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Sarah Wassmer

Massachusetts Eye and Ear Infirmary

12

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248

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MCOLN1 gene therapy corrects neurologic dysfunction in the mouse model of mucolipidosis IV.

2021 Human Molecular Genetics
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MCOLN1 gene-replacement therapy corrects neurologic dysfunction in the mouse model of mucolipidosis IV.

2020 bioRxiv
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Evaluating Efficiencies of Dual AAV Approaches for Retinal Targeting

2017 Frontiers in Neuroscience
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Evaluation of in silico reconstructed ancestral adeno-associated virus for gene augmentation therapy in a mouse model of LCA

2017 Investigative Ophthalmology & Visual Science
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80. Delayed Inflammatory Response to Intravitreal AAV Gene Transfer in Non-Human Primates

2016 Molecular Therapy
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594. Exosome-Associated AAV Enhances Retinal Transduction Following Intravitreal Injection

2016 Molecular Therapy
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269. Novel Synthetic AAV Efficiently Transduces Neurosensory Hair Cells in the Cochlea

2016 Molecular Therapy
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29. Novel Surgical Method for Intravitreal AAV Administration Overcomes Transduction Barriers in Non-Human Primates

2016 Molecular Therapy
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