\u064a\u0631 \u0645\u0646 \u0646\u0627\u0642\u0644\u0627\u062a \u0627\u0644\u0623\u0645\u0631\u0627\u0636 \u0627\u0644\u0641\u064a\u0631\u0648\u0633\u064a\u0629 \u0648\u063a\u064a\u0631 \u0627\u0644\u0641\u064a\u0631\u0648\u0633\u064a\u0629 \u0627\u0644\u0623\u062e\u0631\u0649\u060c \u0628\u0633\u0639\u0629 \u062a\u062d\u0645\u064a\u0644 \u062a\u0628\u0644\u063a \u062d\u0648\u0627\u0644\u064a 4.7 \u0643\u064a\u0644\u0648 \u0628\u0627\u064a\u062a. \u0648\u0642\u062f \u062a\u0645\u062a\u0644\u0643 \u0646\u0627\u0642\u0644\u0627\u062a \u0623\u062e\u0631\u0649 \u062c\u064a\u0646\u0627\u062a \u0645\u062a\u0639\u062f\u062f\u0629.<\/p>\n\n\n\n
\u0635\u0631\u062d \u0634\u0627\u0645\u0628\u0648\u0646 \u0623\u0646 \u062a\u0637\u0648\u064a\u0631 \u0645\u064a\u0643\u0631\u0648\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0641\u0639\u0627\u0644 \u0645\u0646 AAV\u060c \u0648\u0647\u0648 \u0634\u0643\u0644 \u0645\u0643\u062b\u0641 \u0645\u0646 \u0628\u0631\u0648\u062a\u064a\u0646 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0627\u0644\u0645\u0633\u062a\u062e\u062f\u0645 \u0641\u064a \u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a \u0644\u0645\u0631\u0636 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a\u060c \u0627\u0633\u062a\u063a\u0631\u0642 \u0633\u0646\u0648\u0627\u062a\u060c \u0648\u0623\u0646\u0647 \u0642\u062f \u0644\u0627 \u064a\u0632\u0627\u0644 \u0628\u062d\u0627\u062c\u0629 \u0625\u0644\u0649 \u062a\u062d\u0633\u064a\u0646. \u0648\u0623\u0636\u0627\u0641: "\u064a\u064f\u0639\u062f \u062d\u062c\u0645 \u0627\u0644\u062d\u0645\u0648\u0644\u0629 \u0645\u0647\u0645\u064b\u0627 \u0644\u062a\u062d\u0633\u064a\u0646 \u0627\u0644\u062a\u062d\u0643\u0645 \u0641\u064a \u0627\u0644\u062a\u0639\u0628\u064a\u0631 \u0627\u0644\u062c\u064a\u0646\u064a \u0627\u0644\u0639\u0644\u0627\u062c\u064a \u0644\u0644\u062d\u0645\u0636 \u0627\u0644\u0646\u0648\u0648\u064a\u060c \u0648\u062f\u0645\u062c \u0627\u0644\u062a\u0633\u0644\u0633\u0644\u0627\u062a \u0627\u0644\u0643\u0627\u0645\u0644\u0629 \u0644\u0644\u0628\u0631\u0648\u062a\u064a\u0646\u0627\u062a \u0627\u0644\u0643\u0628\u064a\u0631\u0629". \u0628\u0627\u0644\u0625\u0636\u0627\u0641\u0629 \u0625\u0644\u0649 \u0630\u0644\u0643\u060c \u062a\u064f\u062c\u0631\u0649 \u0645\u062d\u0627\u0648\u0644\u0627\u062a \u0644\u062a\u0637\u0648\u064a\u0631 \u062a\u0631\u0643\u064a\u0628\u0627\u062a AAV \u062b\u0646\u0627\u0626\u064a\u0629 \u0648\u062b\u0644\u0627\u062b\u064a\u0629 \u0644\u0644\u062a\u063a\u0644\u0628 \u0639\u0644\u0649 \u0645\u0634\u0643\u0644\u0629 \u062d\u062c\u0645 \u0627\u0644\u062d\u0645\u0648\u0644\u0629\u060c \u0644\u0643\u0646\u0647\u0627 \u0644\u064a\u0633\u062a \u0627\u0644\u062d\u0644 \u0627\u0644\u0623\u0645\u062b\u0644 \u0644\u0623\u0646\u0647\u0627 \u062a\u062a\u0637\u0644\u0628 \u062c\u0631\u0639\u0627\u062a \u0623\u0643\u0628\u0631 \u0648\u0641\u0639\u0627\u0644\u064a\u0629 \u0623\u0642\u0644 \u0628\u0637\u0628\u064a\u0639\u062a\u0647\u0627.<\/p>\n\n\n\n
