Publication: The decision‐making process and criteria in selecting candidate drugs for progeria clinical trials
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Date
2016
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John Wiley and Sons Inc.
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Citation
Gordon, Leslie B, Mark W Kieran, Monica E Kleinman, and Tom Misteli. 2016. “The decision‐making process and criteria in selecting candidate drugs for progeria clinical trials.” EMBO Molecular Medicine 8 (7): 685-687. doi:10.15252/emmm.201606280. http://dx.doi.org/10.15252/emmm.201606280.
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Abstract
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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Keywords
Commentary, Genetics, Gene Therapy & Genetic Disease, Pharmacology & Drug Discovery
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