Therapeutic gene editing in haematological disorders with CRISPR/Cas9.
Br J Haematol
; 185(5): 821-835, 2019 06.
Article
em En
| MEDLINE
| ID: mdl-30864164
ABSTRACT
The Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated (Cas)9 platform offers an efficient way of making precise genetic changes to the human genome. This can be employed for disruption, addition and correction of genes, thereby enabling a new class of genetic therapies that can be applied to haematological disorders. Here we review recent technological advances in the CRISPR/Cas9 methodology and applications in haematology for curing monogenic genetic disorders and for engineering novel chimeric antigen receptor (CAR) T cells to treat haematological malignancies. Furthermore, we discuss current challenges for full clinical implementation of CRISPR/Cas9, and reflect on future trajectories of the technology.
Palavras-chave
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Sistemas CRISPR-Cas
/
Edição de Genes
/
Doenças Hematológicas
Limite:
Humans
Idioma:
En
Ano de publicação:
2019
Tipo de documento:
Article