@inbook{067cabe569234ab690fa9329aa8beb78,
title = "A Discovery with Potential to Revitalize Hammerhead Ribozyme Therapeutics for Treatment of Inherited Retinal Degenerations",
abstract = "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.",
keywords = "Gene therapy, Hammerhead ribozyme, Inherited retinal degenerations, Noncoding RNA, RNA biologic therapies, Retinitis pigmentosa",
author = "Trujillo, \{Alexandria J.\} and Myers, \{Jason M.\} and Fayazi, \{Zahra S.\} and Butler, \{Mark C.\} and Sullivan, \{Jack M.\}",
note = "Publisher Copyright: {\textcopyright} 2019, Springer Nature Switzerland AG.",
year = "2019",
doi = "10.1007/978-3-030-27378-1\_20",
language = "English",
series = "Advances in Experimental Medicine and Biology",
publisher = "Springer",
pages = "119--124",
booktitle = "Advances in Experimental Medicine and Biology",
address = "Germany",
}