БЕРН Барри Джон (US),ФАЛК Дарин Дж. (US),ПЭКЭК Кристина (US),ДЕРУИССО Лара Роберт (US),МАХ Кэтрин (US),ФУЛЛЕР Дэвид Д. (US)
申请号：
RU2015101276
公开号：
RU2015101276A
申请日:
2013.06.19
申请国别(地区):
RU
年份:
2016
代理人:
摘要：
1. A method of improving impaired integrity of a neuromuscular compound, which comprises administering to a subject with impaired integrity of the neuromuscular compound an effective amount of a composition containing the rAAV2 / 9 vector comprising a heterologous nucleic acid molecule operably linked to a promoter, wherein said therapeutic composition is administered to the subject by intramuscular, intrathoracic, intraspinal, intracisternal, intrathecal or intravenous injection. 2. The method of claim 1, wherein the heterologous nucleic acid molecule encodes an acidic α-glucosidase (GAA). The method of claim 1, wherein the promoter is a cytomegalovirus (CMV) based promoter, a desmin based promoter (DES), a synapsin I based promoter (SYN), or a muscle creatine kinase (MSC) based promoter. The method of claim 1, wherein the subject has a neuromuscular junction caused by a neuromuscular disease. The method of claim 1, wherein the subject has a neuromuscular junction caused by a disease selected from the group consisting of Pompe disease, amyotrophic lateral sclerosis, atrophy of the spinal muscles, multiple sclerosis, type 1a glycogen accumulation disease, herpes zoster limb muscular dystrophy , Bart syndrome and myasthenia gravis. 6. The method of claim 1, wherein the subject is a human. The method of claim 2, further comprising administering to the subject an acetylcholinesterase inhibitor (ACI). A method of treating a neuromuscular disease, which comprises administering to a subject in need of such treatment an effective amount of a composition containing the rAAV2 / 9 vector comprising a heterologous nucleic acid molecule,1. Способ улучшения нарушенной целостности нервно-мышечного соединения, который включает введение субъекту с нарушенной целостностью нервно-мышечного соединения эффективного количества композиции, содержащей вектор rAAV2/9, включающий молекулу гетерологичной нуклеиновой кислоты, функционально связанную с промотором, при этом указанную терапевтич
