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Genes
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February 27, 2026
RNAi-Induced Expression of Paternal UBE3A
Hye Ri Kang, Violeta Zaric, Volodymyr Rybalchenko, et al.
Molecular Therapy. Methods & Clinical Development
|
September 2, 2025
Improved AAV9-based gene therapy design for <i>SURF1</i>-related Leigh syndrome with minimal toxicity
Qinglan Ling, Matthew Rioux, Harrison Higgs, et al.
Genes
|
January 23, 2024
The Efficacy of a Human-Ready mini<i>MECP2</i> Gene Therapy in a Pre-Clinical Model of Rett Syndrome
Chanchal Sadhu, Christopher Lyons, Jiyoung Oh, et al.
Biological Psychiatry Global Open Science
|
October 17, 2025
Exploration of a DDX3X Gene Supplementation Therapy Including Expanded Characterization and Novel Findings of Sleep Disturbances in <i>Ddx3x</i> Haploinsufficient Mice
Andrea Boitnott, Anjala Jiji, Erik J Plautz, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
April 14, 2011
Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates
Steven J Gray, Valerie Matagne, Lavanya Bachaboina, et al.
The Journal of Clinical Investigation
|
November 26, 2024
AAV9/SLC6A1 gene therapy rescues abnormal EEG patterns and cognitive behavioral deficiencies in Slc6a1-/- mice
Weirui Guo, Matthew Rioux, Frances Shaffo, et al.
Annals of Clinical and Translational Neurology
|
December 20, 2018
Optical coherence tomography features in brothers with aspartylglucosaminuria
Kimberly Goodspeed, Lana Harder, Samuel Hughes, et al.
Journal of Virology
|
February 13, 2009
Reducing the risk of adeno-associated virus (AAV) vector mobilization with AAV type 5 vectors
F Curtis Hewitt, Chengwen Li, Steven J Gray, et al.
Journal of Neuroscience Research
|
September 18, 2016
Intrathecal administration of AAV/GALC vectors in 10-11-day-old twitcher mice improves survival and is enhanced by bone marrow transplant
Subha Karumuthil-Melethil, Michael S Marshall, Clifford Heindel, et al.
Genes
|
November 27, 2024
RNAi Knockdown of <i>EHMT2</i> in Maternal Expression of Prader-Willi Syndrome Genes
Violeta Zaric, Hye Ri Kang, Volodymyr Rybalchenko, et al.
Page
of 11
Search research articles
Search
Showing results (31-40 of 106) with videos related to
Sort By:
Page
of 11
Genes
|
February 27, 2026
RNAi-Induced Expression of Paternal UBE3A
Hye Ri Kang, Violeta Zaric, Volodymyr Rybalchenko, et al.
Molecular Therapy. Methods & Clinical Development
|
September 2, 2025
Improved AAV9-based gene therapy design for <i>SURF1</i>-related Leigh syndrome with minimal toxicity
Qinglan Ling, Matthew Rioux, Harrison Higgs, et al.
Genes
|
January 23, 2024
The Efficacy of a Human-Ready mini<i>MECP2</i> Gene Therapy in a Pre-Clinical Model of Rett Syndrome
Chanchal Sadhu, Christopher Lyons, Jiyoung Oh, et al.
Biological Psychiatry Global Open Science
|
October 17, 2025
Exploration of a DDX3X Gene Supplementation Therapy Including Expanded Characterization and Novel Findings of Sleep Disturbances in <i>Ddx3x</i> Haploinsufficient Mice
Andrea Boitnott, Anjala Jiji, Erik J Plautz, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
April 14, 2011
Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates
Steven J Gray, Valerie Matagne, Lavanya Bachaboina, et al.
The Journal of Clinical Investigation
|
November 26, 2024
AAV9/SLC6A1 gene therapy rescues abnormal EEG patterns and cognitive behavioral deficiencies in Slc6a1-/- mice
Weirui Guo, Matthew Rioux, Frances Shaffo, et al.
Annals of Clinical and Translational Neurology
|
December 20, 2018
Optical coherence tomography features in brothers with aspartylglucosaminuria
Kimberly Goodspeed, Lana Harder, Samuel Hughes, et al.
Journal of Virology
|
February 13, 2009
Reducing the risk of adeno-associated virus (AAV) vector mobilization with AAV type 5 vectors
F Curtis Hewitt, Chengwen Li, Steven J Gray, et al.
Journal of Neuroscience Research
|
September 18, 2016
Intrathecal administration of AAV/GALC vectors in 10-11-day-old twitcher mice improves survival and is enhanced by bone marrow transplant
Subha Karumuthil-Melethil, Michael S Marshall, Clifford Heindel, et al.
Genes
|
November 27, 2024
RNAi Knockdown of <i>EHMT2</i> in Maternal Expression of Prader-Willi Syndrome Genes
Violeta Zaric, Hye Ri Kang, Volodymyr Rybalchenko, et al.
Page
of 11