| Literature DB >> 31884599 |
Alexandria J Trujillo1, Jason M Myers2, Zahra S Fayazi2, Mark C Butler2, Jack M Sullivan3,4,5,6.
Abstract
Hammerhead ribozymes (hhRzs), RNA enzymes capable of site-specific cleavage of arbitrary target mRNAs, have faced significant hurdles in development and optimization as gene therapeutics for clinical translation. Chemical and biological barriers must be overcome to realize an effective therapeutic. A new Facilitated ribozyme has been identified with greatly enhanced kinetic properties that lead new insight on the capacity of ribozymes to target mutant genes to treat inherited retinal degenerations.Entities:
Keywords: Gene therapy; Hammerhead ribozyme; Inherited retinal degenerations; Noncoding RNA; RNA biologic therapies; Retinitis pigmentosa
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Year: 2019 PMID： 31884599 PMCID： PMC7029959 DOI： 10.1007/978-3-030-27378-1_20
Source DB: PubMed Journal: Adv Exp Med Biol ISSN： 0065-2598 Impact factor: 2.622