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Gene Therapy Initiative

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Seeking a True Cure: Developing Gene Replacement and Editing Therapies to Address the Cause of NF1

GFF's Gene Therapy Initiative seeks to explore and develop gene-targeting therapeutic strategies for neurofibromatosis type 1 (NF1) that address the underlying cause of the disease. Targeting the mutated gene itself could yield a more effective, comprehensive response for a greater range of patients.
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Nothing Nonsensical About Nonsense Mutations: GTI Researchers Target One of the Most Common Types of NF1 Disease-Causing Genetic Mutations

While more than 1000 different mutations of the neurofibromatosis type 1 (NF1) gene have been identified to cause the disease, 1 in 5 are classified as "nonsense mutations". Several projects in GFF's Gene Therapy Initiative are exploring and identifying potential therapeutics to address NF1 nonsense mutations.
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Gilbert Family Foundation Announces $12 Million Gene Therapy Initiative to Address Underlying Cause of NF1

The Gilbert Family Foundation's seeks to eradicate neurofibromatosis type 1 (NF1). With our first initiative, we plan to accelerate the development of curative therapies that address the underlying genetic abnormalities in NF1 patients.
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