Gene Editing Corrects <i>In Vitro</i> a G > A <i>GLB1</i> Transition from a GM1 Gangliosidosis Patient-Reference-Cited by-同舟云学术

Gene Editing Corrects In Vitro a G > A GLB1 Transition from a GM1 Gangliosidosis Patient

Published:2023-02-01 Issue:1 Volume:6 Page:17-31
ISSN:2573-1599
Container-title:The CRISPR Journal
language:en
Short-container-title:The CRISPR Journal

Author:

Leclerc Delphine¹,Goujon Louise²,Jaillard Sylvie³⁴,Nouyou Bénédicte⁴,Cluzeau Laurence⁴,Damaj Léna⁵,Dubourg Christèle⁶⁷,Etcheverry Amandine⁶,Levade Thierry⁸,Froissart Roseline⁹,Dréano Stéphane⁷,Guillory Xavier¹¹⁰,Eriksson Leif A¹¹^ORCID,Launay Erika⁴,Mouriaux Frédéric¹¹²,Belaud-Rotureau Marc-Antoine³⁴,Odent Sylvie²⁷,Gilot David¹⁴^ORCID

Affiliation:

1. INSERM U1242, OSS, Univ Rennes, Rennes, France.

2. CHU Rennes, Service de Génétique Clinique, Centre de Référence Maladies Rares CLAD-Ouest, FHU GenOMEDS, ERN ITHACA, Hôpital Sud, Rennes, France.

3. INSERM, EHESP, IRSET–UMR_S, 1085, Université Rennes 1, Rennes, France.

4. Service de Cytogénétique et Biologie Cellulaire, CHU Rennes, Rennes, France.

5. Department of Pediatrics, Competence Center of Inherited Metabolic Disorders, Rennes Hospital, Rennes, France.

6. Laboratoire de Génétique Moléculaire et Génomique, Centre Hospitalier Universitaire de Rennes, Rennes, France.

7. Univ Rennes, CNRS, IGDR (Institut de Génétique et Développement de Rennes), UMR 6290, ERL U1305, Rennes, France.

8. Laboratoire de Biochimie, CHU de Toulouse, Pôle biologie, Institut Fédératif de Biologie, Toulouse, France.

9. CHU Lyon HCL, LBMMS–Service Biochimie et Biologie Moléculaire, UM Pathologies Héréditaires du Métabolisme et du Globule Rouge, Bron, France.

10. Univ Rennes, CNRS, ISCR (Institut des Sciences Chimiques de Rennes)–UMR 6226, Rennes, France.

11. Department of Chemistry and Molecular Biology, University of Gothenburg, Göteborg, Sweden.

12. Department of Ophthalmology, CHU Rennes, Univ Rennes, Rennes, France.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Biotechnology

Link

https://www.liebertpub.com/doi/pdf/10.1089/crispr.2022.0045

Reference51 articles.

1. The NIH Somatic Cell Genome Editing program

2. The promise and challenge of therapeutic genome editing

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

4. In vivo somatic cell base editing and prime editing

5. Base editing of haematopoietic stem cells rescues sickle cell disease in mice

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. Gene therapy approaches for GM1 gangliosidosis: Focus on animal and cellular studies;Cell Biochemistry and Function;2023-11-29