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First attempt to use CRISPR tool to treat a genetic disorder

Cells are being taken from patients' bone marrow and genetically modified with CRISPR

29-Jul-2019

Key points from article :

CRISPR enables scientists to make very precise changes in DNA. 

CRISPR technology is being used in China to treat cancer. 

Doctors in Boston are planning to use CRISPR to edit cells in patients' retinas. 

Victoria Gray received an infusion of billions of genetically modified cells. 

Patients' cells modified with CRISPR produce a protein that is usually only made by fetuses. 

Patients then undergo chemotherapy for killing the existing cells that are carrying the genetic defect. 

It may take months or even years for noticing the effects on patients' health.

Mentioned in this article:

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CRISPR Therapeutics

Company developing transformative gene-based medicines for serious human diseases

David Altshuler

Executive Vice President and Chief Scientific Officer at Vertex Pharmaceuticals.

Haydar Frangoul

Pediatric hematologist-oncologist

Laurie Zoloth

Margaret E. Burton Professor of Religion and Ethics at University of Chicago

Vertex Pharmaceuticals

Company that invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases

Topics mentioned on this page:
Gene Therapy