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Evaluating a next generation AAV capsid for targeted transduction of Schwann cells

We’ve identified a new adeno-associated virus serotype 9 (AAV9) capsid variant, called adeno-associated virus (AAV) Clone C, which transduces neurofibromas in a mouse model of …
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EV-based Nanocarriers for NF1 Therapies

This project will seek to develop novel delivery systems for therapeutic payloads to treat NF1. The goal is to develop next generation carriers based on …
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Novel vectors for gene therapies targeting primary human Schwann cells

The recent gene therapy clinical successes hinge on continuing progress in the development of AAV­ based gene delivery systems, driven primarily by advances in AAV …
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