دورية أكاديمية
CRISPR-Cas9 DNA Base-Editing and Prime-Editing
| العنوان: | CRISPR-Cas9 DNA Base-Editing and Prime-Editing |
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| المؤلفون: | Ariel Kantor, Michelle E. McClements, Robert E. MacLaren |
| المصدر: | International Journal of Molecular Sciences, Vol 21, Iss 17, p 6240 (2020) |
| بيانات النشر: | MDPI AG, 2020. |
| سنة النشر: | 2020 |
| المجموعة: | LCC:Biology (General) LCC:Chemistry |
| مصطلحات موضوعية: | CRISPR/Cas9, base-editing, prime-editing, adeno-associated vector, genome engineering, gene therapy, Biology (General), QH301-705.5, Chemistry, QD1-999 |
| الوصف: | Many genetic diseases and undesirable traits are due to base-pair alterations in genomic DNA. Base-editing, the newest evolution of clustered regularly interspaced short palindromic repeats (CRISPR)-Cas-based technologies, can directly install point-mutations in cellular DNA without inducing a double-strand DNA break (DSB). Two classes of DNA base-editors have been described thus far, cytosine base-editors (CBEs) and adenine base-editors (ABEs). Recently, prime-editing (PE) has further expanded the CRISPR-base-edit toolkit to all twelve possible transition and transversion mutations, as well as small insertion or deletion mutations. Safe and efficient delivery of editing systems to target cells is one of the most paramount and challenging components for the therapeutic success of BEs. Due to its broad tropism, well-studied serotypes, and reduced immunogenicity, adeno-associated vector (AAV) has emerged as the leading platform for viral delivery of genome editing agents, including DNA-base-editors. In this review, we describe the development of various base-editors, assess their technical advantages and limitations, and discuss their therapeutic potential to treat debilitating human diseases. |
| نوع الوثيقة: | article |
| وصف الملف: | electronic resource |
| اللغة: | English |
| تدمد: | 1422-0067 1661-6596 |
| Relation: | https://www.mdpi.com/1422-0067/21/17/6240; https://doaj.org/toc/1661-6596; https://doaj.org/toc/1422-0067 |
| DOI: | 10.3390/ijms21176240 |
| URL الوصول: | https://doaj.org/article/0c9c3a3bc3164205ab10ef85a66b19aa |
| رقم الأكسشن: | edsdoj.0c9c3a3bc3164205ab10ef85a66b19aa |
| قاعدة البيانات: | Directory of Open Access Journals |
Full Text Finder | تدمد: | 14220067 16616596 |
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| DOI: | 10.3390/ijms21176240 |