NATIONWIDE CHILDRENS HOSPITAL;NATIONWIDE CHILDREN'S HOSPITAL; INC.
发明人:
KASPAR, BRIAN K.,MENDELL, JERRY R.
申请号：
CA2693178
公开号：
CA2693178C
申请日:
2007.11.29
申请国别(地区):
CA
年份:
2018
代理人:
摘要：
The present invention relates to methods for inhibiting myostatin, a regulatorof muscle mass, for muscle enhancement(including inducing hypertrophy and/or hyperplasia) as well as improvingmuscle function (including decreasing atrophy and/orincreasing endurance, force and/or strength). The methods involve deliveringgenes to cells using gene delivery in order to inhibitmyostatin. Examples of genes to be delivered are genes encoding' proteins suchas Follistatin, Follistatin- related gene-1 (FLRG-I),growth differentiation factor associated protein- 1(GASP-I) and myostatinprecursor propeptide. The genes are delivered using arecombinant Adeno-associated virus (rAAV) lacking rep and cap DNA capable ofinfecting the cells. Following introduction, thegenes are expressed in the cell body of the infected cell and the encodedproteins are secreted systemically. In other methods-ofthe invention expression of proteins such as activin lib and myostatin isinhibited by oligonucleotide techniques to effect muscleenhancement. All the methods have applications in the treatment ofmusculoskeletal and neurodegenerative disorders among others,as well as enhancing muscle in livestock.
