Charles P. Venditti

National Human Genome Research Institute

Biochemistry, Genetics and Molecular BiologyMedicine

Pilot corpus only

This score is computed over theSindex pilot corpus and does not cover the full scientific literature. Scores are relative to papers we have ingested — papers, authors, and institutions outside the pilot are not represented. Methodology.

Top 100%percentile

0Author DataRank

Indexed papers

0in pilot corpus

datarank_citation_only_1hop_v6· scope data_onlyMethodology

Papers

Driven by 26 papers. Top paper: “Biomarkers to predict disease progression and therapeutic response in isolated methylmalonic acidemia”.

Biomarkers to predict disease progression and therapeutic response in isolated methylmalonic acidemia

Journal of Inherited Metabolic Disease(2023)10.1002/jimd.12636

N/A

1.1DataRank · unranked

42 citations

Abigael Gebremariam, Samantha McCoy, Alexandra Pass, Jack Gagné, Camryn Hall +11 more

Severity modeling of propionic acidemia using clinical and laboratory biomarkers

Genetics in Medicine(2021)10.1038/s41436-021-01173-2

N/A

0.846DataRank · unranked

23 citations

Irini Manoli, Paul Juneau, Jennifer L. Sloan, Susan Ferry, Jennifer Myles +15 more

1-13C-propionate breath testing as a surrogate endpoint to assess efficacy of liver-directed therapies in methylmalonic acidemia (MMA)

Genetics in Medicine(2021)10.1038/s41436-021-01143-8

N/A

0.688DataRank · unranked

16 citations

Alexandra Pass, Elizabeth A. Harrington, Jennifer L. Sloan, Jack Gagné, Samantha McCoy +16 more

Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors

Molecular Therapy — Methods & Clinical Development(2020)10.1016/j.omtm.2020.11.015

N/A

0.587DataRank · unranked

49 citations

Marialuisa Alliegro, Margherita Dell’Anno, Edoardo Nusco, John M. Cullen, Stephanie Smith +12 more

The Platform Vector Gene Therapies Project: Increasing the Efficiency of Adeno-Associated Virus Gene Therapy Clinical Trial Startup

Human Gene Therapy(2020)10.1089/hum.2020.259

N/A

0.529DataRank · unranked

33 citations

Elizabeth A. Ottinger, Deanna Portero, Richa Madan Lomash, Asaf Alimardanov, Pramod Terse +35 more

Lipodystrophy in methylmalonic acidemia associated with elevated FGF21 and abnormal methylmalonylation

JCI Insight(2024)10.1172/jci.insight.174097

N/A

0.345DataRank · unranked

6 citations

Justin R. Sysol, PamelaSara E. Head, Madeline Epping, Oksana Gavrilova, Melissa K. Crocker +22 more

Probing the functional consequence and clinical relevance of CD320 p.E88del, a variant in the transcobalamin receptor gene

American Journal of Medical Genetics Part A(2022)10.1002/ajmg.a.62627

N/A

0.330DataRank · unranked

8 citations

David Watkins, David J. Bernard, Yue Chen, Ningzheng Dong, Qingyu Wu +15 more

Breath biopsy in inborn errors of metabolism: A proof-of-principle study in propionic acidemia

Molecular Genetics and Metabolism(2024)10.1016/j.ymgme.2024.109005

N/A

0.165DataRank · unranked

2 citations

Huw Davies, Robert P. Mohney, Ace J. Hatch, Owen Birch, Susan Ferry +9 more

In vivo genome editing at the albumin locus to treat methylmalonic acidemia

Molecular Therapy — Methods & Clinical Development(2021)10.1016/j.omtm.2021.11.004

N/A

0DataRank · unranked

19 citations

Ciaran M. Lee, Leah E. Venturoni, Randy J. Chandler, Ang Li, Sangho Myung +6 more

ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia

Molecular Therapy — Methods & Clinical Development(2021)10.1016/j.omtm.2021.06.015

N/A

0DataRank · unranked

37 citations

Alicia Michaud, Gina L. Rizzo, Christopher Roy, Sheldon S. Leung, Stephanie L. Elkins +11 more

Central nervous system-targeted adeno-associated virus gene therapy in methylmalonic acidemia

Molecular Therapy — Methods & Clinical Development(2021)10.1016/j.omtm.2021.04.005

N/A

0DataRank · unranked

6 citations

PamelaSara E. Head, Leah E. Venturoni, Randy J. Chandler, Charles P. Venditti, Francis J. May

Aberrant methylmalonylation underlies methylmalonic acidemia and is attenuated by an engineered sirtuin

