Applying switchable Cas9 variants to in vivo gene editing for therapeutic applications.
Cell Biol Toxicol
; 36(1): 17-29, 2020 02.
Article
em En
| MEDLINE
| ID: mdl-31418127
ABSTRACT
Progress in targeted gene editing by programmable endonucleases has paved the way for their use in gene therapy. Particularly, Cas9 is an endonuclease with high activity and flexibility, rendering it an attractive option for therapeutic applications in clinical settings. Many disease-causing mutations could potentially be corrected by this versatile new technology. In addition, recently developed switchable Cas9 variants, whose activity can be controlled by an external stimulus, provide an extra level of spatiotemporal control on gene editing and are particularly desirable for certain applications. Here, we discuss the considerations and difficulties for implementing Cas9 to in vivo gene therapy. We put particular emphasis on how switchable Cas9 variants may resolve some of these barriers and advance gene therapy in the clinical setting.
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Texto completo:
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Base de dados:
MEDLINE
Assunto principal:
Terapia Genética
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Edição de Genes
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Proteína 9 Associada à CRISPR
Limite:
Humans
Idioma:
En
Ano de publicação:
2020
Tipo de documento:
Article