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Potential of
Gene Therapy

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illustration of both a mutated gene in hemophilia and healthy or functional gene

Gene therapy (transfer or addition) Gene therapy (transfer or addition) The transfer or addition of genetic material into a cell. Once in the cell, a working copy of a gene can help make proteins despite the presence of a faulty or mutated gene. doesn’t just aim to treat symptoms of a disease or disorder. Instead, gene therapy can potentially help treat certain diseases by introducing a healthy (working or functioning) gene that may improve the burden of hemophilia treatment.

illustration of a healthy, working, functioning gene

In the case of hemophilia A or B, the introduced gene is intended to inform the body to produce the factor VIII or factor IX protein involved in blood clotting.

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Gene therapy (transfer or addition) Gene therapy (transfer or addition) The transfer or addition of genetic material into a cell. Once in the cell, a working copy of a gene can help make proteins despite the presence of a faulty or mutated gene. doesn’t just aim to treat symptoms of a disease or disorder. Instead, gene therapy can potentially help treat certain diseases by introducing a healthy (working or functioning) gene may improve the burden of hemophilia treatment.

In the case of hemophilia A or B, the introduced gene is intended to inform the body to produce the factor VIII or factor IX protein involved in blood clotting.

Gene therapy
research in
hemophilia

Gene therapy has been studied for more than 50 years across a variety of medical conditions, and for over 20 years in hemophilia. Research is continuing today with clinical trials ongoing.
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Scientists are currently researching if gene therapy can help patients achieve potential one-time dosing while minimizing their risk of bleeds.
Gene therapy may help some people with hemophilia manage their disease without the need for frequent infusions or injections.
While scientists continue to research gene therapy in hemophilia, it’s important to note gene therapy may not be appropriate for all people with hemophilia.
For more information on who has been included in gene therapy research so far, click here.
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Scientists are currently researching if gene therapy can help patients achieve potential one-time dosing while minimizing their risk of bleeds.

While scientists continue to investigate the promise of gene therapy in hemophilia, it’s important to note gene therapy may not be appropriate for all people with hemophilia.

For more information on who has been included in gene therapy research so far, click here.

Gene therapy may help some people with hemophilia manage their disease without the need for frequent infusions or injections.

As research evolves, this site will be updated to include new information over time.

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