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gene therapy for muscle growth  


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Joined: 2 years ago
Posts: 1367
04/06/2020 6:56 am  

perhaps is not that far off. in the not too distant future, a few simple shots into the local muscle groups could result in dramatic changes.



Sci Transl Med. 2009 Nov 11;1(6):6ra15.

Follistatin gene delivery enhances muscle growth and strength in nonhuman primates.
Kota J, Handy CR, Haidet AM, Montgomery CL, Eagle A, Rodino-Klapac LR, Tucker D, Shilling CJ, Therlfall WR, Walker CM, Weisbrode SE, Janssen PM, Clark KR, Sahenk Z, Mendell JR, Kaspar BK.

Center for Gene Therapy, Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.

Comment in:

Sci Transl Med. 2009 Nov 11;1(6):6ps6.

Antagonists of myostatin, a blood-borne negative regulator of muscle growth produced in muscle cells, have shown considerable promise for enhancing muscle mass and strength in rodent studies and could serve as potential therapeutic agents for human muscle diseases. One of the most potent of these agents, follistatin, is both safe and effective in mice, but similar tests have not been performed in nonhuman primates. To assess this important criterion for clinical translation, we tested an alternatively spliced form of human follistatin that affects skeletal muscle but that has only minimal effects on nonmuscle cells. When injected into the quadriceps of cynomolgus macaque monkeys, a follistatin isoform expressed from an adeno-associated virus serotype 1 vector, AAV1-FS344, induced pronounced and durable increases in muscle size and strength. Long-term expression of the transgene did not produce any abnormal changes in the morphology or function of key organs, indicating the safety of gene delivery by intramuscular injection of an AAV1 vector. Our results, together with the findings in mice, suggest that[b] therapy with AAV1-FS344 may improve muscle mass and function in patients with certain degenerative muscle disorders.[\b]