RESEARCH INSTITUTE AT NATIONWIDE CHILDREN’S HOSPITAL
发明人:
RODINO-KLAPAC, LOUISE R.
申请号：
ARP190100213
公开号：
AR114350A1
申请日:
2019.01.30
申请国别(地区):
AR
年份:
2020
代理人:
摘要：
Carriers of genetic therapy, such as AAV. These include a polynuclear that encodes scabies (sgcg) and methods of using this gene therapy vector to treat patients with muscle failure, such as type 2C belt muscular atrophy (LGMD2C).Vectores de terapia genética tales como vectores AAV. que comprenden un polinucleótido que codifica sarcoglicano (SGCG) y métodos para usar tales vectores de terapia genética para el tratamiento de sujetos que sufren de distrofia muscular, por ejemplo distrofia muscular de cinturas del tipo 2C (LGMD2C).
