FindTrial
Sign In

Gene transfer

GENETIC4 trials

Sponsors

National Institute of Dental and Craniofacial Research (NIDCR), St. Jude Children's Research Hospital, David Williams, Assistance Publique - Hôpitaux de Paris

Conditions

Hemophilia BParotid Salivary DysfunctionSevere Combined ImmunodeficiencyX-linked Severe Combined Immunodeficiency

Phase 1

Effect of AdhAQP1 on Salivary Flow in Patients Treated With Radiation for Head and Neck Cancer
CompletedNCT00372320
National Institute of Dental and Craniofacial Research (NIDCR)Parotid Salivary Dysfunction
Start: 2006-09-01End: 2018-09-05Updated: 2018-09-11
Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B
CompletedNCT00979238
St. Jude Children's Research HospitalHemophilia B
Start: 2010-02-22End: 2026-03-26Updated: 2026-04-02
Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector
CompletedNCT01129544
David WilliamsSevere Combined Immunodeficiency
Start: 2010-04-01End: 2023-03-01Updated: 2026-04-21
Gene Therapy for X-linked Severe Combined Immunodeficiency
CompletedNCT01410019
Assistance Publique - Hôpitaux de ParisX-linked Severe Combined Immunodeficiency
Start: 2010-12-01End: 2015-06-16Updated: 2026-03-11

Related Papers

Decoding Transgene Silencing from Stably Maintained AAV Genomes in Human Liver: Epigenetic Insights to Guide Next-Generation Vector Design
Blood2025-11-03
Sustained Clinical Benefit of AAV Gene Therapy in Severe Hemophilia B
New England Journal of Medicine2025-06-1120 citations
Recent developments in gene therapy research in India.
2024-01-01
Stable Therapeutic Transgenic FIX Levels for More Than 10 Years in Subjects with Severe Hemophilia B Who Received scAAV2/8-LP1-Hfixco Adeno-Associated Virus Gene Therapy
Blood2023-11-0214 citations
Long-Term Persistence of Antibodies to Adeno-Associated Viral Vectors Following Gene Therapy with scAAV8-LP1-Fixco
Blood2023-11-024 citations
Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing "Empty Viral Particles" on Safety and Efficacy of Gene Transfer
Blood2018-11-29112 citations
GO-8: Preliminary Results of a Phase I/II Dose Escalation Trial of Gene Therapy for Haemophilia a Using a Novel Human Factor VIII Variant
Blood2018-11-2956 citations
T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency.
2018-09-2825 citations
Late responses to adenoviral-mediated transfer of the aquaporin-1 gene for radiation-induced salivary hypofunction.
2016-12-2049 citations
Technique Improvement and Experience Sharing of Cannulation of the Mouse Submandibular Salivary Gland via the Wharton's Duct
2016-12-01
Persistence of hAQP1 expression in human salivary gland cells following AdhAQP1 transduction is associated with a lack of methylation of hCMV promoter.
2015-07-1616 citations
325. Persistence of hAQP1 Expression in Human Salivary Gland Cells Following AdhAQP1 Transduction Is Associated With a Lack of Methylation of hCMV Promoter
Molecular Therapy2015-05-011 citations
Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
New England Journal of Medicine2014-11-201235 citations
A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency
New England Journal of Medicine2014-10-08402 citations
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.
2011-12-101401 citations