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Article

First-in-human mutation-targeted siRNA phase Ib trial of an inherited skin disorder.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Feb 2010

Leachman Sancy A, Hickerson Robyn P, Schwartz Mary E, Bullough Emily E, Hutcherson Stephen L, Boucher Kenneth M, Hansen C David, Eliason Mark J, Srivatsa G Susan, Kornbrust Douglas J, Smith Frances Jd, McLean Wh Irwin, Milstone Leonard M, Kaspar Roger L

Abstract excerpt

The rare skin disorder pachyonychia congenita (PC) is an autosomal dominant syndrome that includes a disabling plantar keratoderma for which no satisfactory treatment is currently available. We have completed a phase Ib clinical trial for treatment of PC utilizing the first short-interfering RNA...

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Identifiers

PMID: 19935778
DOI: 10.1038/mt.2009.273
PMCID: PMC2839285

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 2 Sept 2026. Prepared record publication-19935778-2026-09-02.2. Manifest publication-graph-cohort-2026-09-02.2.

Source receipt: PubMed via Helena Literature Corpus, retrieved 2 Sept 2026.

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