Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Filters

Randy J Chandler

Showing results (11-20 of 47) with videos related to

Pageof 5
Sort By:
Translational Science of Rare Diseases|November 18, 2016
Gene Therapy for Metabolic DiseasesRandy J Chandler, Charles P Venditti
Human Gene Therapy|March 16, 2017
Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal ModelsRandy J Chandler, Mark S Sands, Charles P Venditti
Journal of Inherited Metabolic Disease|July 13, 2011
Combined methylmalonic acidemia and homocystinuria, cblC type. I. Clinical presentations, diagnosis and managementNuria Carrillo-Carrasco, Randy J Chandler, Charles P Venditti
Human Gene Therapy|May 22, 2010
Liver-directed recombinant adeno-associated viral gene delivery rescues a lethal mouse model of methylmalonic acidemia and provides long-term phenotypic correctionNuria Carrillo-Carrasco, Randy J Chandler, Suma Chandrasekaran, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|February 25, 2016
Genotoxicity in Mice Following AAV Gene Delivery: A Safety Concern for Human Gene Therapy?Randy J Chandler, Matthew C LaFave, Gaurav K Varshney, et al.
Molecular Therapy. Methods & Clinical Development|June 25, 2021
Central nervous system-targeted adeno-associated virus gene therapy in methylmalonic acidemiaFrancis J May, PamelaSara E Head, Leah E Venturoni, et al.
Human Gene Therapy|January 25, 2023
Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy ExperienceRicha Madan Lomash, Oleg Shchelochkov, Randy J Chandler, et al.
FASEB Journal : Official Publication of the Federation of American Societies for Experimental Biology|December 18, 2008
Mitochondrial dysfunction in mut methylmalonic acidemiaRandy J Chandler, Patricia M Zerfas, Sara Shanske, et al.
Molecular Genetics and Metabolism|July 22, 2022
Growth advantage of corrected hepatocytes in a juvenile model of methylmalonic acidemia following liver directed adeno-associated viral mediated nuclease-free genome editingLeah E Venturoni, Randy J Chandler, Jing Liao, et al.
BMC Medical Genetics|May 2, 2007
Adenoviral-mediated correction of methylmalonyl-CoA mutase deficiency in murine fibroblasts and human hepatocytesRandy J Chandler, Matthew S Tsai, Kenneth Dorko, et al.
Pageof 5

Showing results (11-20 of 47) with videos related to

Sort By:
Pageof 5
Translational Science of Rare Diseases|November 18, 2016
Gene Therapy for Metabolic DiseasesRandy J Chandler, Charles P Venditti
Human Gene Therapy|March 16, 2017
Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal ModelsRandy J Chandler, Mark S Sands, Charles P Venditti
Journal of Inherited Metabolic Disease|July 13, 2011
Combined methylmalonic acidemia and homocystinuria, cblC type. I. Clinical presentations, diagnosis and managementNuria Carrillo-Carrasco, Randy J Chandler, Charles P Venditti
Human Gene Therapy|May 22, 2010
Liver-directed recombinant adeno-associated viral gene delivery rescues a lethal mouse model of methylmalonic acidemia and provides long-term phenotypic correctionNuria Carrillo-Carrasco, Randy J Chandler, Suma Chandrasekaran, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|February 25, 2016
Genotoxicity in Mice Following AAV Gene Delivery: A Safety Concern for Human Gene Therapy?Randy J Chandler, Matthew C LaFave, Gaurav K Varshney, et al.
Molecular Therapy. Methods & Clinical Development|June 25, 2021
Central nervous system-targeted adeno-associated virus gene therapy in methylmalonic acidemiaFrancis J May, PamelaSara E Head, Leah E Venturoni, et al.
Human Gene Therapy|January 25, 2023
Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy ExperienceRicha Madan Lomash, Oleg Shchelochkov, Randy J Chandler, et al.
FASEB Journal : Official Publication of the Federation of American Societies for Experimental Biology|December 18, 2008
Mitochondrial dysfunction in mut methylmalonic acidemiaRandy J Chandler, Patricia M Zerfas, Sara Shanske, et al.
Molecular Genetics and Metabolism|July 22, 2022
Growth advantage of corrected hepatocytes in a juvenile model of methylmalonic acidemia following liver directed adeno-associated viral mediated nuclease-free genome editingLeah E Venturoni, Randy J Chandler, Jing Liao, et al.
BMC Medical Genetics|May 2, 2007
Adenoviral-mediated correction of methylmalonyl-CoA mutase deficiency in murine fibroblasts and human hepatocytesRandy J Chandler, Matthew S Tsai, Kenneth Dorko, et al.
Pageof 5