\u0639\u0644\u0627\u0648\u0629 \u0639\u0644\u0649 \u0630\u0644\u0643\u060c \u0623\u0648\u0636\u062d \u0634\u0627\u0645\u0628\u0648\u0646 \u0623\u0646 \u062a\u0648\u0633\u064a\u0639 \u0646\u0637\u0627\u0642 \u0627\u0644\u0627\u0633\u062a\u062e\u062f\u0627\u0645 \u0645\u0643\u0644\u0641 \u0646\u0638\u0631\u064b\u0627 \u0644\u062a\u0639\u0642\u064a\u062f \u062a\u0635\u0646\u064a\u0639 \u0646\u0648\u0627\u0642\u0644 \u0641\u064a\u0631\u0648\u0633\u0627\u062a \u0627\u0644\u063a\u062f\u0629 \u0627\u0644\u062f\u0631\u0642\u064a\u0629 \u0627\u0644\u0645\u0631\u062a\u0628\u0637\u0629 \u0628\u0627\u0644\u0647\u0631\u0645\u0648\u0646\u0627\u062a (AAV). \u0644\u0630\u0627\u060c \u0641\u0625\u0646 \u0627\u0644\u0628\u062d\u062b \u0639\u0646 \u0641\u064a\u0631\u0648\u0633\u0627\u062a \u063a\u062f\u0627\u0626\u064a\u0629 \u0645\u0631\u062a\u0628\u0637\u0629 \u0628\u0627\u0644\u0647\u0631\u0645\u0648\u0646\u0627\u062a (AAV) \u0645\u064f\u062d\u0633\u0651\u0646\u0629\u060c \u0648\u0631\u0628\u0645\u0627 \u0646\u0648\u0627\u0642\u0644 \u0623\u062e\u0631\u0649 \u063a\u064a\u0631 \u0641\u064a\u0631\u0648\u0633\u0627\u062a \u0627\u0644\u063a\u062f\u0629 \u0627\u0644\u062f\u0631\u0642\u064a\u0629 \u0627\u0644\u0645\u0631\u062a\u0628\u0637\u0629 \u0628\u0627\u0644\u0647\u0631\u0645\u0648\u0646\u0627\u062a (AAV)\u060c \u0623\u0645\u0631\u064c \u0637\u0627\u0644 \u0627\u0646\u062a\u0638\u0627\u0631\u0647.<\/p>\n\n\n\n
\u064a\u062a\u0639\u0644\u0645 \u0623\u0643\u062b\u0631: \u0645\u0645\u062b\u0644 DMD WarrioR \u0627\u0644\u062a\u0631\u0643\u064a \u064a\u0634\u0627\u0631\u0643 \u0622\u0631\u0627\u0621\u0647 \u062d\u0648\u0644 \u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a Elevidys: \u0647\u0644 \u0647\u0648 \u0641\u0639\u0627\u0644 \u0648\u0644\u0645\u0627\u0630\u0627 \u0647\u0648 \u0645\u0643\u0644\u0641\u061f<\/a><\/p>\n\n\n\n\u0645\u0627 \u0648\u0631\u0627\u0621 \u0623\u0628\u062d\u0627\u062b AAV: \u0627\u0644\u0628\u062d\u062b \u0639\u0646 \u0637\u0631\u0642 \u0623\u0643\u062b\u0631 \u0641\u0639\u0627\u0644\u064a\u0629 \u0644\u062a\u0648\u0635\u064a\u0644 \u0627\u0644\u0623\u062f\u0648\u064a\u0629<\/h2>\n\n\n\n
