The gRNA Vector Level Determines the Outcome of Systemic AAV CRISPR Therapy for Duchenne Muscular Dystrophy-Reference-Cited by-同舟云学术

The gRNA Vector Level Determines the Outcome of Systemic AAV CRISPR Therapy for Duchenne Muscular Dystrophy

Published:2022-05-01 Issue:9-10 Volume:33 Page:518-528
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Wasala Nalinda B.¹,Million Emily D.¹^ORCID,Watkins Thais B.¹,Wasala Lakmini P.¹,Han Jin¹,Yue Yongping¹,Lu Baisong²,Chen Shi-jie³⁴⁵,Hakim Chady H.¹,Duan Dongsheng¹⁶⁷⁸^ORCID

Affiliation:

1. Department of Molecular Microbiology and Immunology, The University of Missouri, Columbia, Missouri, USA

2. Wake Forest Institute for Regenerative Medicine, Wake Forest University Health Sciences, Winston-Salem, North Carolina, USA

3. Department of Physics,

4. Department of Biochemistry, and

5. Institute for Data Science and Informatics, The University of Missouri, Columbia, Missouri, USA

6. Departments of Neurology, School of Medicine, The University of Missouri, Columbia, Missouri, USA

7. Department of Biomedical, Biological and Chemical Engineering, College of Engineering, The University of Missouri, Columbia, Missouri, USA

8. Department of Biomedical Sciences, College of Veterinary Medicine, The University of Missouri, Columbia, Missouri, USA.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2021.130

Reference30 articles.

1. Duchenne muscular dystrophy

2. Genome editing with CRISPR–Cas nucleases, base editors, transposases and prime editors

3. CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors

4. The promise and challenge of therapeutic genome editing

5. Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy

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3. CRISPR-Cas9 homology-independent targeted integration of exons 1–19 restores full-length dystrophin in mice;Molecular Therapy - Methods & Clinical Development;2023-09

4. CRISPR-Editing Therapy for Duchenne Muscular Dystrophy;Human Gene Therapy;2023-05-01

5. A Brief Review of Duchenne Muscular Dystrophy Treatment Options, with an Emphasis on Two Novel Strategies;Biomedicines;2023-03-09