CRISPR and another genetic strategy fix cell defects in two common blood disorders
By Jocelyn Kaiser,
Science
| 12. 05. 2020
It is a double milestone: new evidence that cures are possible for many people born with sickle cell disease and another serious blood disorder, beta-thalassemia, and a first for the genome editor CRISPR.
Today in The New England Journal of Medicine (NEJM) and tomorrow at the American Society of Hematology (ASH) meeting, teams report that two strategies for directly fixing malfunctioning blood cells have dramatically improved the health of a handful of people with these genetic diseases. One relies on CRISPR, marking the first inherited disease treated with the powerful tool created just 8 years ago. And both treatments are among a wave of genetic strategies poised to widely expand who can be freed of the two conditions. The only current cure, a bone marrow transplant, is risky, and appropriately matched donors are often scarce.
The novel genetic treatments have the same safety issues as bone marrow transplants for now, and may also be extraordinarily expensive, but there is hope those risks can be eliminated and the costs pared down. “This is an amazing time, and it’s exciting because it’s...
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Following a long-standing CGS tradition, we present a selection of our favorite Biopolitical Times posts of the past year.
In 2025, we published up to four posts every month, written by 12 authors (staff, consultants and allies), some in collaboration and one simply credited to CGS.
These titles are presented in chronological order, except for three In Memoriam notices, which follow. Many more posts that are worth your time can be found in the archive. Scroll down and “VIEW...
