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

被引：8

作者：

Trujillo, Alexandria J. ^{[1
,2
]}

Myers, Jason M. ^{[1
]}

Fayazi, Zahra S. ^{[1
]}

Butler, Mark C. ^{[1
]}

Sullivan, Jack M. ^{[3
,4
,5
,6
,7
,8
]}

机构：

[1] SUNY Buffalo, Jacobs Sch Med & Biomed Sci, VA Western NY Healthcare Syst, Res Serv,Ross Eye Inst,Dept Ophthalmol, Buffalo, NY USA

[2] SUNY Buffalo, Jacobs Sch Med & Biomed Sci, VA Western NY Healthcare Syst, Res Serv,Ross Eye Inst,Dept Pharmacol Toxicol, Buffalo, NY USA

[3] VA Western NY Healthcare Syst, Ross Eye Inst, Program Neurosci, Res Serv,Dept Ophthalmol, Buffalo, NY 14215 USA

[4] VA Western NY Healthcare Syst, Ross Eye Inst, Program Neurosci, Res Serv,Dept Pharmacol Toxicol, Buffalo, NY 14215 USA

[5] VA Western NY Healthcare Syst, Ross Eye Inst, Program Neurosci, Res Serv,Dept Physiol Biophys, Buffalo, NY 14215 USA

[6] SUNY Buffalo, Jacobs Sch Med & Biomed Sci, Buffalo, NY 14260 USA

[7] SUNY Syracuse, Eye Inst, Syracuse, NY 13202 USA

[8] SUNY Albany, RNA Inst, Albany, NY 12222 USA

来源：

RETINAL DEGENERATIVE DISEASES: MECHANISMS AND EXPERIMENTAL THERAPY | 2019年 / 1185卷

关键词：

Gene therapy; Inherited retinal degenerations; Retinitis pigmentosa; Hammerhead ribozyme; RNA biologic therapies; Noncoding RNA; SELF-CLEAVAGE; RNA; MODEL;

D O I：

10.1007/978-3-030-27378-1_20

中图分类号：

R-3 [医学研究方法]; R3 [基础医学];

学科分类号：

1001 ;

摘要：

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.

引用

页码：119 / 124

页数：6

共 32 条

[1] ENZYMATIC CLEAVAGE OF RNA BY RNA, (REPRINTED) [J].