Preclinical Assessment of a Gene-Editing Approach in a Mouse Model of Mitochondrial Neurogastrointestinal Encephalomyopathy-Reference-Cited by-同舟云学术

Preclinical Assessment of a Gene-Editing Approach in a Mouse Model of Mitochondrial Neurogastrointestinal Encephalomyopathy

Published:2021-10-01 Issue:19-20 Volume:32 Page:1210-1223
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Parés Marta¹,Fornaguera Cristina²,Vila-Julià Ferran³,Oh Sejin²,Fan Steven H.Y.⁴,Tam Ying K.⁴,Comes Natalia⁵,Vidal Francisco⁵⁶,Martí Ramon³,Borrós Salvador²,Barquinero Jordi¹^ORCID

Affiliation:

1. Gene and Cell Therapy, Institut de Recerca Hospital Universitari Vall d'Hebron (VHIR), Universitat Autònoma de Barcelona (UAB), Barcelona, Spain.

2. Grup d'Enginyeria de Materials (GEMAT), Institut Químic de Sarrià (IQS), Ramon Llull University (URL), Barcelona, Spain.

3. Research Group on Neuromuscular and Mitochondrial Diseases, Institut de Recerca Hospital Universitari Vall d'Hebron (VHIR), Universitat Autònoma de Barcelona (UAB), and Biomedical Network Research Centre on Rare Diseases (CIBERER), Instituto de Salud Carlos III, Barcelona, Spain.

4. Acuitas Therapeutics, Vancouver, British Columbia, Canada

5. Blood and Tissue Bank, Barcelona, Spain.

6. Centro de Investigación Biomédica en Red de Enfermedades Cardiovasculares (CIBERCV), Madrid, Spain.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2021.152

Reference75 articles.

1. Thymidine Phosphorylase Gene Mutations in MNGIE, a Human Mitochondrial Disorder

2. Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE)