Charles P. Venditti
Affiliations: | Biochemistry and Molecular Biology | The George Washington University, Washington, DC, United States |
Area:
Molecular Biology, Genetics, Medicine and SurgeryGoogle:
"Charles Venditti"Children
Sign in to add trainee| Randy J. Chandler | grad student | 2012 | The George Washington University |
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Publications
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| Baruteau J, Keshavan N, Venditti CP. (2024) Mission possible: Gene therapy for inherited metabolic diseases. Journal of Inherited Metabolic Disease. 47: 5-6 |
| Chandler RJ, Di Pasquale G, Choi EY, et al. (2023) Systemic gene therapy using an AAV44.9 vector rescues a neonatal lethal mouse model of propionic acidemia. Molecular Therapy. Methods & Clinical Development. 30: 181-190 |
| Chandler RJ, Venditti CP. (2023) Gene therapy for organic acidemias: Lessons learned from methylmalonic and propionic acidemia. Journal of Inherited Metabolic Disease |
| Manoli I, Gebremariam A, McCoy S, et al. (2023) Biomarkers to predict disease progression and therapeutic response in isolated methylmalonic acidemia (MMA). Journal of Inherited Metabolic Disease |
| Lomash RM, Shchelochkov O, Chandler RJ, et al. (2023) Successfully Navigating FDA Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The NIH Pave-GT Experience. Human Gene Therapy |
| Chandler RJ, Di Pasquale G, Sloan JL, et al. (2022) Systemic gene therapy for methylmalonic acidemia using the novel adeno-associated viral vector 44.9. Molecular Therapy. Methods & Clinical Development. 27: 61-72 |
| Venturoni LE, Chandler RJ, Liao J, et al. (2022) Growth advantage of corrected hepatocytes in a juvenile model of methylmalonic acidemia following liver directed adeno-associated viral mediated nuclease-free genome editing. Molecular Genetics and Metabolism. 137: 1-8 |
| Head PE, Myung S, Chen Y, et al. (2022) Aberrant methylmalonylation underlies methylmalonic acidemia and is attenuated by an engineered sirtuin. Science Translational Medicine. 14: eabn4772 |
| Schneller JL, Lee CM, Venturoni LE, et al. (2021) genome editing at the albumin locus to treat methylmalonic acidemia. Molecular Therapy. Methods & Clinical Development. 23: 619-632 |
| Ilyinskii PO, Michaud AM, Rizzo GL, et al. (2021) ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia. Molecular Therapy. Methods & Clinical Development. 22: 279-292 |