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Gene Therapy and Repair

Program in Gene and Cell Therapy

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The Program for Cell and Gene Therapy (PCGT), created in 2014 by Seattle Children’s Research Institute, is co-directed by Dr. David Rawlings and Dr. Andrew Scharenberg. The PCGT is bringing curative molecular therapies to patients with inherited diseases of the blood and immune systems.

The PCGT is building a pipeline of molecular therapies for testing and implementation over the next 10 years. Our first trial, a partnership with St. Jude Children’s Research Hospital, will open in the fall of 2014, and will test the safety and efficacy of gene replacement therapy for "bubble boy syndrome" (a form of severe combined immunodeficiency).

We plan to open safety and efficacy trials of gene replacement for related types of inherited immuonodeficiencies each year for the next five years, with an eventual transition to curative gene repair for selected diseases anticipated by 2019–2020.

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