Efficient CRISPR/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells

Author:

Dastidar Sumitava1,Ardui Simon2,Singh Kshitiz1,Majumdar Debanjana1,Nair Nisha1,Fu Yanfang34,Reyon Deepak34,Samara Ermira1,Gerli Mattia F M5,Klein Arnaud F6,De Schrijver Wito1,Tipanee Jaitip1,Seneca Sara7,Tulalamba Warut1,Wang Hui1,Chai Yoke Chin1,In’t Veld Peter8,Furling Denis6,Tedesco Francesco Saverio5,Vermeesch Joris R2,Joung J Keith34,Chuah Marinee K19,VandenDriessche Thierry19

Affiliation:

1. Department of Gene Therapy & Regenerative Medicine, Vrije Universiteit Brussel, Brussels 1090, Belgium

2. Department of Human Genetics, University of Leuven, Leuven 3000, Belgium

3. Molecular Pathology Unit, Center for Cancer Research and Center for Computational and Integrative Biology, Massachusetts General Hospital, Charlestown, MA02129, USA

4. Department of Pathology, Harvard Medical School, Boston, MA 02115, USA

5. Department of Cell and Developmental Biology, University College London, London WC1E6DE, UK

6. Sorbonne Universités, INSERM, Association Institute de Myologie, Center de Recherche en Myologie, F-75013 , France

7. Research Group Reproduction and Genetics (REGE), Center for Medical Genetics, UZ Brussels, Vrije Universiteit Brussel, Brussels 1090, Belgium

8. Department of Pathology, Vrije Universiteit Brussel, Brussels 1090, Belgium

9. Center for Molecular & Vascular Biology, Department of Cardiovascular Sciences, University of Leuven, Leuven 3000, Belgium

Funder

National Institutes of Health

Publisher

Oxford University Press (OUP)

Subject

Genetics

Reference108 articles.

1. The myotonic dystrophies: molecular, clinical, and therapeutic challenges;Udd;Lancet Neurol.,2012

2. Prevalence of muscular dystrophies: a systematic literature review;Theadom;Neuroepidemiology,2014

3. An unstable triplet repeat in a gene related to myotonic muscular dystrophy;Fu;Science,1992

4. Myotonic dystrophy mutation: an unstable CTG repeat in the 3′ untranslated region of the gene;Mahadevan;Science,1992

5. Molecular genetics and genetic testing in myotonic dystrophy type 1;Savic Pavicevic;Biomed. Res. Int.,2013

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