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Blood
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January 18, 2003
Neonatal or hepatocyte growth factor-potentiated adult gene therapy with a retroviral vector results in therapeutic levels of canine factor IX for hemophilia B
Lingfei Xu, Cuihua Gao, Mark S Sands, et al.
Plos One
|
August 23, 2007
Development of sensory, motor and behavioral deficits in the murine model of Sanfilippo syndrome type B
Coy D Heldermon, Anne K Hennig, Kevin K Ohlemiller, et al.
Scientific Reports
|
July 5, 2022
Effects of chronic cannabidiol in a mouse model of naturally occurring neuroinflammation, neurodegeneration, and spontaneous seizures
Joshua T Dearborn, Hemanth R Nelvagal, Nicholas R Rensing, et al.
Journal of Neuroscience Research
|
September 18, 2016
Hematopoietic Stem cell transplantation and lentiviral vector-based gene therapy for Krabbe's disease: Present convictions and future prospects
Peirong Hu, Yedda Li, Nana Nikolaishvili-Feinberg, et al.
Molecular Genetics and Metabolism
|
April 11, 2021
Biochemical evaluation of intracerebroventricular rhNAGLU-IGF2 enzyme replacement therapy in neonatal mice with Sanfilippo B syndrome
Shih-Hsin Kan, Ibrahim Elsharkawi, Steven Q Le, et al.
Human Gene Therapy
|
May 21, 2011
Lentiviral-mediated gene transfer to the sheep brain: implications for gene therapy in Batten disease
Kathryn S Linterman, David N Palmer, Graham W Kay, et al.
Journal of Inherited Metabolic Disease
|
June 11, 2026
Validating a Human Cell Model of Null Galactosylceramidase (GALC) Enzyme Activity That Recapitulates Krabbe Disease
Rodrigo T Starosta, Nazia Khatoon, Marie S Roberts, et al.
International Journal of Molecular Sciences
|
April 14, 2026
Systemic AAV9 Gene Therapy Mitigates Neuromuscular Junction Degeneration and Muscle Atrophy in a Mouse Model of CLN1 Disease
Ewa A Ziółkowska, Albina Jablonka-Shariff, Letitia L Williams, et al.
The Journal of Clinical Investigation
|
April 27, 2023
Gene therapy ameliorates spontaneous seizures associated with cortical neuron loss in a Cln2R207X mouse model
Keigo Takahashi, Elizabeth M Eultgen, Sophie H Wang, et al.
Molecular Genetics and Metabolism
|
June 21, 2025
Secondary accumulation of lyso-platelet activating factors in lysosomal storage diseases
Pamela Kell, Sonali Mishra, Heather L Gray-Edwards, et al.
Page
of 11
Search research articles
Search
Showing results (71-80 of 105) with videos related to
Sort By:
Page
of 11
Blood
|
January 18, 2003
Neonatal or hepatocyte growth factor-potentiated adult gene therapy with a retroviral vector results in therapeutic levels of canine factor IX for hemophilia B
Lingfei Xu, Cuihua Gao, Mark S Sands, et al.
Plos One
|
August 23, 2007
Development of sensory, motor and behavioral deficits in the murine model of Sanfilippo syndrome type B
Coy D Heldermon, Anne K Hennig, Kevin K Ohlemiller, et al.
Scientific Reports
|
July 5, 2022
Effects of chronic cannabidiol in a mouse model of naturally occurring neuroinflammation, neurodegeneration, and spontaneous seizures
Joshua T Dearborn, Hemanth R Nelvagal, Nicholas R Rensing, et al.
Journal of Neuroscience Research
|
September 18, 2016
Hematopoietic Stem cell transplantation and lentiviral vector-based gene therapy for Krabbe's disease: Present convictions and future prospects
Peirong Hu, Yedda Li, Nana Nikolaishvili-Feinberg, et al.
Molecular Genetics and Metabolism
|
April 11, 2021
Biochemical evaluation of intracerebroventricular rhNAGLU-IGF2 enzyme replacement therapy in neonatal mice with Sanfilippo B syndrome
Shih-Hsin Kan, Ibrahim Elsharkawi, Steven Q Le, et al.
Human Gene Therapy
|
May 21, 2011
Lentiviral-mediated gene transfer to the sheep brain: implications for gene therapy in Batten disease
Kathryn S Linterman, David N Palmer, Graham W Kay, et al.
Journal of Inherited Metabolic Disease
|
June 11, 2026
Validating a Human Cell Model of Null Galactosylceramidase (GALC) Enzyme Activity That Recapitulates Krabbe Disease
Rodrigo T Starosta, Nazia Khatoon, Marie S Roberts, et al.
International Journal of Molecular Sciences
|
April 14, 2026
Systemic AAV9 Gene Therapy Mitigates Neuromuscular Junction Degeneration and Muscle Atrophy in a Mouse Model of CLN1 Disease
Ewa A Ziółkowska, Albina Jablonka-Shariff, Letitia L Williams, et al.
The Journal of Clinical Investigation
|
April 27, 2023
Gene therapy ameliorates spontaneous seizures associated with cortical neuron loss in a Cln2R207X mouse model
Keigo Takahashi, Elizabeth M Eultgen, Sophie H Wang, et al.
Molecular Genetics and Metabolism
|
June 21, 2025
Secondary accumulation of lyso-platelet activating factors in lysosomal storage diseases
Pamela Kell, Sonali Mishra, Heather L Gray-Edwards, et al.
Page
of 11