Exons 45–55 Skipping Using Mutation-Tailored Cocktails of Antisense Morpholinos in the DMD Gene

Mutations in the dystrophin (DMD) gene and consequent loss of dystrophin cause Duchenne muscular dystrophy (DMD). A promising therapy for DMD, single-exon skipping using antisense phosphorodiamidate morpholino oligomers (PMOs), currently confronts major issues in that an antisense drug induces the p...

ver descrição completa

Na minha lista:
Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Echigoya, Yusuke, Lim, Kenji Rowel Q., Melo, Dyanna, Bao, Bo, Trieu, Nhu, Mizobe, Yoshitaka, Maruyama, Rika, Mamchaoui, Kamel, Tanihata, Jun, Aoki, Yoshitsugu, Takeda, Shin’ichi, Mouly, Vincent, Duddy, William, Yokota, Toshifumi
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2019
Assuntos:
Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6838919/
https://ncbi.nlm.nih.gov/pubmed/31416775
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2019.07.012
Tags: Adicionar Tag
Sem tags, seja o primeiro a adicionar uma tag!

Registos relacionados