Generation of an Avian Myeloblastosis Virus (AMV) Reverse Transcriptase Prime Editor.
Adv Exp Med Biol
; 1415: 109-114, 2023.
Article
em En
| MEDLINE
| ID: mdl-37440022
Prime editing (PE) is a novel, double-strand break (DSB)-independent gene editing technology that represents an exciting avenue for the treatment of inherited retinal diseases (IRDs). Given the extensive and heterogenous nature of the 280 genes associated with IRDs, genome editing has presented countless complications. However, recent advances in genome editing technologies have identified PE to have tremendous potential, with the capability to ameliorate small deletions and insertions in addition to all twelve possible transition and transversion mutations. The current PE system is based on the fusion of the Streptococcus pyogenes Cas9 (SpCas9) nickase H840A mutant and an optimized Moloney murine leukemia virus (MMLV) reverse-transcriptase (RT) in conjunction with a PE guide RNA (pegRNA). In this study, we developed a prime editor based on the avian myeloblastosis virus (AMV)-RT and showed its applicability for the installation of the PRPH2 c.828+1G>A mutation in HEK293 cells.
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Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
DNA Polimerase Dirigida por RNA
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Vírus da Mieloblastose Aviária
Limite:
Animals
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Humans
Idioma:
En
Revista:
Adv Exp Med Biol
Ano de publicação:
2023
Tipo de documento:
Article