Doctors Alter U.S. Woman's Genes with CRISPR

For the first time ever, doctors in the United States have used CRISPR to alter the genes of an adult woman. CRISPR is a controversial genetic editing tool that has previously been used to edit human embryos.

Victoria Gray is a 41-year-old woman from Mississippi, and the first person in the United States to have her cells altered with CRISPR. A patient in Germany was the first to have their genes altered with CRISPR, by a company called CRISPR Therapeutics. That patient’s name has not been revealed.

The patient in Germany was treated for beta thalassemia, and reportedly has not required blood transfusions for four months. Victoria Grey has sickle cell disease.

The Centers for Disease Control and Prevention explains that sickle cell disease is a group of inherited blood disorders. Healthy red blood cells carry oxygen to all parts of the body. A person who has sickle cell anemia has red blood cells that become hard and sticky and form a C-shape, (like a farm tool called a sickle). These red blood cells do not carry oxygen very well, and can get stuck and clog blood flow.

CRISPR is also known as CRISPR/Cas9. It is a gene editing tool. It works by identifying a specific strand of DNA and replacing it by “cutting” the original DNA and “pasting” in replacement DNA.

CRISPR Therapeutics, which is based in Cambridge, Massachusetts, is focusing on developing gene-based therapies. Vertex Pharmaceuticals of Boston will enroll around 45 people who are between the ages of 18 and 35 in a joint study. The purpose is to learn if it is possible to permanently “fix” blood cells via CRISPR.

The outcome of the study will be interesting. Right now, there is no cure for sickle cell disease. Altering genes with CRISPR has the potential to improve lives.

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