Gene Therapy Initiative

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Gene Therapy Initiative Annual Meeting Highlights

We recently held our Annual Gene Therapy Initiative, highlighting the findings of our gene therapy research team as they make progress with preclinical model systems. Learn more about our findings and how we are accelerating a cure for NF.
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Exciting Updates to Gene Therapy Initiative Presented at Annual Meeting

Recently, we brought together nine research teams for our first-ever in-person Gene Therapy Initiative Annual Meeting.
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Gilbert Family Foundation Furthers Commitment to Curing Neurofibromatosis (NF), Invests Additional $18 Million Toward Gene Therapy Initiative

The Gilbert Family Foundation is excited to announce a new round of three-year grants in the Gene Therapy Initiative to support the development of curative therapies that address the underlying cause of NF1.
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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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