Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

CRISPR/Cas9-mediated genome editing holds clinical potential for treating genetic diseases, such as Duchenne muscular dystrophy (DMD), which is caused by mutations in the dystrophin gene. To correct DMD by skipping mutant dystrophin exons in postnatal muscle tissue in vivo, we used adeno-associated...

Cur síos iomlán

Na minha lista:
Sonraí Bibleagrafaíochta
Foilsithe in:Science
Main Authors: Long, Chengzu, Amoasii, Leonela, Mireault, Alex A., McAnally, John R., Li, Hui, Sanchez-Ortiz, Efrain, Bhattacharyya, Samadrita, Shelton, John M., Bassel-Duby, Rhonda, Olson, Eric N.
Formáid: Artigo
Teanga:Inglês
Foilsithe: 2015
Ábhair:
Article
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC4760628/
https://ncbi.nlm.nih.gov/pubmed/26721683
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/science.aad5725
Clibeanna: Cuir Clib Leis
Gan Chlibeanna, Bí ar an gcéad duine leis an taifead seo a chlibeáil!
Á lódáil...

Míreanna Comhchosúla