Ontology highlight
ABSTRACT:
SUBMITTER: Chamberlain JS
PROVIDER: S-EPMC10210223 | biostudies-literature | 2023 May
REPOSITORIES: biostudies-literature
Chamberlain Jeffrey S JS Robb Melissa M Braun Serge S Brown Kristy J KJ Danos Olivier O Ganot Annie A Gonzalez-Alegre Pedro P Hunter Nina N McDonald Craig C Morris Carl C Tobolowsky Mark M Wagner Kathryn R KR Ziolkowski Olivia O Duan Dongsheng D
Human gene therapy 20230314 9-10
Duchenne muscular dystrophy (DMD) is a serious, rare genetic disease, affecting primarily boys. It is caused by mutations in the <i>DMD</i> gene and is characterized by progressive muscle degeneration that results in loss of function and early death due to respiratory and/or cardiac failure. Although limited treatment options are available, some for only small subsets of the patient population, DMD remains a disease with large unmet medical needs. The adeno-associated virus (AAV) vector is the l ...[more]