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The decision‐making process and criteria in selecting candidate drugs for progeria clinical trials

Hutchinson–Gilford progeria syndrome (progeria) is an extremely rare premature aging disease with a population prevalence of 1 in 20 million. Nevertheless, propelled by the discovery of a causal mutation in the lamin A/C gene (LMNA) (De Sandre‐Giovannoli et al, 2003; Eriksson et al, 2003) and strong...

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Detalles Bibliográficos
Autores principales: Gordon, Leslie B, Kieran, Mark W, Kleinman, Monica E, Misteli, Tom
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2016
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4931284/
https://www.ncbi.nlm.nih.gov/pubmed/27234439
http://dx.doi.org/10.15252/emmm.201606280
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author Gordon, Leslie B
Kieran, Mark W
Kleinman, Monica E
Misteli, Tom
author_facet Gordon, Leslie B
Kieran, Mark W
Kleinman, Monica E
Misteli, Tom
author_sort Gordon, Leslie B
collection PubMed
description Hutchinson–Gilford progeria syndrome (progeria) is an extremely rare premature aging disease with a population prevalence of 1 in 20 million. Nevertheless, propelled by the discovery of a causal mutation in the lamin A/C gene (LMNA) (De Sandre‐Giovannoli et al, 2003; Eriksson et al, 2003) and strong patient advocacy (Gordon & Gordon, 2014), progeria has rapidly become a vibrant field of study, attracting a wide range of researchers from basic cell biologists to clinicians.
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spelling pubmed-49312842016-07-08 The decision‐making process and criteria in selecting candidate drugs for progeria clinical trials Gordon, Leslie B Kieran, Mark W Kleinman, Monica E Misteli, Tom EMBO Mol Med Commentary Hutchinson–Gilford progeria syndrome (progeria) is an extremely rare premature aging disease with a population prevalence of 1 in 20 million. Nevertheless, propelled by the discovery of a causal mutation in the lamin A/C gene (LMNA) (De Sandre‐Giovannoli et al, 2003; Eriksson et al, 2003) and strong patient advocacy (Gordon & Gordon, 2014), progeria has rapidly become a vibrant field of study, attracting a wide range of researchers from basic cell biologists to clinicians. John Wiley and Sons Inc. 2016-05-27 2016-07 /pmc/articles/PMC4931284/ /pubmed/27234439 http://dx.doi.org/10.15252/emmm.201606280 Text en © 2016 The Authors. Published under the terms of the CC BY 4.0 license This is an open access article under the terms of the Creative Commons Attribution 4.0 (http://creativecommons.org/licenses/by/4.0/) License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited.
spellingShingle Commentary
Gordon, Leslie B
Kieran, Mark W
Kleinman, Monica E
Misteli, Tom
The decision‐making process and criteria in selecting candidate drugs for progeria clinical trials
title The decision‐making process and criteria in selecting candidate drugs for progeria clinical trials
title_full The decision‐making process and criteria in selecting candidate drugs for progeria clinical trials
title_fullStr The decision‐making process and criteria in selecting candidate drugs for progeria clinical trials
title_full_unstemmed The decision‐making process and criteria in selecting candidate drugs for progeria clinical trials
title_short The decision‐making process and criteria in selecting candidate drugs for progeria clinical trials
title_sort decision‐making process and criteria in selecting candidate drugs for progeria clinical trials
topic Commentary
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4931284/
https://www.ncbi.nlm.nih.gov/pubmed/27234439
http://dx.doi.org/10.15252/emmm.201606280
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