Plenary paper Gene therapy for adenosine deaminase–deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans
نویسندگان
چکیده
Fabio Candotti,1 Kit L. Shaw,2 Linda Muul,1 Denise Carbonaro,2 Robert Sokolic,1 Christopher Choi,2 Shepherd H. Schurman,1 Elizabeth Garabedian,1 Chimene Kesserwan,1 G. Jayashree Jagadeesh,1 Pei-Yu Fu,2 Eric Gschweng,2 Aaron Cooper,3 John F. Tisdale,4 Kenneth I. Weinberg,5 Gay M. Crooks,6 Neena Kapoor,7 Ami Shah,7 Hisham Abdel-Azim,7 Xiao-Jin Yu,7 Monika Smogorzewska,7 Alan S. Wayne,8 Howard M. Rosenblatt,9 Carla M. Davis,10 Celine Hanson,10 Radha G. Rishi,11 Xiaoyan Wang,12 David Gjertson,6,12 Otto O. Yang,13 Arumugam Balamurugan,13 Gerhard Bauer,14 Joanna A. Ireland,7 Barbara C. Engel,15 Gregory M. Podsakoff,16 Michael S. Hershfield,17 R. Michael Blaese,18 Robertson Parkman,7 and Donald B. Kohn2,19
منابع مشابه
Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans.
We conducted a gene therapy trial in 10 patients with adenosine deaminase (ADA)-deficient severe combined immunodeficiency using 2 slightly different retroviral vectors for the transduction of patients' bone marrow CD34(+) cells. Four subjects were treated without pretransplantation cytoreduction and remained on ADA enzyme-replacement therapy (ERT) throughout the procedure. Only transient (mont...
متن کاملGene therapy of primary T cell immunodeficiencies.
Gene therapy of severe combined immunodeficiencies has been proven to be effective to provide sustained correction of the T cell immunodeficiencies. This has been achieved for 2 forms of SCID, i.e SCID-X1 (γc deficiency) and adenosine deaminase deficiency. Occurrence of gene toxicity generated by integration of first generation retroviral vectors, as observed in the SCID-X1 trials has led to re...
متن کامل[Gene therapy for adenosine deaminase deficiency].
Severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency is a fatal recessive disorder caused by mutations in the gene encoding ADA. Based on the first clinical trial of two young girls with ADA-deficient SCID by recombinant retrovirus-mediated gene transfer at the National Institute of Health of USA, we prepared to treat a four-year-old boy with ADA-deficient SCID wh...
متن کاملSuccessful peripheral T-lymphocyte-directed gene transfer for a patient with severe combined immune deficiency caused by adenosine deaminase deficiency.
Ten patients with adenosine deaminase deficiency (ADA-) have been enrolled in gene therapy clinical trials since the first patient was treated in September 1990. We describe a Japanese ADA- severe combined immune deficiency (SCID) patient who has received periodic infusions of genetically modified autologous T lymphocytes transduced with the human ADA cDNA containing retroviral vector LASN. The...
متن کاملTen years of gene therapy for primary immune deficiencies.
Gene therapy with hematopoietic stem cells (HSC) is an attractive therapeutic strategy for several forms of primary immunodeficiencies. Current approaches are based on ex vivo gene transfer of the therapeutic gene into autologous HSC by vector-mediated gene transfer. In the past decade, substantial progress has been achieved in the treatment of severe combined immundeficiencies (SCID)-X1, adeno...
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