\u0639\u0644\u0649 \u0633\u0628\u064a\u0644 \u0627\u0644\u0645\u062b\u0627\u0644\u060c \u0639\u0644\u0649 \u0627\u0644\u0631\u063a\u0645 \u0645\u0646 \u0642\u0644\u0629 \u0627\u0647\u062a\u0645\u0627\u0645 Vertex Pharmaceuticals\u060c \u062a\u064f\u0643\u0631\u0651\u0633 Affinia Therapeutics \u062c\u0647\u0648\u062f\u0647\u0627 \u0644\u0646\u0634\u0631 \u0645\u0631\u0643\u0628\u0627\u062a\u0647\u0627 \u0627\u0644\u0645\u0636\u0627\u062f\u0629 \u0644\u0644\u0641\u064a\u0631\u0648\u0633\u0627\u062a \u0627\u0644\u063a\u062f\u064a\u0629 (AAVs) \u0639\u0644\u0649 \u0646\u0637\u0627\u0642 \u0623\u0648\u0633\u0639. \u0648\u0644\u062a\u062d\u0633\u064a\u0646 \u0627\u0633\u062a\u0647\u062f\u0627\u0641 \u0627\u0644\u0623\u0646\u0633\u062c\u0629 \u0648\u0627\u0644\u062e\u0644\u0627\u064a\u0627 \u0627\u0644\u062a\u064a \u062a\u064f\u0639\u062f\u0651 \u0645\u0648\u0627\u0642\u0639 \u0627\u0644\u0641\u0639\u0627\u0644\u064a\u0629 \u0627\u0644\u0645\u0631\u063a\u0648\u0628\u0629 \u0645\u0639 \u062a\u062c\u0646\u0628 \u0645\u0648\u0627\u0642\u0639 \u0627\u0644\u0633\u0645\u064a\u0629\u060c \u064a\u062c\u0631\u064a \u062a\u0635\u0645\u064a\u0645 \u0643\u0628\u0633\u0648\u0644\u0627\u062a. \u062a\u064f\u062f\u0645\u062c \u0627\u0644\u0628\u0628\u062a\u064a\u062f\u0627\u062a \u0627\u0644\u0642\u0635\u064a\u0631\u0629 \u0641\u064a \u0627\u0644\u0634\u064a\u0641\u0631\u0629 \u0627\u0644\u0648\u0631\u0627\u062b\u064a\u0629 \u0644\u0640 AAV \u0644\u0625\u0639\u0627\u062f\u0629 \u0647\u0646\u062f\u0633\u0629 AAV9. \u0648\u0628\u064a\u0646\u0645\u0627 \u062a\u0633\u0639\u0649 \u0627\u0644\u0634\u0631\u0643\u0629 \u0625\u0644\u0649 \u062a\u0631\u062e\u064a\u0635 \u0643\u0628\u0633\u0648\u0644\u0627\u062a\u0647\u0627 \u0644\u0634\u0631\u0643\u0627\u062a \u0627\u0644\u062a\u0643\u0646\u0648\u0644\u0648\u062c\u064a\u0627 \u0627\u0644\u062d\u064a\u0648\u064a\u0629 \u0627\u0644\u0623\u062e\u0631\u0649\u060c \u0641\u0625\u0646\u0647\u0627 \u062a\u062a\u0637\u0644\u0639 \u0623\u064a\u0636\u064b\u0627 \u0625\u0644\u0649 \u0627\u0644\u0639\u0645\u0644 \u0645\u0639 \u0627\u0644\u0645\u0624\u0633\u0633\u0627\u062a \u0627\u0644\u062c\u0627\u0645\u0639\u064a\u0629 \u0648\u0634\u0631\u0643\u0627\u062a \u0627\u0644\u0623\u062f\u0648\u064a\u0629 \u0627\u0644\u062d\u064a\u0648\u064a\u0629 \u0627\u0644\u0623\u062e\u0631\u0649 \u0644\u0632\u064a\u0627\u062f\u0629 \u0633\u0639\u0629 \u0627\u0644\u062d\u0645\u0648\u0644\u0629.<\/p>\n\n\n\n
