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Article

Design and validation of therapeutic hammerhead ribozymes for autosomal dominant diseases.

Methods in molecular biology (Clifton, N.J.) - 1 Jan 2004

Fritz Jason J, Gorbatyuk Marina, Lewin Alfred S, Hauswirth William W

Abstract excerpt

Hammerhead ribozymes are small, catalytic RNAs that can be designed to effectively inhibit gene expression in an allele-specific manner. It is the high level of sequence discrimination, coupled with the minimal cleavage-site requirements of hammerhead ribozymes, that makes these catalytic RNAs so amenable for use as therapeutic agents for autosomal dominant diseases. Here, we present a detailed set of protocols...

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PMID: 15017052
DOI: 10.1385/1-59259-746-7:221

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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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