CRISPR/Cas9 genome engineering in hematopoietic cells.
Drug Discov Today Technol
; 28: 33-39, 2018 Aug.
Article
em En
| MEDLINE
| ID: mdl-30205879
ABSTRACT
The development of genome editing tools capable of modifying specific genomic sequences with unprecedented accuracy has opened up a wide range of new possibilities in targeted gene manipulation. In particular, the CRISPR/Cas9 system, a repurposed prokaryotic adaptive immune system, has been widely adopted because of its unmatched simplicity and flexibility. In this review we discuss achievements and current limitations of CRISPR/Cas9 genome editing in hematopoietic cells with special emphasis on its potential use in ex vivo gene therapy of monogenic blood disorders, HIV and cancer.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Terapia Genética
/
Sistemas CRISPR-Cas
/
Edição de Genes
Limite:
Animals
/
Humans
Idioma:
En
Revista:
Drug Discov Today Technol
Ano de publicação:
2018
Tipo de documento:
Article