Single-Dose CRISPR/Cas9 Therapy Extends Lifespan of Mice with Hutchinson-Gilford Progeria Syndrome

Hutchinson-Gilford Progeria Syndrome (HGPS) is a rare lethal genetic disorder characterized by symptoms reminiscent of accelerated aging. The major underlying genetic cause is a substitution mutation in the gene coding for lamin A, causing the production of a toxic isoform called progerin. Here we s...

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Autores principales: Beyret, Ergin, Liao, Hsin-Kai, Yamamoto, Mako, Hernandez-Benitez, Reyna, Fu, Yunpeng, Erikson, Galina, Reddy, Pradeep, Belmonte, Juan Carlos Izpisua
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6541418/
https://www.ncbi.nlm.nih.gov/pubmed/30778240
http://dx.doi.org/10.1038/s41591-019-0343-4
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author Beyret, Ergin
Liao, Hsin-Kai
Yamamoto, Mako
Hernandez-Benitez, Reyna
Fu, Yunpeng
Erikson, Galina
Reddy, Pradeep
Belmonte, Juan Carlos Izpisua
author_facet Beyret, Ergin
Liao, Hsin-Kai
Yamamoto, Mako
Hernandez-Benitez, Reyna
Fu, Yunpeng
Erikson, Galina
Reddy, Pradeep
Belmonte, Juan Carlos Izpisua
author_sort Beyret, Ergin
collection PubMed
description Hutchinson-Gilford Progeria Syndrome (HGPS) is a rare lethal genetic disorder characterized by symptoms reminiscent of accelerated aging. The major underlying genetic cause is a substitution mutation in the gene coding for lamin A, causing the production of a toxic isoform called progerin. Here we show that reduction of lamin A/progerin by a single dose systemic administration of AAV-delivered CRISPR/Cas9 components suppresses HGPS in a mouse model.
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spelling pubmed-65414182019-08-18 Single-Dose CRISPR/Cas9 Therapy Extends Lifespan of Mice with Hutchinson-Gilford Progeria Syndrome Beyret, Ergin Liao, Hsin-Kai Yamamoto, Mako Hernandez-Benitez, Reyna Fu, Yunpeng Erikson, Galina Reddy, Pradeep Belmonte, Juan Carlos Izpisua Nat Med Article Hutchinson-Gilford Progeria Syndrome (HGPS) is a rare lethal genetic disorder characterized by symptoms reminiscent of accelerated aging. The major underlying genetic cause is a substitution mutation in the gene coding for lamin A, causing the production of a toxic isoform called progerin. Here we show that reduction of lamin A/progerin by a single dose systemic administration of AAV-delivered CRISPR/Cas9 components suppresses HGPS in a mouse model. 2019-02-18 2019-03 /pmc/articles/PMC6541418/ /pubmed/30778240 http://dx.doi.org/10.1038/s41591-019-0343-4 Text en 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
Beyret, Ergin
Liao, Hsin-Kai
Yamamoto, Mako
Hernandez-Benitez, Reyna
Fu, Yunpeng
Erikson, Galina
Reddy, Pradeep
Belmonte, Juan Carlos Izpisua
Single-Dose CRISPR/Cas9 Therapy Extends Lifespan of Mice with Hutchinson-Gilford Progeria Syndrome
title Single-Dose CRISPR/Cas9 Therapy Extends Lifespan of Mice with Hutchinson-Gilford Progeria Syndrome
title_full Single-Dose CRISPR/Cas9 Therapy Extends Lifespan of Mice with Hutchinson-Gilford Progeria Syndrome
title_fullStr Single-Dose CRISPR/Cas9 Therapy Extends Lifespan of Mice with Hutchinson-Gilford Progeria Syndrome
title_full_unstemmed Single-Dose CRISPR/Cas9 Therapy Extends Lifespan of Mice with Hutchinson-Gilford Progeria Syndrome
title_short Single-Dose CRISPR/Cas9 Therapy Extends Lifespan of Mice with Hutchinson-Gilford Progeria Syndrome
title_sort single-dose crispr/cas9 therapy extends lifespan of mice with hutchinson-gilford progeria syndrome
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6541418/
https://www.ncbi.nlm.nih.gov/pubmed/30778240
http://dx.doi.org/10.1038/s41591-019-0343-4
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