Hematopoietic stem cell gene therapy: The optimal use of lentivirus and gene editing approaches.
Blood Rev
; 40: 100641, 2020 03.
Article
em En
| MEDLINE
| ID: mdl-31761379
ABSTRACT
Due to pioneering in vitro investigations on gene modification, gene engineering platforms have incredibly improved to a safer and more powerful tool for the treatment of multiple blood and immune disorders. Likewise, several clinical trials have been initiated combining autologous hematopoietic stem cell transplantation (auto-HSCT) with gene therapy (GT) tools. As several GT modalities such as lentivirus and gene editing tools have a long developmental path ahead to diminish its negative side effects, it is hard to decide which modality is optimal for treating a specific disease. Gene transfer by lentiviruses is the platform of choice for loss-of-mutation diseases, whereas gene correction/addition or gene disruption by gene editing tools, mainly CRISPR/Cas9, is likely to be more efficient in diseases where tight regulation is needed. Therefore, in this review, we compiled pertinent information about lentiviral gene transfer and CRISPR/Cas9 gene editing, their evolution to a safer platform for HSCT, and their applications on other types of gene disorders based on the etiology of the disease and cell fitness.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Células-Tronco Hematopoéticas
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Terapia Genética
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Lentivirus
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Transplante de Células-Tronco Hematopoéticas
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Edição de Genes
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Doenças Hematológicas
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Doenças do Sistema Imunitário
Limite:
Humans
Idioma:
En
Revista:
Blood Rev
Assunto da revista:
HEMATOLOGIA
Ano de publicação:
2020
Tipo de documento:
Article
País de afiliação:
Alemanha