Patients & Families

Gene Therapy Experts


Alain Fischer, M.D. PhD.* 
International Member, Scientific Advisory Committee
Director of Pediatric Immunology and Hematology
Hospital Necker Enfants Malades
Local Coordinator 
Federation of Clinical Immunology Societies
Hospital Necker-Enfant Malades
169 rue de Sevres, 75015 Paris, France

Marina Cavazzana-Calvo PhD* 
Head of the Department of Biotherapy 
Immunology and Pediatric Haematology Unit, 
Hospital Necker, 
75743 Paris, Cedex 15, France.
E Mail:

Anne Galy, Pharm D, PhD.* 
Head of "Molecular Immunology and Innovative Biotherapies" Unit
1 bis rue de l'Internationale,
91002 Evry, France
Phone: +33 1 69 47 29 93
 +33 1 60 77 86 98
E Mail:
Web site:


Christoph Klein MD PhD*
Prof. Christoph Klein
Children's Hospital
Ludwig-Maximilians-University Munich
Lindwurmstr. 4
80337 Munich
phone +49 (0)89 5160-7700
fax +49 (0)89 5160-7702


Alessandro Aiuti M.D. PhD*
San Raffaele-Telethon Institute for Gene Therapy. 
Via Olgettina 58. 20132 Milan,
E Mail:

Maria Grazia Roncarolo M.D. PhD.*

Project Leader
Gene therapy for Wiskott-Aldrich Syndrome
Scientific Director of the San Raffaele Scientific Institute, Milan, Italy;
Professor in Pediatrics, School of Medicine and Surgery, “Vita-Salute San Raffaele” University, Milan, Italy; 
Chief of Clinic, Pediatric Immunology and Hematology and Clinical Research Unit (CRU-P) San Raffaele Institute and San Raffaele Scientific Institute, Milan, Italy; 
Director of San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET),  Milan, Italy;
E Mail:

United States

Luigi Notarangelo, M.D* 
Director, Research and Molecular Diagnosis Program on Primary Immunodeficiencies
Professor of Pediatrics and Pathology
Boston Children's Hospital, Harvard Medical School
1 Blackfan Circle
Boston, MA 02115
Tel:(617) 919-2276
Fax: (617)730-0709
E mail:

Sung-Yun Pai MD*
Assistant Professor of Pediatrics, Harvard Medical School
Attending Staff, Combined Department of Pediatric Hematology-Oncology
Children’s Hospital/Dana-Farber Cancer Institute
Karp Family Research Laboratory, Room 08214
300 Longwood Avenue
Boston, MA 02115
Phone: Office: 617-919-2508
Patient Referral Line: 617-632-3961
Fax: 617-730-0934
E Mail:

David Rawlings, M.D.
Children's Guild Association Endowed Chair in Pediatric Immunology
Director, Center for Immunity and Immunotherapies
Seattle Children’s Hospital Research Institute
Chief, Division of Immunology
Children's Hospital
Adjunct Professor, Immunology
University of Washington School of Medicine
1900 Ninth Avenue
UW Box 359300-C9S-7
Seattle, WA 98101 
Phone: 206-987-7319
Fax: 206-987-7310 

Scott Snapper, M.D., Ph.D.
Department of Medicine and Immunology
Massachusetts General Hospital
Jackson 825C
55 Fruit Street
Boston, MA 02114
Tel: (617) 724-8809
Fax: (617) 726-2373

David A. Williams, M.D.* 
Director of Translational Research for Children's Hospital Boston
Leland Fikes Professor of Pediatrics, Harvard Medical School
Children's Hospital Boston
300 Longwood Ave, Karp 08125.3
Boston, MA 02115
Phone : (617)-919-2697
Fax: (617) 730- 0868
E Mail:

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