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Human Gene Therapy
|
June 15, 2018
Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease
Allison M Keeler, Marina Zieger, Sophia H Todeasa, et al.
Molecular Therapy. Methods & Clinical Development
|
March 6, 2024
Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in <i>Cynomolgus macaques</i>
Meghan Blackwood, Alisha M Gruntman, Qiushi Tang, et al.
Genes
|
September 28, 2024
Limb Perfusion Delivery of a rAAV1 Alpha-1 Antitrypsin Vector in Non-Human Primates Is Safe but Insufficient for Therapy
Debora Pires-Ferreira, Darcy Reil, Qiushi Tang, et al.
Iscience
|
September 14, 2019
Pollen-derived RNAs Are Found in the Human Circulation
Milka Koupenova, Eric Mick, Heather A Corkrey, et al.
Molecular Therapy. Methods & Clinical Development
|
March 5, 2019
Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study
Alisha M Gruntman, Gwladys Gernoux, Qiushi Tang, et al.
Frontiers in Immunology
|
May 17, 2021
Novel Combinatorial MicroRNA-Binding Sites in AAV Vectors Synergistically Diminish Antigen Presentation and Transgene Immunity for Efficient and Stable Transduction
Manish Muhuri, Wei Zhan, Yukiko Maeda, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
December 29, 2015
Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector
Sourav R Choudhury, Anne F Harris, Damien J Cabral, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
March 12, 2026
SORT LNPs encapsulating Cas9 mRNA achieve efficient editing in skeletal muscle in a dystrophic mouse model
Sukanya Iyer, Katelyn Daman, Yehui Sun, et al.
Nature Medicine
|
February 11, 2022
AAV gene therapy for Tay-Sachs disease
Terence R Flotte, Oguz Cataltepe, Ajit Puri, et al.
The New England Journal of Medicine
|
February 10, 2026
AAV9 Gene Therapy in Type II GM1 Gangliosidosis - A Phase 1-2 Trial
Connor J Lewis, Precilla D'Souza, Jean M Johnston, et al.
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Search research articles
Search
Showing results (21-30 of 32) with videos related to
Sort By:
Page
of 4
Human Gene Therapy
|
June 15, 2018
Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease
Allison M Keeler, Marina Zieger, Sophia H Todeasa, et al.
Molecular Therapy. Methods & Clinical Development
|
March 6, 2024
Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in <i>Cynomolgus macaques</i>
Meghan Blackwood, Alisha M Gruntman, Qiushi Tang, et al.
Genes
|
September 28, 2024
Limb Perfusion Delivery of a rAAV1 Alpha-1 Antitrypsin Vector in Non-Human Primates Is Safe but Insufficient for Therapy
Debora Pires-Ferreira, Darcy Reil, Qiushi Tang, et al.
Iscience
|
September 14, 2019
Pollen-derived RNAs Are Found in the Human Circulation
Milka Koupenova, Eric Mick, Heather A Corkrey, et al.
Molecular Therapy. Methods & Clinical Development
|
March 5, 2019
Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study
Alisha M Gruntman, Gwladys Gernoux, Qiushi Tang, et al.
Frontiers in Immunology
|
May 17, 2021
Novel Combinatorial MicroRNA-Binding Sites in AAV Vectors Synergistically Diminish Antigen Presentation and Transgene Immunity for Efficient and Stable Transduction
Manish Muhuri, Wei Zhan, Yukiko Maeda, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
December 29, 2015
Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector
Sourav R Choudhury, Anne F Harris, Damien J Cabral, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
March 12, 2026
SORT LNPs encapsulating Cas9 mRNA achieve efficient editing in skeletal muscle in a dystrophic mouse model
Sukanya Iyer, Katelyn Daman, Yehui Sun, et al.
Nature Medicine
|
February 11, 2022
AAV gene therapy for Tay-Sachs disease
Terence R Flotte, Oguz Cataltepe, Ajit Puri, et al.
The New England Journal of Medicine
|
February 10, 2026
AAV9 Gene Therapy in Type II GM1 Gangliosidosis - A Phase 1-2 Trial
Connor J Lewis, Precilla D'Souza, Jean M Johnston, et al.
Page
of 4