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Nature Biotechnology
|
November 17, 2020
Safety questions for AAV gene therapy
Charles P Venditti
Journal of Inherited Metabolic Disease
|
August 27, 2013
Noncompaction of the ventricular myocardium and hydrops fetalis in cobalamin C disease: Response to letter "Disappearance of congenital noncompaction in hereditary cobalamin-C-deficiency 2.5 years after birth" by J. Finsterer and Claudia Stöllberger, DOI 10.1007/s10545-013-9612-4
Pranoot Tanpaiboon, Charles P Venditti
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
October 29, 2009
Long-term rescue of a lethal murine model of methylmalonic acidemia using adeno-associated viral gene therapy
Randy J Chandler, Charles P Venditti
Human Gene Therapy
|
December 7, 2007
Adenovirus-mediated gene delivery rescues a neonatal lethal murine model of mut(0) methylmalonic acidemia
Randy J Chandler, Charles P Venditti
Journal of Inherited Metabolic Disease
|
August 2, 2023
Gene therapy for organic acidemias: Lessons learned from methylmalonic and propionic acidemia
Randy J Chandler, Charles P Venditti
Nature Metabolism
|
January 30, 2023
Anaplerosis in action
PamelaSara E Head, Charles P Venditti
Journal of Inherited Metabolic Disease
|
June 29, 2022
Treatment of metabolic disorders using genomic technologies: Lessons from methylmalonic acidemia
Leah E Venturoni, Charles P Venditti
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
May 3, 2011
A barrel of monkeys: scAAV8 gene therapy for hemophilia in nonhuman primates
Randy J Chandler, Charles P Venditti
Journal of Inherited Metabolic Disease
|
July 13, 2011
Combined methylmalonic acidemia and homocystinuria, cblC type. II. Complications, pathophysiology, and outcomes
Nuria Carrillo-Carrasco, Charles P Venditti
Molecular Genetics and Metabolism
|
October 11, 2012
Pre-clinical efficacy and dosing of an AAV8 vector expressing human methylmalonyl-CoA mutase in a murine model of methylmalonic acidemia (MMA)
Randy J Chandler, Charles P Venditti
Page
of 10
Search research articles
Search
Showing results (1-10 of 96) with videos related to
Sort By:
Page
of 10
Nature Biotechnology
|
November 17, 2020
Safety questions for AAV gene therapy
Charles P Venditti
Journal of Inherited Metabolic Disease
|
August 27, 2013
Noncompaction of the ventricular myocardium and hydrops fetalis in cobalamin C disease: Response to letter "Disappearance of congenital noncompaction in hereditary cobalamin-C-deficiency 2.5 years after birth" by J. Finsterer and Claudia Stöllberger, DOI 10.1007/s10545-013-9612-4
Pranoot Tanpaiboon, Charles P Venditti
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
October 29, 2009
Long-term rescue of a lethal murine model of methylmalonic acidemia using adeno-associated viral gene therapy
Randy J Chandler, Charles P Venditti
Human Gene Therapy
|
December 7, 2007
Adenovirus-mediated gene delivery rescues a neonatal lethal murine model of mut(0) methylmalonic acidemia
Randy J Chandler, Charles P Venditti
Journal of Inherited Metabolic Disease
|
August 2, 2023
Gene therapy for organic acidemias: Lessons learned from methylmalonic and propionic acidemia
Randy J Chandler, Charles P Venditti
Nature Metabolism
|
January 30, 2023
Anaplerosis in action
PamelaSara E Head, Charles P Venditti
Journal of Inherited Metabolic Disease
|
June 29, 2022
Treatment of metabolic disorders using genomic technologies: Lessons from methylmalonic acidemia
Leah E Venturoni, Charles P Venditti
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
May 3, 2011
A barrel of monkeys: scAAV8 gene therapy for hemophilia in nonhuman primates
Randy J Chandler, Charles P Venditti
Journal of Inherited Metabolic Disease
|
July 13, 2011
Combined methylmalonic acidemia and homocystinuria, cblC type. II. Complications, pathophysiology, and outcomes
Nuria Carrillo-Carrasco, Charles P Venditti
Molecular Genetics and Metabolism
|
October 11, 2012
Pre-clinical efficacy and dosing of an AAV8 vector expressing human methylmalonyl-CoA mutase in a murine model of methylmalonic acidemia (MMA)
Randy J Chandler, Charles P Venditti
Page
of 10