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A Discovery with Potential to Revitalize Hammerhead Ribozyme Therapeutics for Treatment of Inherited Retinal Degenerations. | LitMetric
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A Discovery with Potential to Revitalize Hammerhead Ribozyme Therapeutics for Treatment of Inherited Retinal Degenerations.

Alexandria J Trujillo Jason M Myers Zahra S Fayazi Mark C Butler Jack M Sullivan

Adv Exp Med Biol

Research Service, VA Western NY Healthcare System, Departments of Ophthalmology (Ross Eye Institute), Pharmacology/Toxicology, Physiology/Biophysics, Program in Neuroscience, Buffalo, NY, USA.

Published: February 2020

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.

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http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7029959PMC
http://dx.doi.org/10.1007/978-3-030-27378-1_20DOI Listing

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