Therapeutic Gene Editing in Inherited Retinal Disorders.
Cold Spring Harb Perspect Med
; 13(4)2023 04 03.
Article
em En
| MEDLINE
| ID: mdl-36096547
ABSTRACT
Since the development of CRISPR/Cas9 gene editing in 2012, therapeutic editing research has produced several phase 1-2a trials. Here we provide an overview of the mechanisms and applications of various gene-editing technologies including adeno-associated virus vectors, lentiviruses, CRISPR/Cas9 systems, base and prime editing, antisense oligonucleotides, short-hairpin RNAs, Cas13, and adenosine deaminase acting on RNA for the treatment of various inherited retinal diseases (IRDs). We outline the various stages of clinical trials using these technologies and the impacts they have made in advancing the practice of medicine.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Doenças Retinianas
/
Edição de Genes
Limite:
Humans
Idioma:
En
Ano de publicação:
2023
Tipo de documento:
Article