RECOMBINANT ADENO-ASSOCIATED VIRUS DELIVERY OF ALPHA-SARCOGLYCAN POLYNUCLEOTIDES
摘要
The present invention relates to recombinant adeno-associated virus (rAAV) delivery of an alpha-sarcoglycan gene. The invention provides rAAV products and methods of using the rAAV in the treatment of limb girdle muscular dystrophies such as LGMD2D.
申请公布号
WO2013078316(A1)
申请公布日期
2013.05.30
申请号
WO2012US66265
申请日期
2012.11.21
申请人
NATIONWIDE CHILDREN'S HOSPITAL, INC.
发明人
MENDELL, JERRY, R.;CHICOINE, LOUIS;RODINO-KLAPAC, LOUISE;CLARK, KELLY, REED;PRESTON, THOMAS, J.
