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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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Bibliografski detalji
Izdano u:Adv Exp Med Biol
Glavni autori: Trujillo, Alexandria J., Myers, Jason M., Fayazi, Zahra S., Butler, Mark C., Sullivan, Jack M.
Format: Artigo
Jezik:Inglês
Izdano: 2019
Teme:
Online pristup: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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