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PKAN Gene Therapy

The PKAN physician-scientists at OHSU and scientists from the Horae Gene Therapy Center at UMass Chan Medical school have recently formed a partnership to develop gene therapy for PKAN. Early work in PKAN mice was initially done by Dr. Lauriel Earley, a former graduate student in Molecular & Medical Genetics at OHSU. Dr. Earley’s work was completed in the Hayflick lab after she departed to start a new position in industry. Now, using the preliminary mouse studies as a launching pad, OHSU and UMass are working to move the project forward. These experts from OHSU and UMass bring complementary skill sets, decades of experience, and a collaborative approach to the partnership. Meanwhile, the Loving Loic Foundation is working tirelessly to raise the necessary funds for the project. The Loving Loic Foundation was established in late 2023 by the Blackford family in honor of their son, Loic, who had recently been diagnosed with PKAN. Their mission is to fund research for developing a lifetime cure for PKAN for all children through the development of an effective gene therapy. The Loving Loic Foundation works closely with the NBIA Disorders Association and other advocacy groups. Donations to support this project can also be made to a dedicated PKAN Gene Therapy Research Fund through the OHSU Foundation.