[HTML][HTML] Genetic repair. Bruce A. Sullenger, Series Editor Ribozyme-mediated revision of RNA and DNA
MB Long, JP Jones, BA Sullenger… - The Journal of clinical …, 2003 - Am Soc Clin Investig
MB Long, JP Jones, BA Sullenger, J Byun
The Journal of clinical investigation, 2003•Am Soc Clin InvestigGenetic repair| Bruce A. Sullenger, Series Editor sequences are present downstream of the
ribozyme, the ribozyme ligates this portion of the RNA transcript to the cleavage site of the
substrate RNA. These 3′ exon sequences may be changed to potentially any nucleotide
sequence. With the ability to “cut and paste” RNA sequences together, trans-splicing
ribozymes were quickly recognized to have therapeutic potential in the revision of mutant
RNAs.
ribozyme, the ribozyme ligates this portion of the RNA transcript to the cleavage site of the
substrate RNA. These 3′ exon sequences may be changed to potentially any nucleotide
sequence. With the ability to “cut and paste” RNA sequences together, trans-splicing
ribozymes were quickly recognized to have therapeutic potential in the revision of mutant
RNAs.
Genetic repair| Bruce A. Sullenger, Series Editor sequences are present downstream of the ribozyme, the ribozyme ligates this portion of the RNA transcript to the cleavage site of the substrate RNA. These 3′ exon sequences may be changed to potentially any nucleotide sequence. With the ability to “cut and paste” RNA sequences together, trans-splicing ribozymes were quickly recognized to have therapeutic potential in the revision of mutant RNAs.
The Journal of Clinical Investigation