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The Journal of Clinical Investigation
|
May 14, 2009
Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors
Gary C Pien, Etiena Basner-Tschakarjan, Daniel J Hui, et al.
Blood
|
January 18, 2013
Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells
Ashley T Martino, Etiena Basner-Tschakarjan, David M Markusic, et al.
EMBO Molecular Medicine
|
March 12, 2020
Single-domain antibodies targeting antithrombin reduce bleeding in hemophilic mice with or without inhibitors
Elena Barbon, Gabriel Ayme, Amel Mohamadi, et al.
Journal of Inherited Metabolic Disease
|
March 7, 2023
Successful treatment of severe MSUD in Bckdhb<sup>-/-</sup> mice with neonatal AAV gene therapy
Clément Pontoizeau, Clovis Gaborit, Nolan Tual, et al.
Molecular Therapy. Methods & Clinical Development
|
January 29, 2021
Efficacy of AAV8-h<i>UGT1A1</i> with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients
Xiaoxia Shi, Sem J Aronson, Lysbeth Ten Bloemendaal, et al.
Plos One
|
March 6, 2014
Th1 response and systemic treg deficiency in inclusion body myositis
Yves Allenbach, Wahiba Chaara, Michelle Rosenzwajg, et al.
Human Molecular Genetics
|
December 20, 2014
Biochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy
Albert Ribera, Virginia Haurigot, Miguel Garcia, et al.
Nature Communications
|
June 7, 2022
Neonatal gene therapy achieves sustained disease rescue of maple syrup urine disease in mice
Clément Pontoizeau, Marcelo Simon-Sola, Clovis Gaborit, et al.
Molecular Therapy. Methods & Clinical Development
|
October 11, 2016
A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome
Giuseppe Ronzitti, Giulia Bortolussi, Remco van Dijk, et al.
Molecular Therapy. Methods & Clinical Development
|
December 2, 2024
Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I
Xiaoxia Shi, Giulia Bortolussi, Fanny Collaud, et al.
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of 14
Search research articles
Search
Showing results (81-90 of 138) with videos related to
Sort By:
Page
of 14
The Journal of Clinical Investigation
|
May 14, 2009
Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors
Gary C Pien, Etiena Basner-Tschakarjan, Daniel J Hui, et al.
Blood
|
January 18, 2013
Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells
Ashley T Martino, Etiena Basner-Tschakarjan, David M Markusic, et al.
EMBO Molecular Medicine
|
March 12, 2020
Single-domain antibodies targeting antithrombin reduce bleeding in hemophilic mice with or without inhibitors
Elena Barbon, Gabriel Ayme, Amel Mohamadi, et al.
Journal of Inherited Metabolic Disease
|
March 7, 2023
Successful treatment of severe MSUD in Bckdhb<sup>-/-</sup> mice with neonatal AAV gene therapy
Clément Pontoizeau, Clovis Gaborit, Nolan Tual, et al.
Molecular Therapy. Methods & Clinical Development
|
January 29, 2021
Efficacy of AAV8-h<i>UGT1A1</i> with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients
Xiaoxia Shi, Sem J Aronson, Lysbeth Ten Bloemendaal, et al.
Plos One
|
March 6, 2014
Th1 response and systemic treg deficiency in inclusion body myositis
Yves Allenbach, Wahiba Chaara, Michelle Rosenzwajg, et al.
Human Molecular Genetics
|
December 20, 2014
Biochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy
Albert Ribera, Virginia Haurigot, Miguel Garcia, et al.
Nature Communications
|
June 7, 2022
Neonatal gene therapy achieves sustained disease rescue of maple syrup urine disease in mice
Clément Pontoizeau, Marcelo Simon-Sola, Clovis Gaborit, et al.
Molecular Therapy. Methods & Clinical Development
|
October 11, 2016
A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome
Giuseppe Ronzitti, Giulia Bortolussi, Remco van Dijk, et al.
Molecular Therapy. Methods & Clinical Development
|
December 2, 2024
Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I
Xiaoxia Shi, Giulia Bortolussi, Fanny Collaud, et al.
Page
of 14