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Molecular Therapy. Nucleic Acids
|
July 25, 2013
BEAMing and Droplet Digital PCR Analysis of Mutant IDH1 mRNA in Glioma Patient Serum and Cerebrospinal Fluid Extracellular Vesicles
Walter W Chen, Leonora Balaj, Linda M Liau, et al.
Science Advances
|
February 1, 2021
Gene therapy for tuberous sclerosis complex type 2 in a mouse model by delivery of AAV9 encoding a condensed form of tuberin
Pike-See Cheah, Shilpa Prabhakar, David Yellen, et al.
Journal of Neuro-Oncology
|
May 17, 2018
Virus vector-mediated genetic modification of brain tumor stromal cells after intravenous delivery
Adrienn Volak, Stanley G LeRoy, Jeya Shree Natasan, et al.
Molecular Therapy. Methods & Clinical Development
|
November 11, 2024
An <i>in vitro</i> and <i>in vivo</i> efficacy evaluation of gene therapy candidate SBT101 in mouse models of adrenomyeloneuropathy and in NHPs
Vidyullatha Vasireddy, Casey A Maguire, David W Anderson, et al.
Human Gene Therapy
|
June 28, 2023
An Engineered Adeno-Associated Virus Capsid Mediates Efficient Transduction of Pericytes and Smooth Muscle Cells of the Brain Vasculature
Servio H Ramirez, Jonathan F Hale, Siobhan McCarthy, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
June 7, 2024
In vivo selection in non-human primates identifies AAV capsids for on-target CSF delivery to spinal cord
Killian S Hanlon, Ming Cheng, Roberto Montoro Ferrer, et al.
Gene Therapy
|
June 10, 2025
Engineered AAV capsids mediate transduction of murine neurofibroma and sciatic nerve
Edwina Abou Haidar, Shilpa Prabhakar, Pike See Cheah, et al.
Molecular Therapy. Methods & Clinical Development
|
December 25, 2018
Gene Transfer with AAV9-PHP.B Rescues Hearing in a Mouse Model of Usher Syndrome 3A and Transduces Hair Cells in a Non-human Primate
Bence György, Elise J Meijer, Maryna V Ivanchenko, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
April 28, 2016
In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy
Sourav R Choudhury, Zachary Fitzpatrick, Anne F Harris, et al.
Nature Communications
|
October 2, 2019
High levels of AAV vector integration into CRISPR-induced DNA breaks
Killian S Hanlon, Benjamin P Kleinstiver, Sara P Garcia, et al.
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Search research articles
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Showing results (61-70 of 74) with videos related to
Sort By:
Page
of 8
Molecular Therapy. Nucleic Acids
|
July 25, 2013
BEAMing and Droplet Digital PCR Analysis of Mutant IDH1 mRNA in Glioma Patient Serum and Cerebrospinal Fluid Extracellular Vesicles
Walter W Chen, Leonora Balaj, Linda M Liau, et al.
Science Advances
|
February 1, 2021
Gene therapy for tuberous sclerosis complex type 2 in a mouse model by delivery of AAV9 encoding a condensed form of tuberin
Pike-See Cheah, Shilpa Prabhakar, David Yellen, et al.
Journal of Neuro-Oncology
|
May 17, 2018
Virus vector-mediated genetic modification of brain tumor stromal cells after intravenous delivery
Adrienn Volak, Stanley G LeRoy, Jeya Shree Natasan, et al.
Molecular Therapy. Methods & Clinical Development
|
November 11, 2024
An <i>in vitro</i> and <i>in vivo</i> efficacy evaluation of gene therapy candidate SBT101 in mouse models of adrenomyeloneuropathy and in NHPs
Vidyullatha Vasireddy, Casey A Maguire, David W Anderson, et al.
Human Gene Therapy
|
June 28, 2023
An Engineered Adeno-Associated Virus Capsid Mediates Efficient Transduction of Pericytes and Smooth Muscle Cells of the Brain Vasculature
Servio H Ramirez, Jonathan F Hale, Siobhan McCarthy, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
June 7, 2024
In vivo selection in non-human primates identifies AAV capsids for on-target CSF delivery to spinal cord
Killian S Hanlon, Ming Cheng, Roberto Montoro Ferrer, et al.
Gene Therapy
|
June 10, 2025
Engineered AAV capsids mediate transduction of murine neurofibroma and sciatic nerve
Edwina Abou Haidar, Shilpa Prabhakar, Pike See Cheah, et al.
Molecular Therapy. Methods & Clinical Development
|
December 25, 2018
Gene Transfer with AAV9-PHP.B Rescues Hearing in a Mouse Model of Usher Syndrome 3A and Transduces Hair Cells in a Non-human Primate
Bence György, Elise J Meijer, Maryna V Ivanchenko, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
April 28, 2016
In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy
Sourav R Choudhury, Zachary Fitzpatrick, Anne F Harris, et al.
Nature Communications
|
October 2, 2019
High levels of AAV vector integration into CRISPR-induced DNA breaks
Killian S Hanlon, Benjamin P Kleinstiver, Sara P Garcia, et al.
Page
of 8