Science Translational Medicine(2022)10.1126/scitranslmed.abn4772

N/A

0DataRank · unranked

54 citations

Sangho Myung, Yong Chen, Jessica L. Schneller, Cindy Wang, Nicholas R. Duncan +7 more

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(2022)10.1016/j.ymgme.2022.06.011

N/A

0DataRank · unranked

14 citations

Randy J. Chandler, Jing Liao, Victoria Hoffmann, Nikhil Ramesh, Susana Gordo +3 more

The vitamin B12 processing enzyme, mmachc, is essential for zebrafish survival, growth and retinal morphology

Human Molecular Genetics(2020)10.1093/hmg/ddaa044

N/A

0DataRank · unranked

31 citations

Nathan P. Achilly, Madeline L. Arnold, Jerrel L. Catlett, Trevor Blake, Kevin Bishop +11 more

Systemic gene therapy for methylmalonic acidemia using the novel adeno-associated viral vector 44.9

Molecular Therapy — Methods & Clinical Development(2022)10.1016/j.omtm.2022.09.001

N/A

0DataRank · unranked

14 citations

Giovanni Di Pasquale, Jennifer L. Sloan, Samantha McCoy, Brandon T. Hubbard, Tina M. Kilts +4 more

Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe Disease

Molecular Therapy(2020)10.1016/j.ymthe.2020.12.031

N/A

0DataRank · unranked

50 citations

Christopher A. Miller, Lauren Shea, Xuntian Jiang, Miguel A. Guzmán, Randy J. Chandler +8 more

New insights into the pathophysiology of methylmalonic acidemia

Journal of Inherited Metabolic Disease(2023)10.1002/jimd.12617

N/A

0DataRank · unranked

33 citations

Jordan L. Meier, Charles P. Venditti, PamelaSara E. Head

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(2023)10.1089/hum.2022.232

N/A

0DataRank · unranked

10 citations

Oleg A. Shchelochkov, Randy J. Chandler, NIH PaVe-GT Team, Charles P. Venditti, Anne Pariser +38 more

Systemic gene therapy using an AAV44.9 vector rescues a neonatal lethal mouse model of propionic acidemia

Molecular Therapy — Methods & Clinical Development(2023)10.1016/j.omtm.2023.06.008

N/A

0DataRank · unranked

10 citations

Giovanni Di Pasquale, Eun-Young Choi, David K. Chang, Stephanie Smith, Jennifer L. Sloan +5 more

Anaplerosis in action

Nature Metabolism(2023)10.1038/s42255-022-00724-4

N/A

0DataRank · unranked

4 citations

Charles P. Venditti, PamelaSara E. Head

Intellectual disability and autism in propionic acidemia: a biomarker-behavioral investigation implicating dysregulated mitochondrial biology

Molecular Psychiatry(2024)10.1038/s41380-023-02385-5

N/A

0DataRank · unranked

12 citations

Cristan Farmer, Colby Chlebowski, Dee Adedipe, Susan Ferry, Irini Manoli +7 more

Promoterless, Nuclease‐Free Genome Editing Confers a Growth Advantage for Corrected Hepatocytes in Mice With Methylmalonic Acidemia

Hepatology(2020)10.1002/hep.31570

N/A

0DataRank · unranked

55 citations

Leah E. Venturoni, Jing Liao, Brandon T. Hubbard, Jessica L. Schneller, Victoria Hoffmann +9 more

Optimization of systemic AAV9 gene therapy in Niemann-Pick disease type C1 mice

bioRxiv (Cold Spring Harbor Laboratory)(2024)10.1101/2024.06.07.597901

N/A

0DataRank · unranked

2 citations

Alana L. Gibson, Tansy Gu, Cristin Davidson, Art Incao, Katerina Melnyk +7 more

Metabolic rerouting of valine and isoleucine oxidation increases survival in zebrafish models of disorders of propionyl-CoA metabolism

Human Molecular Genetics(2025)10.1093/hmg/ddaf100

N/A

0DataRank · unranked

3 citations

Joel Pardo, PamelaSara E. Head, Katharine T Ellis, Madeline L. Arnold, Nathan P. Achilly +8 more

Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann–Pick disease type C1 mice

Life Science Alliance(2021)10.26508/lsa.202101040

N/A

0DataRank · unranked

25 citations

Alana L. Gibson, Tansy Gu, Laura L. Baxter, Benjamin E. Deverman, Keith Beadle +10 more

Prevalence of Neutralizing Antibodies to AAV2 and AAV9 in Individuals with Niemann-Pick Disease, Type C1

Human Gene Therapy(2025)10.1089/hum.2024.233

N/A

0DataRank · unranked

1 citations

Cristin Davidson, Derek Alexander, Charles P. Venditti, Forbes D. Porter, Avani V. Mylvara