MAR 02, 2019 8:36 AM PST

Why Gene Therapy for Blood Disorders is Especially Challenging

WRITTEN BY: Carmen Leitch

Sickle cell disease is a group of disorders that can cause a range of symptoms that may include fatigue and jaundice, as well as severe pain. The disorders, one of which is sickle cell anemia, are caused by mutations in a gene that encodes for the hemoglobin protein. That protein is an important carrier of gases in the body, ferrying oxygen to cells. When hemoglobin is mutated, it causes the symptoms of the disease. 

Curing genetic diseases is as simple, in theory, as fixing the mutations in the genome that cause them. Naturally, that is actually very difficult to achieve in reality for many reasons. Researchers have been making strides in the development of gene therapies for some disorders, however. Learn more from the video about why it’s especially difficult to come up with a gene therapy for sickle cell anemia, and how scientists are trying to get around those problems.

About the Author
  • Experienced research scientist and technical expert with authorships on over 30 peer-reviewed publications, traveler to over 70 countries, published photographer and internationally-exhibited painter, volunteer trained in disaster-response, CPR and DV counseling.
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