Miguel Sena-Esteves

4PUBLICATIONS
34CO-AUTHORS
Gene and molecular therapyGenetically modified animalsMedical infection agents (incl. prions)
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Publications (4)

|Sep 09, 2026
Intravenous gene therapy improves life span and clinical outcomes in a feline model of Sandhoff disease.

|Aug 15, 2025
Dual-vector rAAVrh8 gene therapy for GM2 gangliosidosis: a phase 1/2 trial.

Florian Eichler, Oguz I Cataltepe, Rrita Daci

|Mar 21, 2024
Expression of ALS-PFN1 impairs vesicular degradation in iPSC-derived microglia.

Salome Funes, Jonathan Jung, Del Hayden Gadd

|Feb 11, 2022
AAV gene therapy for Tay-Sachs disease.

Terence R Flotte, Oguz Cataltepe, Ajit Puri

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Frequent Collaborators

2 joint publications

Terence R Flotte

2 joint publications

Xuntian Jiang

2 joint publications

Allison M Keeler

2 joint publications

Douglas R Martin