In vivo targeting of a variant causing vanishing white matter using CRISPR/Cas9

Vanishing white matter (VWM) is a leukodystrophy caused by recessive variants in subunits of eIF2B. At present, no curative treatment is available and patients often die at young age. Due to its monogenic nature, VWM is a promising candidate for the development of CRISPR/Cas9-mediated gene therapy....

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Detalles Bibliográficos
Autores principales: Hillen, Anne E.J., Hruzova, Martina, Rothgangl, Tanja, Breur, Marjolein, Bugiani, Marianna, van der Knaap, Marjo S., Schwank, Gerald, Heine, Vivi M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8917273/
https://www.ncbi.nlm.nih.gov/pubmed/35317047
http://dx.doi.org/10.1016/j.omtm.2022.02.006

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8917273/
https://www.ncbi.nlm.nih.gov/pubmed/35317047
http://dx.doi.org/10.1016/j.omtm.2022.02.006

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