This Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS), under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) federal grant program, supports the development of a novel adeno-associated viral vector 9 (AAV9) gene therapy treatment for neurofibromatosis type 2 (NF2). The goal is to deliver a healthy copy of the NF2 gene to address the underlying cause of this genetic disorder, which leads to tumors throughout the nervous system and severe neurological symptoms. The $699,559 award will enable Merlin Therapeutics Inc. to validate preliminary data, gather additional information on dose-response and safety, and confirm promoter activity and biodistribution of the lead gene therapy candidate in a larger animal species. This work aims to support the next steps towards translating this therapeutic approach to the clinic, with the long-term goal of providing an effective and safe treatment option for NF2 patients.
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