Gene Therapy for Rare Diseases: Summary of a National Institutes of Health Workshop, September 13, 2012

Author:

O'Reilly Marina1,Kohn Donald B.2,Bartlett Jeffrey3,Benson Janet4,Brooks Philip J.5,Byrne Barry J.6,Camozzi Carlos7,Cornetta Kenneth8,Crystal Ronald G.9,Fong Yuman10,Gargiulo Linda1,Gopal-Srivastava Rashmi5,High Katherine A.11,Jacobson Samuel G.12,Jambou Robert C.1,Montgomery Maureen1,Rosenthal Eugene1,Samulski R. Jude13,Skarlatos Sonia I.14,Sorrentino Brian15,Wilson James M.16,Xie Yun1,Corrigan-Curay Jacqueline1

Affiliation:

1. Office of Biotechnology Activities, Office of Science Policy, Office of the Director, National Institutes of Health, Bethesda, MD 20892.

2. David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA 90095.

3. Calimmune, Inc., Los Angeles, CA 90024.

4. Applied Toxicology and Gene Therapy Pharmacology/Toxicology Program, Lovelace Respiratory Research Institute, Albuquerque, NM 87108.

5. Office of Rare Diseases Research, National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, MD 20892.

6. Powell Gene Therapy Center, Departments of Molecular Genetics and Microbiology and Pediatrics, College of Medicine, University of Florida, Gainesville, FL 32610.

7. Orphazyme, Copenhagen, Denmark.

8. Department of Medical and Molecular Genetics, Vector Production Facility, School of Medicine, Indiana University, Indianapolis, IN 46202.

9. Department of Genetic Medicine, Arthur and Rochelle Belfer Gene Therapy Core Facility, Weill Cornell Medical College, New York, NY 10021.

10. Memorial Sloan-Kettering Cancer Center, New York, NY 10065.

11. Perelman School of Medicine, University of Pennsylvania, Center for Cellular and Molecular Therapeutics and Howard Hughes Medical Institute, The Children's Hospital of Philadelphia, Philadelphia, PA 19104.

12. Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA 19104.

13. Gene Therapy Center, Department of Pharmacology, School of Medicine, The University of North Carolina at Chapel Hill, Chapel Hill, NC 27599.

14. Division of Cardiovascular Sciences, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD 20892.

15. Experimental Hematology Division, St. Jude Children's Research Hospital, Memphis, TN 38105.

16. Departments of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA 19104.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

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