Recombinant Adeno-Associated Virus-Mediated Gene Transfer for the Potential Therapy of Adenosine Deaminase-Deficient Severe Combined Immune Deficiency
نویسندگان
چکیده
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 کاملPlenary paper Gene therapy for adenosine deaminase–deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans
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. ...
متن کاملKinetics of recombinant adeno-associated virus-mediated gene transfer.
Recombinant adeno-associated virus (rAAV) vectors have been shown to be useful for efficient gene delivery to a variety of dividing and nondividing cells. Mechanisms responsible for the long-term, persistent expression of the rAAV transgene are not well understood. In this study we investigated the kinetics of rAAV-mediated human factor IX (hFIX) gene transfer into human primary myoblasts and m...
متن کاملDevelopment of gene therapy: potential in severe combined immunodeficiency due to adenosine deaminase deficiency
A 4-month-old male received a T-lymphocyte-depleted haploidentical bone marrow transplant (BMT) for correction of severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency. Although previous haploidentical bone marrow transplants have been attempted in ADA-deficient SCID, complete reconstitution of both B-lymphocyte and T-lymphocyte function has not been obtained after...
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...
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ژورنال
عنوان ژورنال: Human Gene Therapy
سال: 2011
ISSN: 1043-0342,1557-7422
DOI: 10.1089/hum.2010.121