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Molecular Therapy : the Journal of the American Society of Gene Therapy|November 14, 2025
Non-invasive tracking of rAAV-mediated gene expression in the liver through a genetically encoded MRI reporter geneZinia Mohanta, Aruna Singh, Hernando Lopez-Bertoni, et al.Molecular Therapy. Methods & Clinical Development|October 3, 2022
Systemic gene therapy for methylmalonic acidemia using the novel adeno-associated viral vector 44.9Randy J Chandler, Giovanni Di Pasquale, Jennifer L Sloan, et al.American Journal of Medical Genetics|September 5, 2002
Omodysplasia: an affected mother and sonCharles P Venditti, Jennifer Farmer, Karen L Russell, et al.Behavior Genetics|March 1, 1994
DNA markers associated with high versus low IQ: the IQ Quantitative Trait Loci (QTL) ProjectR Plomin, G E McClearn, D L Smith, et al.JIMD Reports|February 20, 2020
High-dose hydroxocobalamin achieves biochemical correction and improvement of neuropsychiatric deficits in adults with late onset cobalamin C deficiencyTomoyasu Higashimoto, Alexander Y Kim, Jessica T Ogawa, et al.Human Molecular Genetics|November 1, 2016
Systemic AAV9 gene therapy improves the lifespan of mice with Niemann-Pick disease, type C1Randy J Chandler, Ian M Williams, Alana L Gibson, et al.Science Translational Medicine|May 25, 2022
Aberrant methylmalonylation underlies methylmalonic acidemia and is attenuated by an engineered sirtuinPamelaSara E Head, Sangho Myung, Yong Chen, et al.Molecular Biosystems|December 18, 2015
The proteome of methylmalonic acidemia (MMA): the elucidation of altered pathways in patient liversMarianna Caterino, Randy J Chandler, Jennifer L Sloan, et al.Journal of Inherited Metabolic Disease|June 26, 2014
A detailed analysis of methylmalonic acid kinetics during hemodialysis and after combined liver/kidney transplantation in a patient with mut (0) methylmalonic acidemiaHilary J Vernon, C John Sperati, Joshua D King, et al.Molecular Therapy. Methods & Clinical Development|September 25, 2023
Systemic gene therapy using an AAV44.9 vector rescues a neonatal lethal mouse model of propionic acidemiaRandy J Chandler, Giovanni Di Pasquale, Eun-Young Choi, et al.Pageof 18