Charles P Venditti

Publication Activity (10 Years)

Years Active: 2019-2024
Publications (10 Years): 23

Top Topics

Chronic Kidney Disease

Top Venues

Molecular psychiatry

Journal of inherited metabolic disease

Genetics in medicine : official journal of the American College of Medical Genetics

Human gene therapy

This page only lists publications with an associated author ORCID identifier.

Publications

Oleg A Shchelochkov, Cristan A Farmer, Colby Chlebowski, Dee Adedipe, Susan Ferry, Irini Manoli, Alexandra Pass, Samantha McCoy, Carol Van Ryzin, Jennifer Sloan, Audrey Thurm, Charles P Venditti
Intellectual disability and autism in propionic acidemia: a biomarker-behavioral investigation implicating dysregulated mitochondrial biology. Molecular psychiatry 29 (4) (2024)
Oleg A Shchelochkov, Cristan A Farmer, Colby Chlebowski, Dee Adedipe, Susan Ferry, Irini Manoli, Alexandra Pass, Samantha McCoy, Carol Van Ryzin, Jennifer Sloan, Audrey Thurm, Charles P Venditti
Intellectual disability and autism in propionic acidemia: a biomarker-behavioral investigation implicating dysregulated mitochondrial biology. Molecular psychiatry (2024)
Oleg A Shchelochkov, Cristan A Farmer, Colby Chlebowski, Dee Adedipe, Susan Ferry, Irini Manoli, Alexandra Pass, Samantha McCoy, Carol Van Ryzin, Jennifer Sloan, Audrey Thurm, Charles P Venditti
Intellectual disability and autism in propionic acidemia: a biomarker-behavioral investigation implicating dysregulated mitochondrial biology. Molecular psychiatry 29 (4) (2024)
Oleg A Shchelochkov, Cristan A Farmer, Colby Chlebowski, Dee Adedipe, Susan Ferry, Irini Manoli, Alexandra Pass, Samantha McCoy, Carol Van Ryzin, Jennifer Sloan, Audrey Thurm, Charles P Venditti
Intellectual disability and autism in propionic acidemia: a biomarker-behavioral investigation implicating dysregulated mitochondrial biology. Molecular psychiatry 29 (4) (2024)
Avani V Mylvara, Alana L Gibson, Tansy Gu, Cristin D Davidson, Art A Incao, Katerina Melnyk, Dominick Pierre-Jacques, Stephanie M Cologna, Charles P Venditti, Forbes D Porter, William J Pavan
Optimization of systemic AAV9 gene therapy in Niemann-Pick disease type C1 mice. bioRxiv : the preprint server for biology (2024)
Oleg A Shchelochkov, Cristan A Farmer, Colby Chlebowski, Dee Adedipe, Susan Ferry, Irini Manoli, Alexandra Pass, Samantha McCoy, Carol Van Ryzin, Jennifer Sloan, Audrey Thurm, Charles P Venditti
Intellectual disability and autism in propionic acidemia: a biomarker-behavioral investigation implicating dysregulated mitochondrial biology. Molecular psychiatry 29 (4) (2024)
Richa Madan Lomash, Oleg Shchelochkov, Randy J Chandler, Charles P Venditti, Anne R Pariser, Elizabeth A Ottinger, null null
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 Experience. Human gene therapy 34 (5-6) (2023)
Randy J Chandler, Charles P Venditti
Gene Therapy for Organic Acidemias: Lessons Learned from Methylmalonic and Propionic Acidemia. Journal of inherited metabolic disease (2023)
PamelaSara E Head, Jordan L Meier, Charles P Venditti
New insights into the pathophysiology of methylmalonic acidemia. Journal of inherited metabolic disease (2023)
PamelaSara E Head, Charles P Venditti
Anaplerosis in action. Nature metabolism 5 (1) (2023)
Irini Manoli, Abigael Gebremariam, Samantha McCoy, Alexandra R Pass, Jack Gagné, Camryn Hall, Susan Ferry, Carol Van Ryzin, Jennifer L Sloan, Elisa Sacchetti, Giulio Catesini, Cristiano Rizzo, Diego Martinelli, Marco Spada, Carlo Dionisi-Vici, Charles P Venditti
Biomarkers to predict disease progression and therapeutic response in isolated methylmalonic acidemia (MMA). Journal of inherited metabolic disease (2023)
Richa Madan Lomash, Oleg Shchelochkov, Randy J Chandler, Charles P Venditti, Anne R Pariser, Elizabeth A Ottinger, null null
