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

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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Bibliographic Details
Main Authors: Anne E.J. Hillen, Martina Hruzova, Tanja Rothgangl, Marjolein Breur, Marianna Bugiani, Marjo S. van der Knaap, Gerald Schwank, Vivi M. Heine
Format: Article
Language:English
Published: Elsevier 2022-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
vanishing white matter
leukodystrophy
CRISPR/Cas9
gene therapy
gene editing
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050122000225

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http://www.sciencedirect.com/science/article/pii/S2329050122000225

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