\u0641\u064a \u063a\u0636\u0648\u0646 \u0630\u0644\u0643\u060c \u0634\u0643\u0651\u0644\u062a \u0646\u0648\u0627\u0642\u0644 \u0641\u064a\u0631\u0648\u0633 \u0627\u0644\u0647\u0631\u0628\u0633 \u0627\u0644\u0628\u0633\u064a\u0637-1 (HSV-1) \u063a\u064a\u0631 \u0627\u0644\u0645\u062a\u0643\u0627\u062b\u0631\u0629 \u0623\u0633\u0627\u0633\u064b\u0627 \u0644\u062e\u0637 \u0625\u0646\u062a\u0627\u062c \u0627\u0644\u0623\u062f\u0648\u064a\u0629 \u0627\u0644\u062c\u064a\u0646\u064a\u0629 \u0644\u0634\u0631\u0643\u0629 \u0634\u0627\u0645\u0628\u0648\u0646 EG 427. \u0628\u0627\u0644\u0645\u0642\u0627\u0631\u0646\u0629 \u0645\u0639 \u0627\u0644\u0646\u0648\u0627\u0642\u0644 \u0627\u0644\u0641\u064a\u0631\u0648\u0633\u064a\u0629 \u0627\u0644\u0623\u062e\u0631\u0649\u060c \u062a\u062d\u0645\u0644 \u0647\u0630\u0647 \u0627\u0644\u0646\u0648\u0627\u0642\u0644 \u062d\u0645\u0648\u0644\u0629 \u062c\u064a\u0646\u064a\u0629 \u0623\u0643\u0628\u0631 \u062a\u0628\u0644\u063a \u062d\u0648\u0627\u0644\u064a 30 \u0643\u064a\u0644\u0648 \u0628\u0627\u064a\u062a. \u0648\u0642\u062f \u062d\u0627\u0641\u0638\u062a \u062d\u062a\u0649 \u0627\u0644\u0622\u0646 \u0639\u0644\u0649 \u0645\u0633\u062a\u0648\u0649 \u0623\u0645\u0627\u0646 \u0639\u0627\u0644\u064d \u0628\u0641\u0636\u0644 \u0627\u0643\u062a\u0634\u0627\u0641 \u0642\u062f\u0631\u062a\u0647\u0627 \u0639\u0644\u0649 \u062a\u062b\u0628\u064a\u0637 \u0627\u0644\u0627\u0633\u062a\u062c\u0627\u0628\u0627\u062a \u0627\u0644\u0645\u0646\u0627\u0639\u064a\u0629.<\/p>\n\n\n\n
\u0646\u0639\u062a\u0642\u062f \u0623\u0646 \u0647\u0630\u0647 \u0627\u0644\u0627\u0633\u062a\u0631\u0627\u062a\u064a\u062c\u064a\u0629 \u062a\u064f\u0642\u062f\u0645 \u0628\u062f\u064a\u0644\u0627\u064b \u0623\u062b\u0628\u062a \u0633\u0644\u0627\u0645\u062a\u0647 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0633\u0627\u0628\u0642\u064b\u0627\u060c \u0648\u064a\u062a\u064a\u062d \u0625\u0639\u0627\u062f\u0629 \u062a\u062d\u062f\u064a\u062f \u0627\u0644\u062c\u0631\u0639\u0627\u062a\u060c \u0648\u064a\u064f\u0633\u0647\u0651\u0644 \u062a\u0648\u0633\u064a\u0639 \u0646\u0637\u0627\u0642 \u0627\u0644\u0625\u0646\u062a\u0627\u062c \u0627\u0642\u062a\u0635\u0627\u062f\u064a\u064b\u0627\u060c \u0648\u064a\u062a\u0645\u062a\u0639 \u0628\u0633\u0639\u0629 \u062d\u0645\u0648\u0644\u0629 \u0623\u0643\u0628\u0631 \u0628\u0633\u062a\u0629 \u0623\u0636\u0639\u0627\u0641 \u0639\u0644\u0649 \u0627\u0644\u0623\u0642\u0644 \u0645\u0646 \u0644\u0642\u0627\u062d AAV. \u0648\u0642\u062f \u062b\u0628\u062a \u0639\u0644\u0645\u064a\u064b\u0627 \u0623\u0646 \u0627\u0644\u0646\u0648\u0627\u0642\u0644 \u0627\u0644\u0642\u0627\u0626\u0645\u0629 \u0639\u0644\u0649 \u0641\u064a\u0631\u0648\u0633 \u0627\u0644\u0647\u0631\u0628\u0633 \u0627\u0644\u0628\u0633\u064a\u0637 \u0642\u0627\u0628\u0644\u0629 \u0644\u0625\u0639\u0627\u062f\u0629 \u062a\u062d\u062f\u064a\u062f \u0627\u0644\u062c\u0631\u0639\u0627\u062a \u0628\u0623\u0645\u0627\u0646 \u0645\u0646 \u0642\u0650\u0628\u0644 \u0634\u0631\u0643\u0627\u062a \u0623\u062e\u0631\u0649\u060c \u0648\u0623\u0628\u0631\u0632\u0647\u0627 \u0644\u0642\u0627\u062d Krystal Bio\u060c \u0643\u0645\u0627 \u0623\u0634\u0627\u0631 \u0634\u0627\u0645\u0628\u0648\u0646.