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Showing results (151-160 of 178) with videos related to

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Hepatology (Baltimore, Md.)|September 25, 2020
Promoterless, Nuclease-Free Genome Editing Confers a Growth Advantage for Corrected Hepatocytes in Mice With Methylmalonic AcidemiaRandy J Chandler, Leah E Venturoni, Jing Liao, et al.
Journal of Inherited Metabolic Disease|May 27, 2023
Biomarkers to predict disease progression and therapeutic response in isolated methylmalonic acidemiaIrini Manoli, Abigael Gebremariam, Samantha McCoy, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|January 3, 2021
Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe DiseaseYedda Li, Christopher A Miller, Lauren K Shea, et al.
Blood Cells, Molecules & Diseases|January 1, 1996
Mutation analysis in hereditary hemochromatosisE Beutler, T Gelbart, C West, et al.
Molecular Therapy. Methods & Clinical Development|January 1, 2020
Pathogenesis of Hepatic Tumors following Gene Therapy in Murine and Canine Models of Glycogen Storage DiseaseHye-Ri Kang, Monika Gjorgjieva, Stephanie N Smith, et al.
Human Gene Therapy|September 30, 2020
The Platform Vector Gene Therapies Project: Increasing the Efficiency of Adeno-Associated Virus Gene Therapy Clinical Trial StartupPhilip J Brooks, Elizabeth A Ottinger, Deanna Portero, et al.
Life Science Alliance|August 19, 2021
Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann-Pick disease type C1 miceCristin D Davidson, Alana L Gibson, Tansy Gu, et al.
Human Molecular Genetics|March 19, 2020
The vitamin B12 processing enzyme, mmachc, is essential for zebrafish survival, growth and retinal morphologyJennifer L Sloan, Nathan P Achilly, Madeline L Arnold, et al.
Molecular Metabolism|October 1, 2016
Defects in muscle branched-chain amino acid oxidation contribute to impaired lipid metabolismCarles Lerin, Allison B Goldfine, Tanner Boes, et al.
Molecular Therapy. Methods & Clinical Development|January 21, 2021
Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectorsRita Ferla, Marialuisa Alliegro, Margherita Dell'Anno, et al.
Pageof 18

Showing results (151-160 of 178) with videos related to

Sort By:
Pageof 18
Hepatology (Baltimore, Md.)|September 25, 2020
Promoterless, Nuclease-Free Genome Editing Confers a Growth Advantage for Corrected Hepatocytes in Mice With Methylmalonic AcidemiaRandy J Chandler, Leah E Venturoni, Jing Liao, et al.
Journal of Inherited Metabolic Disease|May 27, 2023
Biomarkers to predict disease progression and therapeutic response in isolated methylmalonic acidemiaIrini Manoli, Abigael Gebremariam, Samantha McCoy, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|January 3, 2021
Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe DiseaseYedda Li, Christopher A Miller, Lauren K Shea, et al.
Blood Cells, Molecules & Diseases|January 1, 1996
Mutation analysis in hereditary hemochromatosisE Beutler, T Gelbart, C West, et al.
Molecular Therapy. Methods & Clinical Development|January 1, 2020
Pathogenesis of Hepatic Tumors following Gene Therapy in Murine and Canine Models of Glycogen Storage DiseaseHye-Ri Kang, Monika Gjorgjieva, Stephanie N Smith, et al.
Human Gene Therapy|September 30, 2020
The Platform Vector Gene Therapies Project: Increasing the Efficiency of Adeno-Associated Virus Gene Therapy Clinical Trial StartupPhilip J Brooks, Elizabeth A Ottinger, Deanna Portero, et al.
Life Science Alliance|August 19, 2021
Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann-Pick disease type C1 miceCristin D Davidson, Alana L Gibson, Tansy Gu, et al.
Human Molecular Genetics|March 19, 2020
The vitamin B12 processing enzyme, mmachc, is essential for zebrafish survival, growth and retinal morphologyJennifer L Sloan, Nathan P Achilly, Madeline L Arnold, et al.
Molecular Metabolism|October 1, 2016
Defects in muscle branched-chain amino acid oxidation contribute to impaired lipid metabolismCarles Lerin, Allison B Goldfine, Tanner Boes, et al.
Molecular Therapy. Methods & Clinical Development|January 21, 2021
Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectorsRita Ferla, Marialuisa Alliegro, Margherita Dell'Anno, et al.
Pageof 18