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In Genetic Disorders, One Size Does Not Fit All.

Clinical pharmacology and therapeutics - 1 Aug 2020

Wohlberg Christopher J

Abstract excerpt

In drug development, preclinical studies often do not predict human benefit. Why not, then, go right to the source-patients with rare diseases and, more importantly, with specific various genetic mutations that, in aggregate, define the phenotypic clinical disorder? In this issue, Genova et al. describe how induced pluripotent stem cells from patients with two genetic disorders (ataxia-telangiectasia and...

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PMID: 32369608
DOI: 10.1002/cpt.1851

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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