<\/strong><\/p>\n\n\n\n\u0646\u0638\u0631\u0627\u064b \u0644\u0623\u0646 \u0645\u0641\u0648\u0636 \u0627\u0644\u0644\u062c\u0646\u0629 FDA\u060c \u0645\u0627\u0631\u062a\u064a \u0645\u0627\u0643\u0627\u0631\u064a\u060c \u0623\u0639\u0644\u0646 \u0641\u064a \u0623\u0628\u0631\u064a\u0644 \u0639\u0646 \u062c\u0647\u0648\u062f \u0644\u062a\u0633\u0631\u064a\u0639 \u0627\u0644\u0645\u0648\u0627\u0641\u0642\u0627\u062a \u0639\u0644\u0649 \u0623\u062f\u0648\u064a\u0629 \u0627\u0644\u0623\u0645\u0631\u0627\u0636 \u0627\u0644\u0646\u0627\u062f\u0631\u0629 - \u0648\u0627\u0644\u062a\u064a \u064a\u0633\u062a\u062e\u062f\u0645 \u0627\u0644\u0643\u062b\u064a\u0631 \u0645\u0646\u0647\u0627 \u0639\u0644\u0627\u062c\u0627\u062a \u062c\u064a\u0646\u064a\u0629 \u0639\u0644\u0649 \u0623\u064a \u062d\u0627\u0644 - \u0641\u0645\u0646 \u063a\u064a\u0631 \u0627\u0644\u0645\u0631\u062c\u062d \u0623\u0646 \u064a\u064f\u0628\u0637\u0626 \u0630\u0644\u0643 \u0623\u0628\u062d\u0627\u062b \u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a \u0628\u0634\u0643\u0644 \u0639\u0627\u0645\u060c \u0645\u0639 \u0627\u0633\u062a\u0645\u0631\u0627\u0631 \u0627\u0644\u0628\u062d\u062b \u0639\u0646 \u0628\u062f\u0627\u0626\u0644 \u0644\u0641\u064a\u0631\u0648\u0633\u0627\u062a \u0627\u0644\u063a\u062f\u0651\u0629 \u0627\u0644\u0646\u062e\u0627\u0645\u064a\u0629 (AAVs) \u0627\u0644\u062d\u0627\u0644\u064a\u0629. \u0648\u0645\u0639 \u0630\u0644\u0643\u060c \u0639\u0644\u064a\u0646\u0627 \u0623\u0646 \u0646\u0646\u062a\u0638\u0631 \u0644\u0646\u0631\u0649 \u0625\u0644\u0649 \u0623\u064a\u0646 \u064a\u062a\u062c\u0647 \u0642\u0637\u0627\u0639 \u062a\u0648\u0635\u064a\u0644 \u0627\u0644\u0623\u062f\u0648\u064a\u0629 \u0641\u064a \u0627\u0644\u0633\u0646\u0648\u0627\u062a \u0627\u0644\u0642\u0627\u062f\u0645\u0629.<\/p>\n\n\n\n
\u0627\u0643\u062a\u0634\u0641 \u0627\u0644\u0645\u0632\u064a\u062f: \u0642\u062f \u064a\u0643\u0648\u0646 \u0645\u0646 \u0627\u0644\u0645\u0645\u0643\u0646 \u0627\u0633\u062a\u062e\u062f\u0627\u0645 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0643\u0627\u0645\u0644 \u0627\u0644\u0637\u0648\u0644 \u0644\u0639\u0644\u0627\u062c \u0645\u0631\u0636 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a<\/a><\/p>","protected":false},"excerpt":{"rendered":"Adeno-associated