Treatment of a Mouse Model of ALS by In Vivo Base Editing

Amyotrophic lateral sclerosis (ALS) is a debilitating and fatal disorder that can be caused by mutations in the superoxide dismutase 1 (SOD1) gene. Although ALS is currently incurable, CRISPR base editors hold the potential to treat the disease through their ability to create nonsense mutations that...

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Enregistré dans:
Détails bibliographiques
Publié dans:Mol Ther
Auteurs principaux: Lim, Colin K.W., Gapinske, Michael, Brooks, Alexandra K., Woods, Wendy S., Powell, Jackson E., Zeballos C., M. Alejandra, Winter, Jackson, Perez-Pinera, Pablo, Gaj, Thomas
Format: Artigo
Langue:Inglês
Publié: American Society of Gene & Cell Therapy 2020
Sujets:
Original Article
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC7132599/
https://ncbi.nlm.nih.gov/pubmed/31991108
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.01.005
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