Gene therapy for rare diseases: Summary of a National Institutes of Health Workshop, September 13, 2012

Human Gene Therapy

Volumn 24, Issue 4, 2013, Pages 355-362

  O'Reilly, Marina ^a   Kohn, Donald B ^b   Bartlett, Jeffrey ^c   Benson, Janet ^d   Brooks, Philip J ^e   Byrne, Barry J ^f   Camozzi, Carlos ^g   Cornetta, Kenneth ^h   Crystal, Ronald G ⁱ   Fong, Yuman ^j   Gargiulo, Linda ^a   Gopal Srivastava, Rashmi ^e   High, Katherine A ^k   Jacobson, Samuel G ^l   Jambou, Robert C ^a   Montgomery, Maureen ^a   Rosenthal, Eugene ^a   Samulski, R Jude ^m   Skarlatos, Sonia I ⁿ   Sorrentino, Brian ^o   Wilson, James M ^p   Xie, Yun ^a   Corrigan Curay, Jacqueline ^a   more..

^a NATIONAL INSTITUTES OF HEALTH   (United States)

^b UNIVERSITY OF CALIFORNIA   (United States)

^c Calimmune Australia Inc   (United States)

^d LOVELACE RESPIRATORY RESEARCH INSTITUTE   (United States)

^e NATIONAL INSTITUTES OF HEALTH   (United States)

^f UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

^g ORPHAZYME A S   (Denmark)

^h INDIANA UNIVERSITY   (United States)

ⁱ WEILL CORNELL MEDICAL COLLEGE   (United States)

^j MEMORIAL SLOAN KETTERING CANCER CENTER   (United States)

^k UNIVERSITY OF PENNSYLVANIA   (United States)

^l UNIVERSITY OF PENNSYLVANIA   (United States)

^m UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

ⁿ NATIONAL HEART LUNG AND BLOOD INSTITUTE   (United States)

^o ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

^p UNIVERSITY OF PENNSYLVANIA   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

CLINICAL EFFECTIVENESS; CLINICAL TRIAL (TOPIC); FUNDING; GENE THERAPY; HUMAN; RARE DISEASE; REVIEW; WORKSHOP;

CLINICAL TRIALS AS TOPIC; GENETIC THERAPY; HUMANS; NATIONAL INSTITUTES OF HEALTH (U.S.); RARE DISEASES; TRANSLATIONAL MEDICAL RESEARCH; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 84876539014     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2013.064     Document Type: Review

References (16)

1. Aiuti, A., Cattaneo, F., Galimberti, S., et al. (2009). Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N. Engl. J. Med. 360, 447-458.
2. Aiuti, A., Bacchetta, R., Seger, R., et al. (2012). Gene therapy for primary immunodeficiencies: part 2. Curr. Opin. Immunol. 24, 585-591.
3. Booth, C., Gaspar, H.B., and Thrasher, A. (2011). Gene therapy for primary immunodeficiency. Curr. Opin. Pediatr. 23, 659-666.
4. Boztug, K., Schmidt, M., Schwarzer, A., et al. (2010). Stem-cell gene therapy for Wiskott-Aldrich syndrome. N. Engl. J. Med. 363, 1918-1927.
5. Cartier, N., Hacein-Bey-Abina, S., Bartholomae, C. et al. (2009). Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 326, 818-823.
6. Cavazzana-Calvo, M., Payen, E., Negre, O., et al. (2010). Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia. Nature 467, 318-322.
7. Cideciyan, A.V. (2010). Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy. Prog. Retin. Eye Res. 29, 398-427.
8. Fangone, P., Wright, J.F., Nathwani, A., et al. (2012). Systemic errors in quantitative polymerase chain reaction titration of self-complementary adeno-associated viral vectors and improved alternative methods. Hum. Gene Ther. Methods 23, 1-7.
9. Hacein-Bey-Abina, S., von Kalle, C., Schmidt, M., et al. (2003). LMO-2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302, 415-419.
10. Hacein-Bey-Abina, S., Hauer, J., Lim, A., et al. (2010). Efficacy of gene therapy for X-linked severe combined immunodeficiency. N. Engl. J. Med. 363, 355-364.
11. Jacobson, S., Cideciyan, A., Ratnakaram, R., et al. (2012). Gene therapy for Leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years. Arch. Ophthalmol. 130, 9-24.
12. Manno, C., Pierce, G., Arruda, V., et al. (2006). Successful trans-duction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat. Med. 12, 342-347.
13. Mingozzi, F., Maus, M., Hui, D., et al. (2007). CD8 + T-cell responses to adeno-associated virus capsid in humans. Nat. Med. 13, 419-422.
14. Nathwani, A.C., Tuddenham, E.G., Rangarajan, S., et al. (2011). Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N. Engl. J. Med. 365, 2357-2365.
15. Stein, S., Ott, M., Schultze-Strasser, S., et al. (2010). Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granuloma-tous disease. Nat. Med. 16, 198-204.
16. Stroes, E.S., Nierman, M., Meulenberg, J., et al. (2008). Intramuscular administration of AAV1-lipoprotein lipase S447X lowers triglycerides in lipoprotein lipase-deficient patients. Arterioscler. Thromb. Vasc. Biol. 28, 2303-2304.