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...

Descripción completa

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
_version_ 1783423540683341824
author 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
author_facet 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
author_sort Santiago-Fernández, Olaya
collection PubMed
description 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.
format Online
Article
Text
id pubmed-6546610
institution National Center for Biotechnology Information
language English
publishDate 2019
record_format MEDLINE/PubMed
spelling pubmed-65466102019-08-18 Development of a CRISPR/Cas9-based therapy for Hutchinson-Gilford progeria syndrome 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 Nat Med Article 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. 2019-03-01 2019-02-18 /pmc/articles/PMC6546610/ /pubmed/30778239 http://dx.doi.org/10.1038/s41591-018-0338-6 Text en http://www.nature.com/authors/editorial_policies/license.html#terms Users may view, print, copy, and download text and data-mine the content in such documents, for the purposes of academic research, subject always to the full Conditions of use:http://www.nature.com/authors/editorial_policies/license.html#terms
spellingShingle Article
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
Development of a CRISPR/Cas9-based therapy for Hutchinson-Gilford progeria syndrome
title Development of a CRISPR/Cas9-based therapy for Hutchinson-Gilford progeria syndrome
title_full Development of a CRISPR/Cas9-based therapy for Hutchinson-Gilford progeria syndrome
title_fullStr Development of a CRISPR/Cas9-based therapy for Hutchinson-Gilford progeria syndrome
title_full_unstemmed Development of a CRISPR/Cas9-based therapy for Hutchinson-Gilford progeria syndrome
title_short Development of a CRISPR/Cas9-based therapy for Hutchinson-Gilford progeria syndrome
title_sort development of a crispr/cas9-based therapy for hutchinson-gilford progeria syndrome
topic Article
url 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
work_keys_str_mv AT santiagofernandezolaya developmentofacrisprcas9basedtherapyforhutchinsongilfordprogeriasyndrome
AT osoriofernandog developmentofacrisprcas9basedtherapyforhutchinsongilfordprogeriasyndrome
AT quesadavictor developmentofacrisprcas9basedtherapyforhutchinsongilfordprogeriasyndrome
AT rodriguezfrancisco developmentofacrisprcas9basedtherapyforhutchinsongilfordprogeriasyndrome
AT bassosammy developmentofacrisprcas9basedtherapyforhutchinsongilfordprogeriasyndrome
AT maesodaniel developmentofacrisprcas9basedtherapyforhutchinsongilfordprogeriasyndrome
AT rolasloic developmentofacrisprcas9basedtherapyforhutchinsongilfordprogeriasyndrome
AT barkawayanna developmentofacrisprcas9basedtherapyforhutchinsongilfordprogeriasyndrome
AT noursharghsussan developmentofacrisprcas9basedtherapyforhutchinsongilfordprogeriasyndrome
AT folguerasaliciar developmentofacrisprcas9basedtherapyforhutchinsongilfordprogeriasyndrome
AT freijejosemp developmentofacrisprcas9basedtherapyforhutchinsongilfordprogeriasyndrome
AT lopezotincarlos developmentofacrisprcas9basedtherapyforhutchinsongilfordprogeriasyndrome

Ejemplares similares