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Article

In vivo application of an RNAi strategy for the selective suppression of a mutant allele.

Human gene therapy - 1 Jan 2011

Kubodera Takayuki, Yamada Hiromi, Anzai Masayuki, Ohira Shinga, Yokota Shigefumi, Hirai Yukihiko, Mochizuki Hideki, Shimada Takashi, Mitani Tasuku, Mizusawa Hidehiro, Yokota Takanori

Abstract excerpt

Gene therapy for dominantly inherited diseases with small interfering RNA (siRNA) requires mutant allele-specific suppression when genes in which mutation causes disease normally have an important role. We previously proposed a strategy for selective suppression of mutant alleles; both mutant and...

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Source

Identifiers

PMID: 20649474
DOI: 10.1089/hum.2010.054

Genes

SOD1

Variants

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 2 Sept 2026. Prepared record publication-20649474-2026-09-02.2. Manifest publication-graph-cohort-2026-09-02.2.

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

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