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Molecular Therapy : the Journal of the American Society of Gene Therapy
|
July 20, 2007
Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten disease
Mario A Cabrera-Salazar, Eric M Roskelley, Jie Bu, et al.
ACS Nano
|
April 4, 2025
Lipid Nanoparticles for <i>In Vivo</i> Lung Delivery of CRISPR-Cas9 Ribonucleoproteins Allow Gene Editing of Clinical Targets
Rebecca M Haley, Marshall S Padilla, Rakan D El-Mayta, et al.
Molecular Genetics and Metabolism
|
December 31, 2023
A novel iPSC model reveals selective vulnerability of neurons in multiple sulfatase deficiency
Vi Pham, Livia Sertori Finoti, Margaret M Cassidy, et al.
Nature Communications
|
May 6, 2023
Mapping PTBP2 binding in human brain identifies SYNGAP1 as a target for therapeutic splice switching
Jennine M Dawicki-McKenna, Alex J Felix, Elisa A Waxman, et al.
Nature Communications
|
June 25, 2025
AAV-based delivery of RNAi targeting ataxin-2 improves survival and pathology in TDP-43 mice
Defne A Amado, Ashley B Robbins, Katherine R Whiteman, et al.
The Journal of Neuroscience : the Official Journal of the Society for Neuroscience
|
February 3, 2006
Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis
Marco A Passini, James C Dodge, Jie Bu, et al.
American Journal of Human Genetics
|
April 2, 2026
Implications of the FDA's new plausible mechanism framework for the development of a personalized in vivo prime editing platform
Emily R Feierman, Madelynn N Whittaker, Aidan Quigley, et al.
Human Molecular Genetics
|
July 23, 2021
Immortalized striatal precursor neurons from Huntington's disease patient-derived iPS cells as a platform for target identification and screening for experimental therapeutics
Sergey S Akimov, Mali Jiang, Amanda J Kedaigle, et al.
Molecular Psychiatry
|
July 14, 2026
Translatable electrophysiological and behavioral abnormalities in a humanized model of SYNGAP1-disorder
Alex J Felix, Brandon L Brown, Nicolas Marotta, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
May 19, 2022
Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders
Beverly L Davidson, Guangping Gao, Elizabeth Berry-Kravis, et al.
Page
of 19
Search research articles
Search
Showing results (171-180 of 187) with videos related to
Sort By:
Page
of 19
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
July 20, 2007
Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten disease
Mario A Cabrera-Salazar, Eric M Roskelley, Jie Bu, et al.
ACS Nano
|
April 4, 2025
Lipid Nanoparticles for <i>In Vivo</i> Lung Delivery of CRISPR-Cas9 Ribonucleoproteins Allow Gene Editing of Clinical Targets
Rebecca M Haley, Marshall S Padilla, Rakan D El-Mayta, et al.
Molecular Genetics and Metabolism
|
December 31, 2023
A novel iPSC model reveals selective vulnerability of neurons in multiple sulfatase deficiency
Vi Pham, Livia Sertori Finoti, Margaret M Cassidy, et al.
Nature Communications
|
May 6, 2023
Mapping PTBP2 binding in human brain identifies SYNGAP1 as a target for therapeutic splice switching
Jennine M Dawicki-McKenna, Alex J Felix, Elisa A Waxman, et al.
Nature Communications
|
June 25, 2025
AAV-based delivery of RNAi targeting ataxin-2 improves survival and pathology in TDP-43 mice
Defne A Amado, Ashley B Robbins, Katherine R Whiteman, et al.
The Journal of Neuroscience : the Official Journal of the Society for Neuroscience
|
February 3, 2006
Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis
Marco A Passini, James C Dodge, Jie Bu, et al.
American Journal of Human Genetics
|
April 2, 2026
Implications of the FDA's new plausible mechanism framework for the development of a personalized in vivo prime editing platform
Emily R Feierman, Madelynn N Whittaker, Aidan Quigley, et al.
Human Molecular Genetics
|
July 23, 2021
Immortalized striatal precursor neurons from Huntington's disease patient-derived iPS cells as a platform for target identification and screening for experimental therapeutics
Sergey S Akimov, Mali Jiang, Amanda J Kedaigle, et al.
Molecular Psychiatry
|
July 14, 2026
Translatable electrophysiological and behavioral abnormalities in a humanized model of SYNGAP1-disorder
Alex J Felix, Brandon L Brown, Nicolas Marotta, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
May 19, 2022
Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders
Beverly L Davidson, Guangping Gao, Elizabeth Berry-Kravis, et al.
Page
of 19