A Discovery with Potential to Revitalize Hammerhead Ribozyme Therapeutics for Treatment of Inherited Retinal Degenerations
| Autor: | Jason Myers, Mark C. Butler, Zahra Fayazi, Jack M. Sullivan, Alexandria J Trujillo |
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| Rok vydání: | 2019 |
| Předmět: |
Hammerhead ribozyme
biology Genetic enhancement Mutant Retinal Degeneration Ribozyme RNA Computational biology Non-coding RNA biology.organism_classification medicine.disease Article 03 medical and health sciences 0302 clinical medicine Retinitis pigmentosa biology.protein medicine Humans RNA Catalytic 030212 general & internal medicine RNA Messenger Gene |
| Zdroj: | Retinal Degenerative Diseases ISBN: 9783030273774 |
| ISSN: | 0065-2598 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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