Gene Therapy Prolongs Survival and Restores Function in Murine and Canine Models of Myotubular Myopathy-Reference-Cited by-同舟云学术

Gene Therapy Prolongs Survival and Restores Function in Murine and Canine Models of Myotubular Myopathy

Published:2014-01-22 Issue:220 Volume:6 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Childers Martin K.¹²,Joubert Romain³,Poulard Karine³,Moal Christelle³,Grange Robert W.⁴,Doering Jonathan A.⁴,Lawlor Michael W.⁵⁶,Rider Branden E.⁵,Jamet Thibaud³,Danièle Nathalie³,Martin Samia³,Rivière Christel³,Soker Thomas⁶,Hammer Caroline³,Van Wittenberghe Laetitia³,Lockard Mandy⁷,Guan Xuan⁷,Goddard Melissa⁷,Mitchell Erin⁷,Barber Jane⁷,Williams J. Koudy⁷,Mack David L.¹,Furth Mark E.⁸,Vignaud Alban³,Masurier Carole³,Mavilio Fulvio³,Moullier Philippe³⁹¹⁰,Beggs Alan H.⁵,Buj-Bello Anna³

Affiliation:

1. Department of Rehabilitation Medicine, School of Medicine, University of Washington, Campus Box 358056, Seattle, WA 98109, USA.

2. Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA 98109, USA.

3. Généthon, 1 bis rue de l’Internationale, 91002 Evry, France.

4. Department of Human Nutrition, Foods and Exercise, Virginia Polytechnic Institute and State University, Blacksburg, VA 24061, USA.

5. Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children’s Hospital, Harvard Medical School, 300 Longwood Avenue, Boston, MA 02115, USA.

6. Department of Pathology and Laboratory Medicine, Children’s Hospital and Medical College of Wisconsin, Milwaukee, WI 53226, USA.

7. Wake Forest Institute for Regenerative Medicine, 391 Technology Way, Winston-Salem, NC 27101, USA.

8. Comprehensive Cancer Center, Wake Forest University Health Sciences, School of Medicine, Medical Center Boulevard, Winston-Salem, NC 27157, USA.

9. INSERM U649, Atlantic Gene Therapies, CHU Hôtel Dieu, 44300 Nantes, France.

10. Molecular Genetics and Microbiology Department, University of Florida, Gainesville, FL 32611, USA.

Abstract

Intravenous injection of an adeno-associated viral vector expressing the myotubularin ( MTM1 ) gene improves survival and rescues skeletal muscle function in mice and dogs affected by myotubular myopathy.

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference44 articles.

1. Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

2. Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis

3. AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness

4. Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

5. Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D

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