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Adenine base editing to treat progeria syndrome and extend the lifespan

Hutchinson-Gilford progeria syndrome (HGPS) is an exceedingly rare and hitherto incurable and fatal disease marked by accelerated aging simultaneously affecting a number of organs. Most cases of HGPS are caused by a single copy of a specific single-nucleotide mutation, c.C1824T, in the LMNA (lamin A...

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Detalles Bibliográficos
Autor principal: Musunuru, Kiran
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8302045/
https://www.ncbi.nlm.nih.gov/pubmed/34308436
http://dx.doi.org/10.20517/jca.2021.10