The University of Alberta researchers develop a molecule that bypasses genetic mutation to restore a crucial muscle-repairing protein. Learn more about OligomicsTx Gene Therapy Study…
A University of Alberta research team has taken the first step toward proving its innovative gene therapy can successfully treat a rare genetic condition called dysferlinopathy that impairs the body’s ability to repair damaged muscles.
In newly published research findings, the team reports it has developed a new antisense oligonucleotide to treat dysferlinopathy. The molecule effectively restored the function of the muscle-repairing protein dysferlin when used to treat cells from dysferlinopathy patients. The molecule triggers a process called “exon skipping” that allows cells to bypass defective genetic instructions and produce the protein needed to rebuild muscle tissue. [Read More]
“Our goal is to develop new therapeutics and to eventually cure muscular dystrophy and neuromuscular diseases using new technologies like antisense oligonucleotides and CRISPR genome editing to help patients walk again.” says medical genetics professor Toshifumi Yokota. [Who is Toshifumi Yokota?]
