Welcome to Gene Therapy Science!

Gene Therapy Science is an educational platform intended to further your understanding of the science of gene therapy, with focus on principles specific to hemophilia.

Gene therapy is the introduction, removal or change in genetic material—specifically DNA or RNA—into the cells of a patient to treat a specific disease.1 In its broadest interpretation, the term “gene therapy” may refer to:

Gene transfer² (gene addition): addition of a functional copy of a missing gene or augmentation of a gene that is non-functional into target cells to produce more of a protein ²

Gene editing:² removal, disruption or correction of faulty elements of DNA within the gene

Cell therapy:² transfer of intact, live cells into a patient

Throughout these webpages, when referring to “gene therapy”, the focus will be on the first of these approaches: gene transfer.

The content on this website will explore areas as they relate to hemophilia, including:

Visit our learning center and news and events page for the latest updates and to explore further resources.​​​​​​​

References1. American Society of Gene and Cell Therapy: Gene and Cell Therapy. www.asgct.org/education/more-resources/gene-and-cell-therapy-faqs (Accessed April 2021). 2. National Hemophilia Foundation: Future Therapies FAQs. www.hemophilia.org/bleeding-disorders-a-z/treatment/future-therapies/fr… (Accessed April  2021). ​​​​​​​PP-HEM-USA-1447
Date of preparation: February 2022. 

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