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Human Gene Therapy. Clinical Development
|
November 19, 2016
Transfer of Therapeutic Genes into Fetal Rhesus Monkeys Using Recombinant Adeno-Associated Type I Viral Vectors
Thomas J Conlon, Cathryn S Mah, Christina A Pacak, et al.
Human Gene Therapy
|
September 9, 2008
Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial
William W Hauswirth, Tomas S Aleman, Shalesh Kaushal, et al.
Human Gene Therapy. Clinical Development
|
November 22, 2017
Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease
Manuela Corti, Cristina Liberati, Barbara K Smith, et al.
Journal of Huntington'S Disease
|
October 16, 2018
Selective Neuronal Uptake and Distribution of AAVrh8, AAV9, and AAVrh10 in Sheep After Intra-Striatal Administration
Erica Mondo, Richard Moser, Guangping Gao, et al.
The New England Journal of Medicine
|
August 14, 2009
Vision 1 year after gene therapy for Leber's congenital amaurosis
Artur V Cideciyan, William W Hauswirth, Tomas S Aleman, et al.
Human Gene Therapy
|
April 11, 2013
Phase I/II trial of adeno-associated virus-mediated alpha-glucosidase gene therapy to the diaphragm for chronic respiratory failure in Pompe disease: initial safety and ventilatory outcomes
Barbara K Smith, Shelley W Collins, Thomas J Conlon, et al.
Human Gene Therapy
|
November 23, 2006
Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults
Mark L Brantly, L Terry Spencer, Margaret Humphries, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
August 27, 2009
Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy
Mark L Brantly, Jeffrey D Chulay, Lili Wang, et al.
Investigative Ophthalmology & Visual Science
|
October 25, 2014
LHON gene therapy vector prevents visual loss and optic neuropathy induced by G11778A mutant mitochondrial DNA: biodistribution and toxicology profile
Rajeshwari Koilkonda, Hong Yu, Venu Talla, et al.
Journal of Biomedicine & Biotechnology
|
February 15, 2011
Glycogen storage disease type Ia in canines: a model for human metabolic and genetic liver disease
Andrew Specht, Laurie Fiske, Kirsten Erger, et al.
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Search research articles
Search
Showing results (21-30 of 42) with videos related to
Sort By:
Page
of 5
Human Gene Therapy. Clinical Development
|
November 19, 2016
Transfer of Therapeutic Genes into Fetal Rhesus Monkeys Using Recombinant Adeno-Associated Type I Viral Vectors
Thomas J Conlon, Cathryn S Mah, Christina A Pacak, et al.
Human Gene Therapy
|
September 9, 2008
Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial
William W Hauswirth, Tomas S Aleman, Shalesh Kaushal, et al.
Human Gene Therapy. Clinical Development
|
November 22, 2017
Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease
Manuela Corti, Cristina Liberati, Barbara K Smith, et al.
Journal of Huntington'S Disease
|
October 16, 2018
Selective Neuronal Uptake and Distribution of AAVrh8, AAV9, and AAVrh10 in Sheep After Intra-Striatal Administration
Erica Mondo, Richard Moser, Guangping Gao, et al.
The New England Journal of Medicine
|
August 14, 2009
Vision 1 year after gene therapy for Leber's congenital amaurosis
Artur V Cideciyan, William W Hauswirth, Tomas S Aleman, et al.
Human Gene Therapy
|
April 11, 2013
Phase I/II trial of adeno-associated virus-mediated alpha-glucosidase gene therapy to the diaphragm for chronic respiratory failure in Pompe disease: initial safety and ventilatory outcomes
Barbara K Smith, Shelley W Collins, Thomas J Conlon, et al.
Human Gene Therapy
|
November 23, 2006
Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults
Mark L Brantly, L Terry Spencer, Margaret Humphries, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
August 27, 2009
Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy
Mark L Brantly, Jeffrey D Chulay, Lili Wang, et al.
Investigative Ophthalmology & Visual Science
|
October 25, 2014
LHON gene therapy vector prevents visual loss and optic neuropathy induced by G11778A mutant mitochondrial DNA: biodistribution and toxicology profile
Rajeshwari Koilkonda, Hong Yu, Venu Talla, et al.
Journal of Biomedicine & Biotechnology
|
February 15, 2011
Glycogen storage disease type Ia in canines: a model for human metabolic and genetic liver disease
Andrew Specht, Laurie Fiske, Kirsten Erger, et al.
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of 5