Charles P. Venditti

Medical Genetics

9000 Rockville Pike National Institutes Of Health
Building 49 Rm 4a18
Bethesda, MD 20892

Last Updated: 09/26/2026

Save publications for later

Sign Up

Not sure about your diagnosis?

Check Your Symptoms

Found 84 publications

Adeno-Associated Virus Gene Therapy Translation: Lessons from Early Regulatory Meetings.

Adeno-Associated Virus Gene Therapy Translation: Lessons from Early Regulatory Meetings.

Journal: Human gene therapy

Published: June 19, 2026

Cobalamin-Related Remethylation Disorders: Pregnancy Outcomes and Prenatal Treatment-New Cases and a Literature Study.

Cobalamin-Related Remethylation Disorders: Pregnancy Outcomes and Prenatal Treatment-New Cases and a Literature Study.

Journal: Journal of inherited metabolic disease

Published: January 16, 2026

Non-invasive tracking of rAAV-mediated gene expression in the liver through a genetically encoded MRI reporter gene.

Non-invasive tracking of rAAV-mediated gene expression in the liver through a genetically encoded MRI reporter gene.

Journal: Molecular therapy : the journal of the American Society of Gene Therapy

Published: May 09, 2025

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

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

Journal: Human molecular genetics

Published: March 17, 2025

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

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

Journal: Human gene therapy

Published: February 27, 2025

Adeno-Associated Virus Gene Therapy Development: Early Planning and Regulatory Considerations to Advance the Platform Vector Gene Therapy Program.

Adeno-Associated Virus Gene Therapy Development: Early Planning and Regulatory Considerations to Advance the Platform Vector Gene Therapy Program.

Journal: Human gene therapy

Published: February 20, 2025

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

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

Journal: Molecular genetics and metabolism

Published: October 09, 2024

Dissecting the effect of mitochondrial BCAT inhibition in methylmalonic acidemia.

Dissecting the effect of mitochondrial BCAT inhibition in methylmalonic acidemia.

Journal: JCI insight

Published: October 04, 2024

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

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

Journal: bioRxiv : the preprint server for biology

Published: June 19, 2024

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

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

Journal: Life science alliance

Published: June 06, 2024

Severe heart failure in a unique case of cobalamin-C-deficiency resolved with LVAD implantation and subsequent heart transplantation.

Severe heart failure in a unique case of cobalamin-C-deficiency resolved with LVAD implantation and subsequent heart transplantation.

Journal: Molecular genetics and metabolism reports

Published: March 23, 2024

Mission possible: Gene therapy for inherited metabolic diseases.

Mission possible: Gene therapy for inherited metabolic diseases.

Journal: Journal of inherited metabolic disease

Published: December 20, 2023

Showing 1-12 of 84

Last Updated: 09/26/2026