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 Experience. Human gene therapy 34 (5-6) (2023)
PamelaSara E Head, Charles P Venditti
Anaplerosis in action. Nature metabolism 5 (1) (2023)
Dulika Sumathipala, Petter Strømme, Zohreh Fattahi, Torben Lüders, Ying Sheng, Kimia Kahrizi, Ingunn Holm Einarsen, Jennifer L Sloan, Hossein Najmabadi, Lambert van den Heuvel, Ron A Wevers, Sergio Guerrero-Castillo, Lars Mørkrid, Vassili Valayannopoulos, Paul Hoff Backe, Charles P Venditti, Clara D van Karnebeek, Hilde Nilsen, Eirik Frengen, Doriana Misceo
ZBTB11 dysfunction: spectrum of brain abnormalities, biochemical signature and cellular consequences. Brain : a journal of neurology 145 (7) (2022)
PamelaSara E Head, Sangho Myung, Yong Chen, Jessica L Schneller, Cindy Wang, Nicholas R Duncan, Pauline R Hoffman, David Chang, Abigael Gebremariam, Marjan Gucek, Irini Manoli, Charles P Venditti
Aberrant methylmalonylation underlies methylmalonic acidemia and is attenuated by an engineered sirtuin. Science translational medicine 14 (646) (2022)
Leah E Venturoni, Charles P Venditti
Treatment of metabolic disorders using genomic technologies: Lessons from methylmalonic acidemia. Journal of inherited metabolic disease 45 (5) (2022)
Cristin D Davidson, Alana L Gibson, Tansy Gu, Laura L Baxter, Benjamin E Deverman, Keith Beadle, Arturo A Incao, Jorge L Rodriguez-Gil, Hideji Fujiwara, Xuntian Jiang, Randy J Chandler, Daniel S Ory, Viviana Gradinaru, Charles P Venditti, William J Pavan
Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann-Pick disease type C1 mice. Life science alliance 4 (10) (2021)
Charles P Venditti
Safety questions for AAV gene therapy. Nature biotechnology 39 (1) (2021)
Randy J Chandler, Leah E Venturoni, Jing Liao, Brandon T Hubbard, Jessica L Schneller, Victoria Hoffmann, Susana Gordo, Shengwen Zang, Chih-Wei Ko, Nelson Chau, Kyle Chiang, Mark A Kay, Adi Barzel, Charles P Venditti
Promoterless, Nuclease-Free Genome Editing Confers a Growth Advantage for Corrected Hepatocytes in Mice With Methylmalonic Acidemia. Hepatology (Baltimore, Md.) 73 (6) (2021)
Oleg A Shchelochkov, Irini Manoli, Paul Juneau, Jennifer L Sloan, Susan Ferry, Jennifer Myles, Megan Schoenfeld, Alexandra Pass, Samantha McCoy, Carol Van Ryzin, Olivia Wenger, Mark Levin, Wadih Zein, Laryssa Huryn, Joseph Snow, Colby Chlebowski, Audrey Thurm, Jeffrey B Kopp, Kong Y Chen, Charles P Venditti
Severity modeling of propionic acidemia using clinical and laboratory biomarkers. Genetics in medicine : official journal of the American College of Medical Genetics 23 (8) (2021)
Irini Manoli, Alexandra R Pass, Elizabeth A Harrington, Jennifer L Sloan, Jack Gagné, Samantha McCoy, Sarah L Bell, Jacob D Hattenbach, Brooks P Leitner, Courtney J Duckworth, Laura A Fletcher, Thomas M Cassimatis, Carolina I Galarreta, Audrey Thurm, Joseph Snow, Carol Van Ryzin, Susan Ferry, Nicholas Ah Mew, Oleg A Shchelochkov, Kong Y Chen, Charles P Venditti
1-13C-propionate breath testing as a surrogate endpoint to assess efficacy of liver-directed therapies in methylmalonic acidemia (MMA). Genetics in medicine : official journal of the American College of Medical Genetics 23 (8) (2021)
KowsalyaDevi Pavuluri, Irini Manoli, Alexandra Pass, Yuguo Li, Hilary J Vernon, Charles P Venditti, Michael T McMahon
Noninvasive monitoring of chronic kidney disease using pH and perfusion imaging. Science advances 5 (8) (2019)
Oleg A Shchelochkov, Irini Manoli, Jennifer L Sloan, Susan Ferry, Alexandra Pass, Carol Van Ryzin, Jennifer Myles, Megan Schoenfeld, Peter McGuire, Douglas R Rosing, Mark D Levin, Jeffrey B Kopp, Charles P Venditti
Chronic kidney disease in propionic acidemia. Genetics in medicine : official journal of the American College of Medical Genetics 21 (12) (2019)