The committee focuses on transplantation and gene therapy for nonmalignant disease, and has the following goals:

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• To promote and publicize the progress and outcomes of current PTCTC trials for nonmalignant disease

• To serve as an intellectual resource for development and implementation of future trials for nonmalignant disease

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To guide investigators seeking to receive PTCTC support for their trials 4 disease groups:

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1. Inborn Errors of Immunity,

2. Hemoglobinopathies

3. Metabolic/Neurologic

4. Bone Marrow Failure