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JCI Insight
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July 9, 2025
Gene therapy enhances deoxyribonuclease I treatment in antimyeloperoxidase glomerulonephritis
Anne Cao Le, Virginie Oudin, Jonathan Dick, et al.
Molecular Therapy. Methods & Clinical Development
|
April 1, 2024
Novel AAV variants with improved tropism for human Schwann cells
Matthieu Drouyer, Tak-Ho Chu, Elodie Labit, et al.
Molecular Therapy. Methods & Clinical Development
|
January 3, 2022
Novel human liver-tropic AAV variants define transferable domains that markedly enhance the human tropism of AAV7 and AAV8
Marti Cabanes-Creus, Renina Gale Navarro, Erhua Zhu, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
April 4, 2023
Structural and functional characterization of capsid binding by anti-AAV9 monoclonal antibodies from infants after SMA gene therapy
Grant J Logan, Mario Mietzsch, Neeta Khandekar, et al.
Journal of Neuroinflammation
|
March 28, 2024
Liver-specific adiponectin gene therapy suppresses microglial NLRP3-inflammasome activation for treating Alzheimer's disease
Roy Chun-Laam Ng, Min Jian, Oscar Ka-Fai Ma, et al.
JHEP Reports : Innovation in Hepatology
|
February 11, 2020
Efficient <i>in vivo</i> editing of OTC-deficient patient-derived primary human hepatocytes
Samantha L Ginn, Anais K Amaya, Sophia H Y Liao, et al.
The Journal of Clinical Investigation
|
August 24, 2021
The self-peptide repertoire plays a critical role in transplant tolerance induction
Eric T Son, Pouya Faridi, Moumita Paul-Heng, et al.
Molecular Therapy. Methods & Clinical Development
|
January 26, 2023
Characterization of the humanized FRG mouse model and development of an AAV-LK03 variant with improved liver lobular biodistribution
Marti Cabanes-Creus, Renina Gale Navarro, Sophia H Y Liao, et al.
Molecular Therapy. Methods & Clinical Development
|
December 12, 2018
Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality
Marti Cabanes-Creus, Samantha L Ginn, Anais K Amaya, et al.
Molecular Therapy. Nucleic Acids
|
March 23, 2017
Limiting Thymic Precursor Supply Increases the Risk of Lymphoid Malignancy in Murine X-Linked Severe Combined Immunodeficiency
Samantha L Ginn, Claus V Hallwirth, Sophia H Y Liao, et al.
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Search research articles
Search
Showing results (101-110 of 131) with videos related to
Sort By:
Page
of 14
JCI Insight
|
July 9, 2025
Gene therapy enhances deoxyribonuclease I treatment in antimyeloperoxidase glomerulonephritis
Anne Cao Le, Virginie Oudin, Jonathan Dick, et al.
Molecular Therapy. Methods & Clinical Development
|
April 1, 2024
Novel AAV variants with improved tropism for human Schwann cells
Matthieu Drouyer, Tak-Ho Chu, Elodie Labit, et al.
Molecular Therapy. Methods & Clinical Development
|
January 3, 2022
Novel human liver-tropic AAV variants define transferable domains that markedly enhance the human tropism of AAV7 and AAV8
Marti Cabanes-Creus, Renina Gale Navarro, Erhua Zhu, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
April 4, 2023
Structural and functional characterization of capsid binding by anti-AAV9 monoclonal antibodies from infants after SMA gene therapy
Grant J Logan, Mario Mietzsch, Neeta Khandekar, et al.
Journal of Neuroinflammation
|
March 28, 2024
Liver-specific adiponectin gene therapy suppresses microglial NLRP3-inflammasome activation for treating Alzheimer's disease
Roy Chun-Laam Ng, Min Jian, Oscar Ka-Fai Ma, et al.
JHEP Reports : Innovation in Hepatology
|
February 11, 2020
Efficient <i>in vivo</i> editing of OTC-deficient patient-derived primary human hepatocytes
Samantha L Ginn, Anais K Amaya, Sophia H Y Liao, et al.
The Journal of Clinical Investigation
|
August 24, 2021
The self-peptide repertoire plays a critical role in transplant tolerance induction
Eric T Son, Pouya Faridi, Moumita Paul-Heng, et al.
Molecular Therapy. Methods & Clinical Development
|
January 26, 2023
Characterization of the humanized FRG mouse model and development of an AAV-LK03 variant with improved liver lobular biodistribution
Marti Cabanes-Creus, Renina Gale Navarro, Sophia H Y Liao, et al.
Molecular Therapy. Methods & Clinical Development
|
December 12, 2018
Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality
Marti Cabanes-Creus, Samantha L Ginn, Anais K Amaya, et al.
Molecular Therapy. Nucleic Acids
|
March 23, 2017
Limiting Thymic Precursor Supply Increases the Risk of Lymphoid Malignancy in Murine X-Linked Severe Combined Immunodeficiency
Samantha L Ginn, Claus V Hallwirth, Sophia H Y Liao, et al.
Page
of 14