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Transient expression of an adenine base editor corrects the Hutchinson-Gilford progeria syndrome mutation and improves the skin phenotype in mice

Hutchinson-Gilford progeria syndrome (HGPS) is a rare premature ageing disorder caused by a point mutation in the LMNA gene (LMNA c.1824 C > T), resulting in the production of a detrimental protein called progerin. Adenine base editors recently emerged with a promising potential for HGPS gene the...

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Detalles Bibliográficos
Autores principales: Whisenant, Daniel, Lim, Kayeong, Revêchon, Gwladys, Yao, Haidong, Bergo, Martin O., Machtel, Piotr, Kim, Jin-Soo, Eriksson, Maria
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9163128/
https://www.ncbi.nlm.nih.gov/pubmed/35654881
http://dx.doi.org/10.1038/s41467-022-30800-y