Efficient in vivo neuronal genome editing in the mouse brain using nanocapsules containing CRISPR-Cas9 ribonucleoproteins

Author:

Metzger Jeanette M.,Wang Yuyuan,Neuman Samuel S.,Snow Kathy J.,Murray Stephen A.,Lutz Cathleen M.,Bondarenko Viktoriya,Felton Jesi,Gimse KirstanORCID,Xie RuosenORCID,Li Dongdong,Zhao Yi,Flowers Matthew T.,Simmons Heather A.ORCID,Roy Subhojit,Saha Krishanu,Levine Jon E.,Emborg Marina E.,Gong ShaoqinORCID

Funder

National Institutes of Health

NIH Office of the Director

Publisher

Elsevier BV

Subject

Mechanics of Materials,Biomaterials,Biophysics,Ceramics and Composites,Bioengineering

Reference56 articles.

1. CRISPR-Cas9: a preclinical and clinical perspective for the treatment of human diseases;Sharma;Mol. Ther.,2021

2. The NIH somatic cell genome editing program;Saha;Nature,2021

3. CRISPR-engineered T cells in patients with refractory cancer;Stadtmauer;Science,2020

4. CRISPR-Cas9 in vivo gene editing for transthyretin amyloidosis;Gillmore;N. Engl. J. Med.,2021

5. Efficacy and safety of an investigational single-course CRISPR base editing therapy targeting PCSK9 in non-human primate and mouse models;Lee;Circulation,2022

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