| Autor: |
Angela, Lek, Kaiyue, Ma, Keryn G, Woodman, Monkol, Lek |
| Rok vydání: |
2021 |
| Předmět: |
|
| Zdroj: |
Human gene therapy. 32(5-6) |
| ISSN: |
1557-7422 |
| Popis: |
Clustered regularly interspaced short palindromic repeat (CRISPR)-based technology has been adapted to achieve a wide range of genome modifications, including transcription regulation. The focus of this review is on the application of CRISPR-based platforms such as nuclease-deficient Cas9 and Cas12a, to achieve targeted gene activation. We review studies to date that have used CRISPR-based activation technology for the elucidation of biological mechanism and disease correction, as well as its application in genetic screens as a powerful tool for high-throughput genotype-phenotype mapping. In addition to our synthesis and critical analysis of published studies, we explore key considerations for the potential clinical translation of CRISPR-based activation technology. |
| Databáze: |
OpenAIRE |
| Externí odkaz: |
|
