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Development of a CRISPR/Cas9-based therapy for Hutchinson–Gilford progeria syndrome
- Source :
- Nature Medicine, Nature medicine, RUO. Repositorio Institucional de la Universidad de Oviedo, instname
- Publication Year :
- 2019
-
Abstract
- The Instituto Universitario de Oncología del Principado de Asturias is supported by Fundación Bancaria Caja de Ahorros de Asturias. J.M.P.F. is supported by Ministerio de Economía y Competitividad (MINECO/FEDER: No. SAF2015-64157-R) and Gobierno del Principado de Asturias. C.L.-O. is supported by grants from the European Research Council (ERC-2016-ADG, DeAge), Ministerio de Economía y Competitividad (MINECO/FEDER: Nos. SAF2014-52413-R and SAF2017-87655-R), Instituto de Salud Carlos III (RTICC) and Progeria Research Foundation (No. PRF2016-66). O.S.-F. is recipient of an FPU fellowship. A.R.F. is recipient of a Ramón y Cajal fellowship. The generation of progerin antibody was funded by the Wellcome Trust (No. 098291/Z/12/Z to S.N.).
- Subjects :
- 0301 basic medicine
congenital, hereditary, and neonatal diseases and abnormalities
Genetic enhancement
Biology
General Biochemistry, Genetics and Molecular Biology
Article
Frameshift mutation
LMNA
03 medical and health sciences
Mice
0302 clinical medicine
Progeria
medicine
CRISPR
Animals
Humans
Point Mutation
Gene
Genetics
integumentary system
Cas9
Point mutation
nutritional and metabolic diseases
General Medicine
Genetic Therapy
medicine.disease
Lamin Type A
3. Good health
030104 developmental biology
HEK293 Cells
030220 oncology & carcinogenesis
embryonic structures
CRISPR-Cas Systems
Subjects
Details
- ISSN :
- 10788956
- Database :
- OpenAIRE
- Journal :
- Nature Medicine
- Accession number :
- edsair.doi.dedup.....29797ad020a2f7150853854ecca92a0f
- Full Text :
- https://doi.org/10.1038/s41591-018-0338-6