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Molecular Therapy : the Journal of the American Society of Gene Therapy
|
February 26, 2009
Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice
Ryan L Boudreau, Jodi L McBride, Inês Martins, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
September 13, 2005
Workshop on long-term follow-up of participants in human gene transfer research
Kara Nyberg, Barrie J Carter, Theresa Chen, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
April 10, 2008
Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi
Jodi L McBride, Ryan L Boudreau, Scott Q Harper, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
July 2, 2014
Charting a clear path: the ASGCT Standardized Pathways Conference
Hans-Peter Kiem, Christopher Baum, Frederic D Bushman, et al.
Human Gene Therapy
|
July 30, 2003
Phase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: a two-part clinical study
Terence R Flotte, Pamela L Zeitlin, Thomas C Reynolds, et al.
Human Gene Therapy
|
August 10, 2007
Repeated aerosolized AAV-CFTR for treatment of cystic fibrosis: a randomized placebo-controlled phase 2B trial
Richard B Moss, Carlos Milla, John Colombo, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
February 5, 2004
AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice
Robin J Ziegler, Scott M Lonning, Donna Armentano, et al.
Human Gene Therapy
|
August 7, 2002
A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies
John A Wagner, Ilynn B Nepomuceno, Anna H Messner, et al.
The New England Journal of Medicine
|
April 29, 2008
Effect of gene therapy on visual function in Leber's congenital amaurosis
James W B Bainbridge, Alexander J Smith, Susie S Barker, et al.
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of 2
Search research articles
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Showing results (11-20 of 19) with videos related to
Sort By:
Page
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You have reached the last page of results.
This site can display upto 19 results.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
February 26, 2009
Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice
Ryan L Boudreau, Jodi L McBride, Inês Martins, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
September 13, 2005
Workshop on long-term follow-up of participants in human gene transfer research
Kara Nyberg, Barrie J Carter, Theresa Chen, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
April 10, 2008
Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi
Jodi L McBride, Ryan L Boudreau, Scott Q Harper, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
July 2, 2014
Charting a clear path: the ASGCT Standardized Pathways Conference
Hans-Peter Kiem, Christopher Baum, Frederic D Bushman, et al.
Human Gene Therapy
|
July 30, 2003
Phase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: a two-part clinical study
Terence R Flotte, Pamela L Zeitlin, Thomas C Reynolds, et al.
Human Gene Therapy
|
August 10, 2007
Repeated aerosolized AAV-CFTR for treatment of cystic fibrosis: a randomized placebo-controlled phase 2B trial
Richard B Moss, Carlos Milla, John Colombo, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
February 5, 2004
AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice
Robin J Ziegler, Scott M Lonning, Donna Armentano, et al.
Human Gene Therapy
|
August 7, 2002
A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies
John A Wagner, Ilynn B Nepomuceno, Anna H Messner, et al.
The New England Journal of Medicine
|
April 29, 2008
Effect of gene therapy on visual function in Leber's congenital amaurosis
James W B Bainbridge, Alexander J Smith, Susie S Barker, et al.
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of 2