Gene Therapy and Hemophilia A

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Adrian Woolfson, MD, PhD, of Sangamo Therapeutics talks about the physical and psychological advantages of using gene therapy to treat hemophilia A.

Hemophilia A is a genetic blood disorder caused by a missing or defective factor VIII. Symptoms are dependent on the level factor VIII present but in more severe cases, excessive bleeding can lead to severe pain, especially at joints.

Currently, most person with severe hemophilia A take a recombinant factor VIII product to control bleeding. Unlike those treatments that require a lifetime of regular infusions, the gene therapy being developed by Sangamo Therapeutics would require just one injection that would ‘cure’ the person of the disease.
  1. CheckRare
  2. Adrian Woolfson MD
  3. Sangamo Therapeutics
  4. gene therapy
  5. hemophilia A
  6. defective factor VII
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Комментарии
Автор

This could be life changing for us hemophiliacs

tylerpeterson
Автор

IAM hemophilia .I suffer hemophilia factor eight . please tell me about gene therapy permanent treatment of this disease

sakibforazi
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Sir plz...All update News and Vdieo give Your Chelan Hemophilia (A) and Gene therapy😥🙂😥
20+ thousand hemophilia present in Bangladesh😥 🙂😥
I am a Hemophilia (A) present😥

jesunkarim
Автор

Thank you sir for this information. Really sir it helps me alot because i have hemophilia a moderate 3% 😭but sir i have a question without prophylaxis, may gene therapy one time treatment of hemophilia patients? Im 22 years old. im from india .and sir please tell me sir when it will be available in our country and How much money can be used for this treatment? 🙏 Please sir tell me😭

ClipsSala