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Science Translational Medicine
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April 9, 2010
Safety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindness
Defne Amado, Federico Mingozzi, Daniel Hui, et al.
Molecular Therapy. Methods & Clinical Development
|
January 21, 2021
Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector
Giulia De Sabbata, Florence Boisgerault, Corrado Guarnaccia, et al.
Scientific Reports
|
November 5, 2016
Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients
Elisa Masat, Pascal Laforêt, Marie De Antonio, et al.
Nature Medicine
|
March 21, 2007
CD8(+) T-cell responses to adeno-associated virus capsid in humans
Federico Mingozzi, Marcela V Maus, Daniel J Hui, et al.
Hepatology (Baltimore, Md.)
|
March 21, 2017
Autophagy determines efficiency of liver-directed gene therapy with adeno-associated viral vectors
Marianna Hösel, Anke Huber, Susanne Bohlen, et al.
The Journal of Clinical Investigation
|
July 19, 2013
Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy
Virginia Haurigot, Sara Marcó, Albert Ribera, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
April 29, 2010
Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs
Virginia Haurigot, Federico Mingozzi, George Buchlis, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
July 17, 2013
Modulation of CD8+ T cell responses to AAV vectors with IgG-derived MHC class II epitopes
Daniel J Hui, Etiena Basner-Tschakarjan, Yifeng Chen, et al.
Ebiomedicine
|
October 11, 2020
Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects
Pasqualina Colella, Pauline Sellier, Manuel J Gomez, et al.
Human Gene Therapy
|
September 11, 2019
Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler-Najjar Syndrome
Sem J Aronson, Philippe Veron, Fanny Collaud, et al.
Page
of 14
Search research articles
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Showing results (91-100 of 138) with videos related to
Sort By:
Page
of 14
Science Translational Medicine
|
April 9, 2010
Safety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindness
Defne Amado, Federico Mingozzi, Daniel Hui, et al.
Molecular Therapy. Methods & Clinical Development
|
January 21, 2021
Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector
Giulia De Sabbata, Florence Boisgerault, Corrado Guarnaccia, et al.
Scientific Reports
|
November 5, 2016
Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients
Elisa Masat, Pascal Laforêt, Marie De Antonio, et al.
Nature Medicine
|
March 21, 2007
CD8(+) T-cell responses to adeno-associated virus capsid in humans
Federico Mingozzi, Marcela V Maus, Daniel J Hui, et al.
Hepatology (Baltimore, Md.)
|
March 21, 2017
Autophagy determines efficiency of liver-directed gene therapy with adeno-associated viral vectors
Marianna Hösel, Anke Huber, Susanne Bohlen, et al.
The Journal of Clinical Investigation
|
July 19, 2013
Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy
Virginia Haurigot, Sara Marcó, Albert Ribera, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
April 29, 2010
Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs
Virginia Haurigot, Federico Mingozzi, George Buchlis, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
July 17, 2013
Modulation of CD8+ T cell responses to AAV vectors with IgG-derived MHC class II epitopes
Daniel J Hui, Etiena Basner-Tschakarjan, Yifeng Chen, et al.
Ebiomedicine
|
October 11, 2020
Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects
Pasqualina Colella, Pauline Sellier, Manuel J Gomez, et al.
Human Gene Therapy
|
September 11, 2019
Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler-Najjar Syndrome
Sem J Aronson, Philippe Veron, Fanny Collaud, et al.
Page
of 14