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Gene Therapy in Combination with Nitrogen Scavenger Pretreatment Corrects Biochemical and Behavioral Abnormalities of Infant Citrullinemia Type 1 Mice
International journal of molecular sciences, 2022-11, Vol.23 (23), p.14940
2022
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Rescue of infant progressive familial intrahepatic cholestasis type 3 mice by repeated dosing of AAV gene therapy
JHEP reports, 2023-05, Vol.5 (5), p.100713-100713, Article 100713
2023
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Outcomes of 38 patients with PFIC3: Impact of genotype and of response to ursodeoxycholic acid therapy
JHEP reports, 2023-10, Vol.5 (10), p.100844-100844, Article 100844
2023
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Safety of rAAV2/2-ND4 Gene Therapy for Leber Hereditary Optic Neuropathy
Ophthalmology (Rochester, Minn.), 2018-06, Vol.125 (6), p.945-947
2018
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Epidemiology, treatment and burden of Wilson disease in France: A 10-year analysis of the national health insurance database
Clinics and research in hepatology and gastroenterology, 2022-12, Vol.46 (10), p.101992, Article 101992
2022
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A single dose, three-arm, placebo-controlled, phase i study of the bradykinin B2 receptor antagonist anatibant (LF16-0687Ms) in patients with severe traumatic brain injury
Journal of neurotrauma, 2005-12, Vol.22 (12), p.1444-1455
2005
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Optimising the IgG‐degrading enzyme treatment regimen for enhanced adeno‐associated virus transduction in the presence of neutralising antibodies
Clinical & translational immunology, 2022, Vol.11 (2), p.e1375-n/a
2022
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High value of 64Cu as a tool to evaluate the restoration of physiological copper excretion after gene therapy in Wilson’s disease
Molecular therapy. Methods & clinical development, 2022-09, Vol.26, p.98-106
2022
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A longitudinal study of epidemiology and treatment management of Wilson disease in France based on the French national claims database SNDS
Journal of hepatology, 2023-06, Vol.78, p.S989
2023
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THU-314 - A longitudinal study of epidemiology and treatment management of Wilson disease in France based on the French national claims database SNDS
Journal of hepatology, 2023-06, Vol.78, p.S989-S989
2023
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Multigram syntheses of the disaccharide repeating units of chondroitin 4- and 6-sulfates
Carbohydrate research, 1998-12, Vol.314 (3), p.283-288
1998
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Characteristics of patients with Wilson disease in the United States: An insurance claims database study
World journal of hepatology, 2024-05, Vol.16 (5), p.791-799
2024
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Targeting the kallikrein-kinin system as a new therapeutic approach to diabetic retinopathy
Current opinion in investigational drugs (London, England : 2000), 2010-05, Vol.11 (5), p.507
2010
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WED-164 Interim safety results of the ongoing international phase I/II GATEWAY gene therapy trial with VTX-801 conducted in adult patients with Wilson disease
Journal of hepatology, 2024-06, Vol.80, p.S714-S715
2024
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Chemical modification of the adeno-associated virus capsid to improve gene delivery
Chemical science (Cambridge), 2020, Vol.11 (4), p.1122-1131
2020
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Liver Expression of a MiniATP7B Gene Results in Long‐Term Restoration of Copper Homeostasis in a Wilson Disease Model in Mice
Hepatology (Baltimore, Md.), 2019-07, Vol.70 (1), p.108-126
2019
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The application of electrospray and neutral-loss mass spectrometry to the identification of the metabolites of S12813 in rat liver slices
Rapid communications in mass spectrometry, 1994-05, Vol.8 (5), p.361-365
1994
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FRI-430-Preclinical validation of copper 64 as a translational tool for evaluating the pharmacodynamics of VTX-801 genen therapy in Wilson’s disease
Journal of hepatology, 2019-04, Vol.70 (1), p.e583-e583
2019
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267. Phase I Gene Therapy Preliminary Clinical Results for Treatment of ND4 Leber Hereditary Optic Neuropathy with rAAV2-2-ND4
Molecular therapy, 2016-05, Vol.24, p.S106-S106
2016
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83. Relationship Between Immune Responses and Ocular Inflammation: What Is Learnt from Intravitreal Injection of rAAV2-2-ND4 (GS010) in Non-Human Primates and Leber Hereditary Optic Neuropathy (LHON) Patients
Molecular therapy, 2016-05, Vol.24, p.S36-S37
2016
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ChemInform Abstract: Multigram Syntheses of the Disaccharide Repeating Units of Chondroitin 4- and 6-Sulfates
ChemInform, 1999-10, Vol.30 (41), p.no-no
1999
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