Development of a CRISPR/Cas9-based therapy for Hutchinson-Gilford progeria syndrome

CRISPR/Cas9-based therapies hold an important promise for the treatment of genetic diseases. Among these, Hutchinson-Gilford progeria syndrome (HGPS) – caused by a point mutation in the LMNA gene – stands out as a potential candidate. Here, we explore the efficacy of a CRISPR/Cas9-based approach tha...

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Detalles Bibliográficos
Autores principales: Santiago-Fernández, Olaya, Osorio, Fernando G., Quesada, Víctor, Rodríguez, Francisco, Basso, Sammy, Maeso, Daniel, Rolas, Loïc, Barkaway, Anna, Nourshargh, Sussan, Folgueras, Alicia R., Freije, José M. P., López-Otín, Carlos
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6546610/
https://www.ncbi.nlm.nih.gov/pubmed/30778239
http://dx.doi.org/10.1038/s41591-018-0338-6
Descripción
Sumario:CRISPR/Cas9-based therapies hold an important promise for the treatment of genetic diseases. Among these, Hutchinson-Gilford progeria syndrome (HGPS) – caused by a point mutation in the LMNA gene – stands out as a potential candidate. Here, we explore the efficacy of a CRISPR/Cas9-based approach that reverts several alterations in HGPS cells and mice by introducing frameshift mutations in the LMNA gene.

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