virus (AAV) vectors were once heralded as the answer to delivering gene therapies, but in recent years, their prominence has diminished. It was first hailed as having the ability to treat several hereditary illnesses, but the decline in research has already been recognized. Vertex Pharmaceuticals is the most recent biopharma to reduce its investment […]<\/p>\n","protected":false},"author":1,"featured_media":3843,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[282],"tags":[368,359,61],"class_list":["post-3833","post","type-post","status-publish","format-standard","has-post-thumbnail","category-editorials","tag-aav-based-gene-therapy","tag-full-length-dystrophin","tag-gene-therapy"],"subtitle":"\u0644\u0642\u062f \u062a\u0631\u0627\u062c\u0639\u062a \u0627\u0644\u0623\u0628\u062d\u0627\u062b \u0627\u0644\u0645\u062a\u0639\u0644\u0642\u0629 \u0628\u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a \u0644\u0645\u0631\u0636 \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u062f\u0648\u0634\u064a\u0646 (DMD) \u0627\u0644\u0645\u0633\u062a\u0646\u062f \u0625\u0644\u0649 AAV \u0639\u0646 \u0627\u0644\u0627\u0647\u062a\u0645\u0627\u0645 \u0628\u0633\u0628\u0628 \u062a\u0643\u0644\u0641\u062a\u0647 \u0627\u0644\u0639\u0627\u0644\u064a\u0629 \u0648\u0641\u0639\u0627\u0644\u064a\u062a\u0647 \u0627\u0644\u0645\u062d\u062f\u0648\u062f\u0629\u060c \u062d\u064a\u062b \u0645\u0646 \u0627\u0644\u0645\u062a\u0648\u0642\u0639 \u0623\u0646 \u062a\u0631\u0643\u0632 \u0634\u0631\u0643\u0627\u062a \u0627\u0644\u0623\u062f\u0648\u064a\u0629 \u0639\u0644\u0649 \u062a\u0637\u0648\u064a\u0631 \u0627\u0644\u0623\u062f\u0648\u064a\u0629 \u0627\u0644\u0642\u0627\u0626\u0645\u0629 \u0639\u0644\u0649 tRNA \u0648mRNA \u0644\u0625\u0646\u062a\u0627\u062c \u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0643\u0627\u0645\u0644 \u0627\u0644\u0637\u0648\u0644 \u0628\u062a\u0643\u0627\u0644\u064a\u0641 \u0623\u0642\u0644.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts\/3833","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/comments?post=3833"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts\/3833\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/media\/3843"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/media?parent=3833"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/categories?post=3833"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/tags?post=3833"}],"curies":[{"name":"\u0648\u0648\u0631\u062f\u0628\u0631\u064a\u0633","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}