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AAV1.NT-3 gene therapy in a CMT2D model: phenotypic improvements in Gars(P278KY/+) mice

Glycyl–tRNA synthetase mutations are associated to the Charcot–Marie–Tooth disease type-2D. The Gars(P278KY/+) model for Charcot–Marie–Tooth disease type-2D is known best for its early onset severe neuropathic phenotype with findings including reduced axon size, slow conduction velocities and abnorm...

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Detalles Bibliográficos
Autores principales: Ozes, Burcak, Moss, Kyle, Myers, Morgan, Ridgley, Alicia, Chen, Lei, Murrey, Darren, Sahenk, Zarife
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8568849/
https://www.ncbi.nlm.nih.gov/pubmed/34755111
http://dx.doi.org/10.1093/braincomms/fcab252