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Gene Therapy|July 22, 2011
Gene therapy in a murine model of methylmalonic acidemia using rAAV9-mediated gene deliveryJ S Sénac, R J Chandler, J R Sysol, et al.
Nucleic Acids Research|August 1, 1998
Assembly of MMTV promoter minichromosomes with positioned nucleosomes precludes NF1 access but not restriction enzyme cleavageP Venditti, L Di Croce, M Kauer, et al.
Human Gene Therapy|February 27, 2025
Prevalence of Neutralizing Antibodies to AAV2 and AAV9 in Individuals with Niemann-Pick Disease, Type C1Avani V Mylvara, Cristin D Davidson, Derek Alexander, et al.
Frontiers in Physiology|April 3, 2026
Potential opportunities and challenges of utilizing brewer's spent grain in sustainable fish feeding within the circular economyE Geremia, E Buonocore, P Venditti, et al.
The Journal of Experimental Biology|February 17, 2006
Differential effects of experimental and cold-induced hyperthyroidism on factors inducing rat liver oxidative damageP Venditti, R Pamplona, V Ayala, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics|August 14, 2015
A critical reappraisal of dietary practices in methylmalonic acidemia raises concerns about the safety of medical foods. Part 1: isolated methylmalonic acidemiasIrini Manoli, Jennifer G Myles, Jennifer L Sloan, et al.
Archives of Physiology and Biochemistry|February 1, 1997
Action potential configuration in heart papillary muscles from female rats in different thyroid statesS Di Meo, P de Martino Rosaroll, P Venditti, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics|May 4, 2013
Renal growth in isolated methylmalonic acidemiaPaul S Kruszka, Irini Manoli, Jennifer L Sloan, et al.
Molecular Therapy. Methods & Clinical Development|June 25, 2021
Central nervous system-targeted adeno-associated virus gene therapy in methylmalonic acidemiaFrancis J May, PamelaSara E Head, Leah E Venturoni, et al.
Human Gene Therapy|January 25, 2023
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 ExperienceRicha Madan Lomash, Oleg Shchelochkov, Randy J Chandler, et al.
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