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...

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Bibliografiset tiedot
Julkaisussa:Science
Päätekijät: 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.
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: 2015
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Article
Linkit: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
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