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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.
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.
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.
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.
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.
American Journal of Respiratory and Critical Care Medicine
|
July 17, 2020
Designer Receptors Exclusively Activated by Designer Drugs Approach to Treatment of Sleep-disordered Breathing
Thomaz Fleury Curado, Huy Pho, Carla Freire, et al.
Human Molecular Genetics
|
April 27, 2011
Pompe disease gene therapy
Barry J Byrne, Darin J Falk, Christina A Pacak, et al.
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of 13
Search research articles
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Showing results (121-130 of 129) with videos related to
Sort By:
Page
of 13
You have reached the last page of results.
This site can display upto 129 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.
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.
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.
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.
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.
American Journal of Respiratory and Critical Care Medicine
|
July 17, 2020
Designer Receptors Exclusively Activated by Designer Drugs Approach to Treatment of Sleep-disordered Breathing
Thomaz Fleury Curado, Huy Pho, Carla Freire, et al.
Human Molecular Genetics
|
April 27, 2011
Pompe disease gene therapy
Barry J Byrne, Darin J Falk, Christina A Pacak, et al.
Page
of 13