AAV-based CRISPR-Cas9 genome editing: Challenges and engineering opportunities

Author:

Kabadi Ami M.,Mejia-Guerra Maria Katherine,Graef John D.,Khan Sohrab Z.,Walton Eric M.,Wang Xinzhu,Gersbach Charles A.,Potter Rachael

Funder

Sarepta Therapeutics Inc

Publisher

Elsevier BV

Subject

Biomedical Engineering,Biomaterials,Medicine (miscellaneous),Bioengineering

Reference71 articles.

1. Gene editing and CRISPR in the clinic: current and future perspectives;Hirakawa;Biosci Rep,2020

2. Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates;Burdo;Gene Ther,2023

3. Vertex Press Release. Vertex and CRISPR Therapeutics Announce Authorization of the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), by the United Kingdom MHRA for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia. https://investors.vrtx.com/news-releases/news-release-details/vertex-and-crispr-therapeutics-announce-authorization-first (Accessed 4 January 2024).

4. CRISPR technologies for precise epigenome editing;Nakamura;Nat Cell Biol,2021

5. To cut or not to cut: next-generation genome editors for precision genome engineering;Zhang;Curr Opin Biomed Eng,2023

Cited by 1 articles. 订阅此论文施引文献 订阅此论文施引文献,注册后可以免费订阅5篇论文的施引文献,订阅后可以查看论文全部施引文献

1. The realization of CRISPR gene therapy;Nature Chemical Biology;2024-06-13

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