Development and Application of Gene Therapy Technologies
Author:
Affiliation:
1. Division of Hematology, Department of Medicine, Division of Cell Transplantation and Transfusion, Division of Genetic Therapeutics, Center for Molecular Medicine
Publisher
Japanese Association of Virology
Subject
General Medicine
Link
https://www.jstage.jst.go.jp/article/jsv/54/1/54_1_49/_pdf
Reference45 articles.
1. Gene therapy using AAV (adeno-associated virus) vectors
2. 2) Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, Gross F, Yvon E, Nusbaum P, Selz F, Hue C, Certain S, Casanova JL, Bousso P, Deist FL, Fischer A : Gene therapy of human severe combined immunodeficiency (SCID)-X 1 disease. Science 288 : 669-672, 2000.
3. 3) Hacein-Bey-Abina S, Le Deist F, Carlier F, Bouneaud C, Hue C, De Villartay JP, Thrasher AJ, Wulffraat N, Sorensen R, Dupuis-Girod S, Fischer A, Davies EG, Kuis W, Leiva L, Cavazzana-Calvo M : Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N Engl J Med 346 : 1185-1193, 2002.
4. 4) European Society of Gene Therapy : French gene therapy group reports on the adverse event in a clinical trial of gene therapy for X-linked severe combined immune deficiency (X-SCID). J Gene Med 5 : 82-84, 2003.
5. 5) Hacein-Bey-Abina S, von Kalle C, Schmidt M, Le Deist F, Wulffraat N, McIntyre E, Radford I, Villeval JL, Fraser CC, Cavazzana-Calvo M, Fischer A : A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 348 : 255-256, 2003.
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