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Molecular Therapy. Methods & Clinical Development
|
October 30, 2019
AAV Gene Therapy Utilizing Glycosylation-Independent Lysosomal Targeting Tagged GAA in the Hypoglossal Motor System of Pompe Mice
Brendan M Doyle, Sara M F Turner, Michael D Sunshine, et al.
Genetic Vaccines and Therapy
|
June 20, 2012
An acidic oligopeptide displayed on AAV2 improves axial muscle tropism after systemic delivery
Ni-Chung Lee, Darin J Falk, Barry J Byrne, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
January 28, 2010
Gel-mediated delivery of AAV1 vectors corrects ventilatory function in Pompe mice with established disease
Cathryn S Mah, Darin J Falk, Sean A Germain, et al.
Human Molecular Genetics
|
August 23, 2022
Disease correction in mucopolysaccharidosis type IIIB mice by intraparenchymal or cisternal delivery of a capsid modified AAV8 codon-optimized NAGLU vector
Courtney J Rouse, Kimberley Hawkins, Nadia Kabbej, et al.
Biorxiv : the Preprint Server for Biology
|
January 7, 2025
Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease
David D Fuller, Sabhya Rana, Prajwal Thakre, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
June 16, 2025
Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease
David D Fuller, Sabhya Rana, Prajwal P Thakre, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
July 1, 2026
Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease
David D Fuller, Sabhya Rana, Prajwal P Thakre, et al.
Human Molecular Genetics
|
April 27, 2011
Pompe disease gene therapy
Barry J Byrne, Darin J Falk, Christina A Pacak, et al.
Human Gene Therapy
|
February 19, 2010
Adeno-associated virus-mediated correction of a canine model of glycogen storage disease type Ia
David A Weinstein, Catherine E Correia, Thomas Conlon, et al.
Page
of 5
Search research articles
Search
Showing results (41-50 of 49) with videos related to
Sort By:
Page
of 5
You have reached the last page of results.
This site can display upto 49 results.
Molecular Therapy. Methods & Clinical Development
|
October 30, 2019
AAV Gene Therapy Utilizing Glycosylation-Independent Lysosomal Targeting Tagged GAA in the Hypoglossal Motor System of Pompe Mice
Brendan M Doyle, Sara M F Turner, Michael D Sunshine, et al.
Genetic Vaccines and Therapy
|
June 20, 2012
An acidic oligopeptide displayed on AAV2 improves axial muscle tropism after systemic delivery
Ni-Chung Lee, Darin J Falk, Barry J Byrne, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
January 28, 2010
Gel-mediated delivery of AAV1 vectors corrects ventilatory function in Pompe mice with established disease
Cathryn S Mah, Darin J Falk, Sean A Germain, et al.
Human Molecular Genetics
|
August 23, 2022
Disease correction in mucopolysaccharidosis type IIIB mice by intraparenchymal or cisternal delivery of a capsid modified AAV8 codon-optimized NAGLU vector
Courtney J Rouse, Kimberley Hawkins, Nadia Kabbej, et al.
Biorxiv : the Preprint Server for Biology
|
January 7, 2025
Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease
David D Fuller, Sabhya Rana, Prajwal Thakre, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
June 16, 2025
Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease
David D Fuller, Sabhya Rana, Prajwal P Thakre, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
July 1, 2026
Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease
David D Fuller, Sabhya Rana, Prajwal P Thakre, et al.
Human Molecular Genetics
|
April 27, 2011
Pompe disease gene therapy
Barry J Byrne, Darin J Falk, Christina A Pacak, et al.
Human Gene Therapy
|
February 19, 2010
Adeno-associated virus-mediated correction of a canine model of glycogen storage disease type Ia
David A Weinstein, Catherine E Correia, Thomas Conlon, et al.
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of 5