Author

Elizabeth A. Ramsburg

Spark Therapeutics (United States)

Listed by name: these papers carry no ORCID for this author, so different people with the same name may be mixed together.

Biology Disciplines

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Neuroscience· 2 Bioengineering· 1

Signature Assays, Organisms & Methods

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Approach:experimental assay· 2 Organism:mouse· 2 Organism:non-human primate· 2 Assay:bulk RNA-seq· 1 Research Focus:drug response / drug discovery· 1 Organism:human· 1 Organism:rat· 1 Approach:statistical model· 1

Frequent Co-Authors

Esteban A. EngeliD· 2 shared Greglynn D Walton-Gibbs· 2 shared Yulia Dzhashiashvili· 2 shared Alec J. BattsiD· 1 shared Alexander B. RomanoviD· 1 shared Alexandra J Gilsrud· 1 shared Apeksha Khatiwada· 1 shared Ariel A. HippeniD· 1 shared

Activity by Year

2025-2026 · peak 2/yr

Co-Author Collaboration Network

10 top collaborators · 23 mutual co-author links

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2 shared papersNeuroscience

Esteban A. EngelORCID

Mutual co-authors in cluster

Author profile →

3 papers

Recent·Most cited

Restoring Parkin Function: An AAV Gene Therapy Approach for Early-Onset Parkinson's Disease

Sambuddha Basu, Tyler G. Demarest, Nicholas J. Gattone, Alexandra J Gilsrud, Brittany Wicks, Apeksha Khatiwada, Mohammad Nayal, Renee C. Gentzel, Daniel Cohen, Eric Wildon Kostuk, Derek P. Narendra, Pedro Gonzalez‐Alegre, Maria Grazia Biferi, Elizabeth A. Ramsburg

This paper investigates AAV-mediated PRKN gene replacement in Parkin-deficient models to restore mitophagy and demonstrate a feasible gene therapy approach for early-onset Parkinson's disease.

bioRxiv · Jul 13, 2026 · PreprintNeuroscience

Administration of barcoded AAV capsid library to the putamen of non-human primates identifies variants with efficient retrograde transport

Yulia Dzhashiashvili, Jodi L. McBride, Emily B. Fabyanic, Xin Huang, Brian M. Kelly, Greglynn D Walton-Gibbs, Vimala Vemireddi, Joan R. Wicks, Mohamad Nayal, Ariel A. Hippen, Zhenming Yu, Pichai Raman, Elizabeth A. Ramsburg, Marcus Davidsson, Esteban A. Engel, Tomas Björklund

The paper tests AAV capsid variants in non-human primates to identify those with improved retrograde transport properties.

Molecular Therapy · Dec 10, 2025Peer-reviewedNeuroscience

Portable transcranial therapeutic ultrasound enhances targeted gene delivery for Parkinson's disease: from rodent models to non-human primates

Alec J. Batts, Robin Ji, Sua Bae, Fotios N. Tsitsos, Sergio Jiménez-Gambín, Nancy Kwon, Samantha L. Gorman, Deny Tsakri, Rebecca Lynn Noel, Jonas Bendig, Daniella Amanda Jimenez, Melody R. DiBenedetto, Sofia A. Del Castillo, Filimon B. Keleta, James Caicedo, , Elisa E. Konofagou

Portable therapeutic ultrasound enhanced AAV9 delivery to the brain in mice and rhesus macaques compared to systemic injection alone.

bioRxiv · Mar 2, 2025 · PreprintBioengineering