Gene-editing used for sickle cell patients for first time

The same gene-editing technology Temple used to eliminate HIV in mice is being tested for removing sickle cell disease in humans and researchers say this is a really promising technology.

This is the first time the CRISPR technique is being used to treat a patient.

The sickle cell trials are underway at eight centers in the US, Canada, and Europe, including Children's Hospital of Philadelphia.

Vertex Pharmaceuticals has developed the treatment, called CTX001 in its testing phases.

Doctors start with cells from the patients' own bone marrow, then genetically modify them, inserting material that will produce a protein usually only made by babies after birth.

The hope is the new protein will compensate for the defective one that causes red blood cells to become deformed.

Those deformed cells cause pain, and raise the risk of infection and organ damage.

It will be months before it's known if the genetically-edited cells will produce enough good protein, but doctors will be watching closely.
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