Xavier M. Anguela

Nanotherapeutics (United States) — United States

Biochemistry, Genetics and Molecular Biology · Life Sciences

22h-index4.1kcitations43works14.52yr avg
OpenAlex↗ORCID↗Google Scholar↗Lab page↗

Accepting Students?

No reports yet. Know if this professor is taking students?

Research Topics

Virus-based gene therapy research(33), CRISPR and Genetic Engineering(16), CAR-T cell therapy research(15), Viral Infectious Diseases and Gene Expression in Insects(8), RNA Interference and Gene Delivery(7)

Publications43 total

Abstract C007: A versatile microRNA-based platform for activation-dependent regulation of armored CAR T cell payloads
Cancer Immunology Research·2026
128P Non-coding DNA lipid nanoparticles elicit antitumor immune responses and synergize with anti-PDL1 antibodies in mouse models of hepatocellular carcinoma
Immuno-Oncology Technology·2024Open Access
ARI0003: Co-transduced CD19/BCMA dual-targeting CAR-T cells for the treatment of non-Hodgkin lymphoma
Molecular Therapy·2024· 23 citedOpen Access
Hemophilia B and gene therapy: a new chapter with etranacogene dezaparvovec
Blood Advances·2024· 35 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
Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A
New England Journal of Medicine·2021· 235 citedOpen Access
Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates
Nature Communications·2021· 36 citedOpen Access
IdeS: An enabling technology to overcome the limitation of neutralizing antibodies to AAV gene therapy
Molecular Genetics and Metabolism·2021· 1 cited
Experimental Variables that Affect Human Hepatocyte AAV Transduction in Liver Chimeric Mice
2020
Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B
Molecular Therapy·2020· 198 citedOpen Access
Experimental Variables that Affect Human Hepatocyte AAV Transduction in Liver Chimeric Mice
Molecular Therapy — Methods & Clinical Development·2020· 25 citedOpen Access
IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies
Nature Medicine·2020· 296 citedOpen Access
Preclinical development of , an investigational liver-directed AAV gene therapy for the treatment of Pompe disease
Molecular Genetics and Metabolism·2020
Efficacy and Safety in 15 Hemophilia B Patients Treated with the AAV Gene Therapy Vector Fidanacogene Elaparvovec and Followed for at Least 1 Year
Blood·2019· 36 cited
O.6Pre-clinical development of SPK-3006, an investigational liver-directed AAV gene therapy for the treatment of Pompe disease
Neuromuscular Disorders·2019· 2 cited
Oracle or false prophet? Can we predict AAV efficacy based on preexisting antibody titers?
Research and Practice in Thrombosis and Haemostasis·2019· 2 citedOpen Access
Safety and efficacy evaluation of investigational liver gene transfer for secretable GAA in the treatment of Pompe disease
Molecular Genetics and Metabolism·2019· 1 cited
A Phase 1/2 Trial of Investigational Spk-8011 in Hemophilia a Demonstrates Durable Expression and Prevention of Bleeds
Blood·2018· 57 cited
Entering the Modern Era of Gene Therapy
Annual Review of Medicine·2018· 457 cited
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant
New England Journal of Medicine·2017· 736 citedOpen Access
page 1 of 3Next →

Frequent Co-authors

Katherine A. High(27), Virginia Haurigot(10), Shangzhen Zhou(9), Yannick Doyon(8), Rajiv P. Sharma(8), Philip D. Gregory(8), Michael C. Holmes(8), Sean M. Armour(8), Federico Mingozzi(8), Fátima Bosch(7), Lindsey A. George(6), David E. Paschon(6), Robert J. Davidson(6), Spencer K. Sullivan(5), Margaret V. Ragni(5), Daniel J. Hui(5), J. Fraser Wright(5), Hojun Li(5), Edward J. Rebar(5), Robert J. Davidson(5)