CADTH Lecture Series: Gene Therapy

For diseases caused by inherited or acquired genetic mutations, the ideal remedy is to correct the relevant mutations. Recently, promising results have emerged about gene therapy for patients with inherited conditions as well as in patients with acquired hematological malignancies, with positive results for the latter being obtained by infusing chimeric antigen receptor T-cell therapy (CART). Using CART as the most prominent example of gene therapy, this talk will provide a background to gene therapy and to blood and marrow transplantation. Dr. Seftel will summarize the available data about CART therapy and will also outline the clinical, economic, and regulatory challenges associated with CART and other gene therapeutics.

A live stream broadcast will also be available online for those unavailable to attend in person.

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