Imagine CRISPR cures
By Fyodor D. Urnov,
Molecular Therapy
| 10. 27. 2021
A decade of progress in sequencing and in CRISPR/Cas technologies has created a situation without precedent in the history of medicine. Starting with an individual patient, next-generation sequencing can diagnose, in less than 24 h, the genetic basis of a Mendelian disorder.1 Once the causative mutation is found, CRISPR/Cas, in principle, represents a targeted therapy, a first-pass iteration of which can be designed in silico within minutes thanks to straightforward principles of target locus recognition by Cas9.2 The juxtaposition of the two to yield a treatment is not a hypothetical. For Mendelian disorders of hematopoiesis and those that can be treated by editing genes in the liver or the eye, a charted path exists to (1) engineer a CRISPR/Cas-based therapeutic, (2) complete IND-enabling preclinical safety, efficacy, and manufacturing studies, and (3) perform a phase 1/2 clinical trial. Three ongoing such trials have reduced all of this to practice,3,4 with a good safety record in ∼22 (sickle cell disease and transfusion-dependent β-thalassemia), 6 (TTR amyloidosis), and 6 (Leber's congenital amauropathy) subjects dosed to date; in the first case, all subjects for whom...
Related Articles
The Center for Genetics and Society is delighted to recommend the current edition of GMWatch Review – Number 589. UK-based GMWatch, a long-standing ally, was founded in 1998 by Jonathan Matthews as an independent organization seeking to counter the enormous corporate political power and propaganda of the GMO industry and its supporters. Matthews and Claire Robinson are its directors and managing editors.
CGS works to ensure that social justice, equity, human rights, and democratic governance are front...
If you’ve been online or caught the news in the past few weeks, you’ve probably come across Sydney Sweeney, her “great genes jeans,” and much debate over whether they reflect a resurgence of eugenics in American politics and culture.
In case you missed it, here’s what happened. At the end of July, US-based clothing company American Eagle released a new ad campaign. In one ad, Sweeney breathily recites the following, while lying back to zip up her jeans:
Genes are...
By Ryan Cross, Endpoints News | 08.19.2025
Human eggs are incredibly rare cells. The ovary typically produces only 400 mature eggs across a woman’s life. But biologists in George Church’s lab at Harvard University — a group that’s never content with nature’s limits — just got a...
By Staff, National Women's Law Center | 08.13.2025
INTRODUCTION
Baby bonuses. Motherhood medals. Fertility tracking. You may have heard of these policy proposals as solutions from the Trump administration to help encourage women to have more children.
Besides falling short of ensuring that people have what they need...