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...
Related Articles
By Sarojini Nadimpally and Gargi Mishra, The Wire | 12.15.2024
In-vitro fertilisation (IVF) as assisted reproductive technology (ART) has been in vogue for quite a few decades now. While IVF has been hailed as a significant scientific advancement, with many advantages, here are some limitations which bear keeping in mind...
Image by Mohamed Hassan from Pixabay
It is hard to make predictions, especially about the future, as Yogi Berra, Niels Bohr, and other luminaries have remarked. But there are already signs that the incoming Trump administration may have some difficulty establishing consistent policies about controversial issues concerning human reproduction.
On the one hand, consider “the conservative blueprint for a second Trump administration.”
The notorious Project 2025’s Mandate for Leadership seeks to delete terms such as “reproductive rights” from “every federal...
By Susan Dominus, The New York Times | 11.30.2024
In the days after Daphna Cardinale delivered her second child, she experienced a rare sense of calm and wonder. The feeling was a relief after so much worrying: She and her husband, Alexander, had tried for three years to conceive...
