Melissa A. Pratt

AAV9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities in a Mouse Model of CLN8-Batten Disease

Tyler B. Johnson, Katherine A. White, Jon J. Brudvig et al. · Molecular Therapy · 2020 · 41 citations · Full text

Early postnatal administration of an AAV9 gene therapy is safe and efficacious in CLN3 disease

Tyler B. Johnson, Jon J. Brudvig, Shibi Likhite et al. · Frontiers in Genetics · 2023 · 20 citations · Full text

Cln3 Gene, Immunology, Pathology +18

Protracted CLN3 Batten disease in mice that genetically model an exon-skipping therapeutic approach

Jessica L. Centa, Matthew P. Stratton, Melissa A. Pratt et al. · Molecular Therapy — Nucleic Acids · 2023 · 20 citations · Full text

Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice

Katherine A. White, Hemanth R. Nelvagal, Timothy A. Poole et al. · Molecular Therapy — Methods & Clinical Development · 2021 · 18 citations · Full text

Glycerophosphoinositol is Elevated in Blood Samples From <i>CLN3</i>^Δex7-8 pigs, <i>Cln3</i>^Δex7-8 Mice, and CLN3-Affected Individuals

Jon J. Brudvig, Vicki J. Swier, Tyler B. Johnson et al. · Biomarker Insights · 2022 · 16 citations · Full text