Juan Ruiz

National Institute of Health Sciences — LK

Medicine · Health Sciences

8h-index401citations31works0.52yr avg
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Research Topics

Lysosomal Storage Disorders Research(13), Glycosylation and Glycoproteins Research(4), CRISPR and Genetic Engineering(4), Virus-based gene therapy research(3), Retinal Development and Disorders(3)

Publications31 total

RETINOPATIA DIABÉTICA: AMPLA ABORDAGEM DA CLÍNICA E DO TRATAMENTO
Brazilian Journal of Implantology and Health Sciences·2024· 1 citedOpen Access
Colonoscopy findings in patients with a positive FIT (Fecal Immunochemical Test)
Endoscopy·2024
Updated results of Transpher A, a multicenter, single-dose, Phase 1/2 clinical trial of ABO-102 investigational gene therapy for Sanfilippo syndrome type A (mucopolysaccharidosis IIIA) (S39.008)
Neurology·2022· 5 cited
Interim results of Transpher A, a multicentre, single-dose, phase 1/2 clinical trial of ABO-102 investigational gene therapy for Sanfilippo syndrome type A (mucopolysaccharidosis IIIA)
Molecular Genetics and Metabolism·2022· 1 cited
Updated results of Transpher A: multicenter, single-dose, phase 1/2 clinical trial of ABO-102 for Sanfilippo syndrome type A (mucopolysaccharidosis IIIA)
Molecular Genetics and Metabolism·2021· 3 cited
A disease progression model for trials in mucopolysaccharidosis type IIIA
Molecular Genetics and Metabolism·2021
Transpher A, a multicenter, single-dose, phase 1/2 clinical trial of ABO-102, an intravenous AAV9-based gene therapy for Sanfilippo Syndrome Type A (Mucopolysaccharidosis IIIA) (4898)
Neurology·2020· 3 cited
Safety, Tolerability, and Preliminary Evidence of Biopotency in Transpher B, a Multicenter, Singledose, Phase 1/2 Clinical Trial of ABO-101 Gene Therapy for Sanfilippo Syndrome Type B (Mucopolysaccharidosis IIIB) (5175)
Neurology·2020· 5 cited
Safety, tolerability and preliminary evidence of biopotency in Transpher B, a multicenter, single-dose, phase 1/2 clinical trial of ABO-101 gene therapy for Sanfilippo syndrome type B (mucopolysaccharidosis type IIIB)
Molecular Genetics and Metabolism·2020· 1 cited
Interim results of Transpher A, a multicenter, single-dose, phase 1/2 clinical trial of ABO-102 gene therapy for Sanfilippo syndrome type A (mucopolysaccharidosis type IIIA)
Molecular Genetics and Metabolism·2020· 4 cited
Phase I/II gene transfer clinical trial of scAAV9.U1a.hSGSH for MPS IIIA vector shedding results over 6 months post-gene transfer
Molecular Genetics and Metabolism·2019
Phase 1/2 clinical trial of systemic gene transfer of scAAV9.U1a.hSGSH for MPS IIIA demonstrates 2 years of safety, tolerability, and biopotency
Molecular Genetics and Metabolism·2019· 2 cited
A phase 1/2 clinical trial of systemic gene transfer of scAAV9.U1a.HSGSH for MPS IIIA: Safety, tolerability, and preliminary evidence of biopotency
Molecular Genetics and Metabolism·2018· 4 cited
Étude rétrospective sur la prescription de traitements antibactériens chez les patients drépanocytaires adultes hospitalisés dans les services de soins d’institut universitaire du cancer Oncopôle de Toulouse
La Revue de Médecine Interne·2016
Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria
Journal of Hepatology·2016· 139 citedOpen Access
Association of Obesity and Inflammatory Marker Levels on Treatment Outcome
The Journal of Clinical Psychiatry·2015· 55 cited
Association of obesity and levels of Inflammatory markers on treatment outcome with adjunctive L-methylfolate (Deplin) in major depressive disorder patients who are inadequate responders to selective serotonin reuptake Inhibitors
2015
Safety and efficacy of velaglucerase alfa in Gaucher disease type 1 patients previously treated with imiglucerase
American Journal of Hematology·2012· 61 citedOpen Access
Fabry Disease Antibody Standardization Initiative (FASI)
Molecular Genetics and Metabolism·2012· 2 cited
Safety evaluation of chronic intrathecal administration of heparan N-sulfatase in juvenile cynomolgus monkeys
Drug Delivery and Translational Research·2011· 13 cited
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Frequent Co-authors

Maria Fuller(8), Kevin M. Flanigan(7), Kristen Truxal(7), Kim L. McBride(7), María L. Couce(6), Astrid Pañeda(4), Louise Jaensch(4), Tabatha R. Simmons(4), Nicholas Smith(3), Maria L. Escolar(3), Maria Teresa Oreiro(3), Michael Pencina(2), Lori W. Barrentine(2), Maurizio Fava(2), John Zajecka(2), George I. Papakostas(2), B. Felice(2), Robert B. Boyd(2), Mark T. Butt(2), Richard W. Pfeifer(2)