Denise A. Carbonaro

Biochemistry, Genetics and Molecular Biology · Life Sciences

21h-index2.0kcitations43works0.02yr avg
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Research Topics

Virus-based gene therapy research(28), CAR-T cell therapy research(16), RNA Interference and Gene Delivery(13), Immunodeficiency and Autoimmune Disorders(7), Animal Behavior and Welfare Studies(3)

Publications43 total

Transcatheter Aortic Valve with Embolic Filter: Experiments and Simulations
2020· 1 cited
Clinical efficacy of gene-modified stem cells in adenosine deaminase–deficient immunodeficiency
Journal of Clinical Investigation·2017· 79 citedOpen Access
Towards a phase I clinical trial for autologous hematopoietic stem cells transplantation in cystinosis
Molecular Genetics and Metabolism·2017
Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency
Human Gene Therapy·2016· 53 citedOpen Access
32. Towards a Phase I Clinical Trial for Cystinosis
Molecular Therapy·2016· 2 citedOpen Access
292. Lentivirus Vector Mediated Gene Correction in Artemis-Deficient Severe Combined Immunodeficiency
Molecular Therapy·2015Open Access
84. Pharmacology/Toxicology Studies for Gene-Modified Hematopoietic Stem Cell Transplantation for Cystinosis
Molecular Therapy·2015Open Access
30. Phase II Clinical Trial of Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA-SCID) Using a γ-Retroviral Vector
Molecular Therapy·2015· 2 citedOpen Access
C-8. Immunological and Metabolic Correction After Lentiviral Vector Gene Therapy for ADA Deficiency
Molecular Therapy·2015· 10 citedOpen Access
Autologous Transplant/Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immune Deficiency
Biology of Blood and Marrow Transplantation·2015Open Access
Cell and gene therapy in the murine model of adenosine deaminase deficiency
University of Southern California Digital Library·2015Open Access
Preclinical Demonstration of Lentiviral Vector-mediated Correction of Immunological and Metabolic Abnormalities in Models of Adenosine Deaminase Deficiency
2014
Preclinical Demonstration of Lentiviral Vector-mediated Correction of Immunological and Metabolic Abnormalities in Models of Adenosine Deaminase Deficiency
Molecular Therapy·2013· 80 citedOpen Access
Gene therapy for adenosine deaminase–deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans
Blood·2012· 233 citedOpen Access
Gene therapy/bone marrow transplantation in ADA-deficient mice: roles of enzyme-replacement therapy and cytoreduction
Blood·2012· 42 citedOpen Access
Immune Reconstitution After Gene Therapy (GTx) for Adenosine Deaminase Deficient Severe Combined Immune Deficiency (ADA-SCID)
Biology of Blood and Marrow Transplantation·2009· 3 citedOpen Access
Neonatal bone marrow transplantation of ADA-deficient SCID mice results in immunologic reconstitution despite low levels of engraftment and an absence of selective donor T lymphoid expansion
Blood·2008· 25 citedOpen Access
68: Gene Therapy for Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID): Comparative Results with or without PEG-ADA Withdrawal and Myelosuppressive Chemotherapy
Biology of Blood and Marrow Transplantation·2008· 2 citedOpen Access
Comparative Results of Gene Therapy for Adenosine Deaminase Deficiency with or without PEG-ADA Withdrawal and Myelosuppressive Chemotherapy.
Blood·2007· 3 cited
Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report
Blood·2006· 35 citedOpen Access
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Frequent Co-authors

Donald B. Kohn(31), Barbara C. Engel(11), Fabio Candotti(10), Robert Sokolic(8), Michael S. Hershfield(8), Linda Muul(7), Elizabeth Garabedian(7), Greg M. Podsakoff(7), Xiangyang Jin(7), Gay M. Crooks(5), Aaron Cooper(5), Alan S. Wayne(5), Xiaoyan Wang(5), Kenneth Cornetta(5), E Smogorzewska(4), Neena Kapoor(4), Kenneth I. Weinberg(4), Gerhard Bauer(4), Kit L. Shaw(4), Ami J. Shah(4)