Terapia gênica com folistatina em macacos sem doença muscular

USA - a folistatina antagoniza o efeito da miostatina e aumenta a força muscular; camundongos tratados com gene da folistatina apresentam aumento da massa muscular. Nesta pesquisa macacos sem doença muscular receberam o gene da folistatina através de um vetor viral. Os animais apresentaram aumento da força e da massa muscular. Não houve nenhum efeito colateral detectado com este tratamento.

O resumo da pesquisa pode ser lido abaixo:

(Science Translational Medicine, Nov 2009; 1: 6ra15) Follistatin Gene Delivery Enhances Muscle Growth and Strength in Nonhuman Primates

Janaiah Kota, Chalonda R. Handy, Amanda M. Haidet, Chrystal L. Montgomery, Amy Eagle, Louise R. Rodino-Klapac, Danielle Tucker, Christopher J. Shilling, Walter R. Therlfall, Christopher M. Walker, Steven E. Weisbrode, Paul M. L. Janssen, K. Reed Clark, Zarife Sahenk, Jerry R. Mendell, and Brian K. Kaspar

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 therapy with AAV1-FS344 may improve muscle mass and function in patients with certain degenerative muscle disorders.

Fonte: http://distrofiamuscular.net/noticias.htm