Preclinical Demonstration of Lentiviral Vector-mediated Correction of Immunological and Metabolic Abnormalities in Models of Adenosine Deaminase Deficiency

Author:

Carbonaro Denise A,Zhang Lin,Jin Xiangyang,Montiel-Equihua Claudia,Geiger Sabine,Carmo Marlene,Cooper Aaron,Fairbanks Lynette,Kaufman Michael L,Sebire Neil J,Hollis Roger P,Blundell Michael P,Senadheera Shantha,Fu Pei-Yu,Sahaghian Arineh,Chan Rebecca Y,Wang Xiaoyan,Cornetta Kenneth,Thrasher Adrian J,Kohn Donald B,Gaspar H Bobby

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference54 articles.

1. Adenosine deaminase deficiency: clinical expression, molecular basis, and therapy;Hershfield;Semin Hematol,1998

2. Adenosine deaminase deficiency increases thymic apoptosis and causes defective T cell receptor signaling;Apasov;J Clin Invest,2001

3. Bilateral sensorineural deafness in adenosine deaminase-deficient severe combined immunodeficiency;Albuquerque;J Pediatr,2004

4. Pathologic findings in adenosine deaminase-deficient severe combined immunodeficiency. I. Kidney, adrenal, and chondro-osseous tissue alterations;Ratech;Am J Pathol,1985

5. Adenosine deaminase-deficient mice generated using a two-stage genetic engineering strategy exhibit a combined immunodeficiency;Blackburn;J Biol Chem,1998

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