Ontology highlight
ABSTRACT:
SUBMITTER: Wasala NB
PROVIDER: S-EPMC7054741 | biostudies-literature | 2020 Mar
REPOSITORIES: biostudies-literature
Wasala Nalinda B NB Yue Yongping Y Lostal William W Wasala Lakmini P LP Niranjan Nandita N Hajjar Roger J RJ Babu Gopal J GJ Duan Dongsheng D
Molecular therapy : the journal of the American Society of Gene Therapy 20200110 3
Loss of dystrophin leads to Duchenne muscular dystrophy (DMD). A pathogenic feature of DMD is the significant elevation of cytosolic calcium. Supraphysiological calcium triggers protein degradation, membrane damage, and eventually muscle death and dysfunction. Sarcoplasmic/endoplasmic reticulum (SR) calcium ATPase (SERCA) is a calcium pump that transports cytosolic calcium to the SR during excitation-contraction coupling. We hypothesize that a single systemic delivery of SERCA2a with adeno-assoc ...[more]