Denise E. Sabatino

Biochemistry, Genetics and Molecular Biology · Life Sciences

27h-index5.6kcitations87works5.72yr avg
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Research Topics

Virus-based gene therapy research(51), Hemophilia Treatment and Research(40), CAR-T cell therapy research(19), Platelet Disorders and Treatments(15), CRISPR and Genetic Engineering(15)

Publications87 total

Small molecule inhibition of SUMOylation increases expression from AAV vectors both during and after initial transduction in mice
Molecular Therapy·2025Open Access
Modulation of AAV transduction and integration targeting by topoisomerase poisons
Molecular Therapy — Methods & Clinical Development·2024· 1 citedOpen Access
Improving the Assessment of Risk Factors Relevant to Potential Carcinogenicity of Gene Therapies: A Consensus Article
Human Gene Therapy·2024· 6 citedOpen Access
Optimizing liver health before and after gene therapy for hemophilia A
Blood Advances·2024· 13 citedOpen Access
Integration and the risk of liver cancer—Is there a real risk?
Journal of Viral Hepatitis·2024· 14 citedOpen Access
Promise of gene therapy for congenital neurologic disease due to GPI deficiency
Molecular Therapy — Methods & Clinical Development·2024Open Access
OC 30.5 Expression and Activity of Human Factor VIII (hFVIII) Following Single and Repeat Administration of an Integrating Non-Viral Gene Therapy in Multiple Mouse Models of Severe Hemophilia A
Research and Practice in Thrombosis and Haemostasis·2023Open Access
OC 30.4 Efficacy and Safety of AAV Delivery of a Novel Factor VIII Variant with Improved Secretion in a Non-Human Primate Model
Research and Practice in Thrombosis and Haemostasis·2023Open Access
Coagulation factor VIII regulates von Willebrand factor homeostasis in vivo
Journal of Thrombosis and Haemostasis·2023· 6 citedOpen Access
Altered cleavage of human factor VIII at the B-domain and acidic region 3 interface enhances expression after gene therapy in hemophilia A mice
Journal of Thrombosis and Haemostasis·2023· 8 citedOpen Access
Second Generation Adeno-Associated Viral Vector Using a Novel Factor VIII Variant with Improved Secretion Achieves Higher Factor VIII Expression in Non-Human Primates
Blood·2022
Coagulation Factor VIII Enhances the Cleavage of Von Willebrand Factor By ADAMTS13 <i>In Vivo</i>
Blood·2022
Sustained Factor VIII Activity Following Single Dose of Non-Viral Integrating Gene Therapy
Blood·2022· 2 cited
Evaluating the state of the science for adeno-associated virus integration: An integrated perspective
Molecular Therapy·2022· 180 citedOpen Access
Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A
Molecular Therapy — Methods & Clinical Development·2021· 15 citedOpen Access
Topics in AAV integration come front and center at ASGCT AAV Integration Roundtable
Molecular Therapy·2021· 9 citedOpen Access
Challenges in estimating numbers of vectors integrated in gene-modified cells using DNA sequence information
Molecular Therapy·2021· 13 citedOpen Access
Efficacy and Safety of Long-term Prophylaxis in Severe Hemophilia A Dogs Following Liver Gene Therapy Using AAV Vectors
UNC Libraries·2021Open Access
A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells
Nature Biotechnology·2020· 428 citedOpen Access
Animal Models of Hemophilia
UNC Libraries·2020Open Access
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Frequent Co-authors

Valder R. Arruda(13), Katherine A. High(11), Patrick G. Gallagher(8), Timothy C. Nichols(7), Rodney M. Camire(7), Amanda P. Cline(7), Giang N. Nguyen(6), Elizabeth P. Merricks(6), Haig H. Kazazian(6), Glenn F. Pierce(5), Margaret V. Ragni(5), Haiyan Jiang(5), Federico Mingozzi(5), Shangzhen Zhou(5), David M. Bodine(5), David M. Bodine(5), Lisa Garrett(5), Catherine S. Manno(4), John E.J. Rasko(4), Margareth C. Ozelo(4)