A Curative DNA Code for Hematopoietic Defects: Novel Cell Therapies for Monogenic Diseases of the Blood and Immune System.
Hematol Oncol Clin North Am
; 36(4): 647-665, 2022 08.
Article
en En
| MEDLINE
| ID: mdl-35773054
ABSTRACT
Innovations in programmable nucleases have expanded genetic engineering capabilities, raising the possibility of a new approach to curing monogenic hematological diseases. Feasibility studies using ex vivo targeted genome-editing, and nonintegrating viral vectors show outstanding potential for correcting genetic conditions at their root cause. This article reviews the latest technological advances in the CRISPR/Cas9 system alone and combined with engineered viruses as editing tools for human hematopoietic stem and progenitor cells (HSPCs). We discuss the early phase in human trials of genome editing-based therapies for hemoglobinopathies.
Palabras clave
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Edición Génica
/
Vectores Genéticos
Límite:
Humans
Idioma:
En
Revista:
Hematol Oncol Clin North Am
Asunto de la revista:
HEMATOLOGIA
/
NEOPLASIAS
Año:
2022
Tipo del documento:
Article