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Molecular Therapy. Methods & Clinical Development
|
December 13, 2021
<i>In vivo</i> genome editing at the albumin locus to treat methylmalonic acidemia
Jessica L Schneller, Ciaran M Lee, Leah E Venturoni, et al.
Biorxiv : the Preprint Server for Biology
|
June 19, 2024
Optimization of systemic AAV9 gene therapy in Niemann-Pick disease type C1 mice
Avani V Mylvara, Alana L Gibson, Tansy Gu, et al.
Journal of Inherited Metabolic Disease
|
October 27, 2004
Congenital cardiomyopathy and pulmonary hypertension: another fatal variant of cytochrome-c oxidase deficiency
C P Venditti, M C Harris, D Huff, et al.
The Journal of Clinical Investigation
|
January 22, 2015
Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy
Randy J Chandler, Matthew C LaFave, Gaurav K Varshney, et al.
Journal of Molecular Medicine (Berlin, Germany)
|
May 5, 2019
Macrophage derived TNFα promotes hepatic reprogramming to Warburg-like metabolism
Tatyana N Tarasenko, Maxim Jestin, Shingo Matsumoto, et al.
Molecular Genetics and Metabolism
|
August 5, 2008
Characterization of the null murine sodium/myo-inositol cotransporter 1 (Smit1 or Slc5a3) phenotype: myo-inositol rescue is independent of expression of its cognate mitochondrial ribosomal protein subunit 6 (Mrps6) gene and of phosphatidylinositol levels in neonatal brain
Roberto Buccafusca, Charles P Venditti, Lawrence C Kenyon, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
June 29, 2019
Chronic kidney disease in propionic acidemia
Oleg A Shchelochkov, Irini Manoli, Jennifer L Sloan, et al.
Human Molecular Genetics
|
July 6, 2025
Metabolic rerouting of valine and isoleucine oxidation increases survival in zebrafish models of disorders of propionyl-CoA metabolism
Sungkook Hong, Joel Pardo, PamelaSara E Head, et al.
Life Science Alliance
|
March 30, 2026
Optimization of systemic AAV9 gene therapy in Niemann-Pick disease, type C1 mice
Avani V Mylvara, Alana L Gibson, Tansy Gu, et al.
Molecular Genetics and Metabolism
|
January 9, 2025
Breath biopsy in inborn errors of metabolism: A proof-of-principle study in propionic acidemia
Oleg A Shchelochkov, Huw Davies, Robert P Mohney, et al.
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Search research articles
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Showing results (141-150 of 178) with videos related to
Sort By:
Page
of 18
Molecular Therapy. Methods & Clinical Development
|
December 13, 2021
<i>In vivo</i> genome editing at the albumin locus to treat methylmalonic acidemia
Jessica L Schneller, Ciaran M Lee, Leah E Venturoni, et al.
Biorxiv : the Preprint Server for Biology
|
June 19, 2024
Optimization of systemic AAV9 gene therapy in Niemann-Pick disease type C1 mice
Avani V Mylvara, Alana L Gibson, Tansy Gu, et al.
Journal of Inherited Metabolic Disease
|
October 27, 2004
Congenital cardiomyopathy and pulmonary hypertension: another fatal variant of cytochrome-c oxidase deficiency
C P Venditti, M C Harris, D Huff, et al.
The Journal of Clinical Investigation
|
January 22, 2015
Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy
Randy J Chandler, Matthew C LaFave, Gaurav K Varshney, et al.
Journal of Molecular Medicine (Berlin, Germany)
|
May 5, 2019
Macrophage derived TNFα promotes hepatic reprogramming to Warburg-like metabolism
Tatyana N Tarasenko, Maxim Jestin, Shingo Matsumoto, et al.
Molecular Genetics and Metabolism
|
August 5, 2008
Characterization of the null murine sodium/myo-inositol cotransporter 1 (Smit1 or Slc5a3) phenotype: myo-inositol rescue is independent of expression of its cognate mitochondrial ribosomal protein subunit 6 (Mrps6) gene and of phosphatidylinositol levels in neonatal brain
Roberto Buccafusca, Charles P Venditti, Lawrence C Kenyon, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
June 29, 2019
Chronic kidney disease in propionic acidemia
Oleg A Shchelochkov, Irini Manoli, Jennifer L Sloan, et al.
Human Molecular Genetics
|
July 6, 2025
Metabolic rerouting of valine and isoleucine oxidation increases survival in zebrafish models of disorders of propionyl-CoA metabolism
Sungkook Hong, Joel Pardo, PamelaSara E Head, et al.
Life Science Alliance
|
March 30, 2026
Optimization of systemic AAV9 gene therapy in Niemann-Pick disease, type C1 mice
Avani V Mylvara, Alana L Gibson, Tansy Gu, et al.
Molecular Genetics and Metabolism
|
January 9, 2025
Breath biopsy in inborn errors of metabolism: A proof-of-principle study in propionic acidemia
Oleg A Shchelochkov, Huw Davies, Robert P Mohney, et al.
Page
of 18