What a Clinician Needs to Know About Genome Editing: Status and Opportunities for Inborn Errors of Immunity.
J Allergy Clin Immunol Pract
; 12(5): 1139-1149, 2024 May.
Article
em En
| MEDLINE
| ID: mdl-38246560
ABSTRACT
During the past 20 years, gene editing has emerged as a novel form of gene therapy. Since the publication of the first potentially therapeutic gene editing platform for genetic disorders, increasingly sophisticated editing technologies have been developed. As with viral vector-mediated gene addition, inborn errors of immunity are excellent candidate diseases for a corrective autologous hematopoietic stem cell gene editing strategy. Research on gene editing for inborn errors of immunity is still entirely preclinical, with no trials yet underway. However, with editing techniques maturing, scientists are investigating this novel form of gene therapy in context of an increasing number of inborn errors of immunity. Here, we present an overview of these studies and the recent progress moving these technologies closer to clinical benefit.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Temas:
Geral
Base de dados:
MEDLINE
Assunto principal:
Terapia Genética
/
Edição de Genes
Limite:
Animals
/
Humans
Idioma:
En
Revista:
J Allergy Clin Immunol Pract
Ano de publicação:
2024
Tipo de documento:
Article
País de afiliação:
Reino Unido