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

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....

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Bibliografiske detaljer
Udgivet i:Adv Exp Med Biol
Main Authors: Trujillo, Alexandria J., Myers, Jason M., Fayazi, Zahra S., Butler, Mark C., Sullivan, Jack M.
Format: Artigo
Sprog:Inglês
Udgivet: 2019
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7029959/
https://ncbi.nlm.nih.gov/pubmed/31884599
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/978-3-030-27378-1_20
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