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Article

Cleavage of collagen RNA transcripts by hammerhead ribozymes in vitro is mutation-specific and shows competitive binding effects.

Nucleic acids research - 1 Sept 1997

Grassi G, Forlino A, Marini J C

Abstract excerpt

We report here the in vitro use of hammerhead ribozymes as an approach to the gene therapy of osteogenesis imperfecta (OI). Our strategy for the treatment of this dominant genetic disorder is based on selective reduction of the level of the mRNA transcripts from the mutant allele. We studied the in vitro cleavage activity of five different hammerhead ribozymes targeted against synthetic transcripts of two...

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Identifiers

PMID: 9254703
DOI: 10.1093/nar/25.17.3451
PMCID: PMC146924

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