Skip to main content
Premium Trial:

Request an Annual Quote

For the Rare Diseases

Researchers are pursuing CRISPR-Cas9 gene editing-based treatments for an increasing number of rare diseases, the Washington Post reports.

It notes that researchers are testing CRISPR gene-editing treatments for rare conditions with few or no treatment options, with some promising results. For instance, it, as well as NPR and others, note that researchers with Vertex Pharmaceuticals and CRISPR Therapeutics have tested an approach using CRISPR to turn on fetal hemoglobin within bone marrow from people with sickle cell anemia and beta-thalassemia.

Another trial from Intellia Therapeutics, the Post adds, is taking a different tack and examining the use of CRISPR to knock out the affected gene in people with transthyretin amyloidosis, which leads to heart failure and incontinence. The Post notes that there are some 30 million people in the US with a rare diseases and that the 70,000 different conditions may need their own approach.

It adds, though, that Mario Amendola from the French nonprofit group leader Généthon and colleagues are developing a set of tools with the aim of making the development process easier. "We would like to use this as a plug-and-play platform for many different diseases, so that we don't have to re-optimize the whole system every time," Amendola tells the Post. "We just need to change it a small bit, which hopefully can speed up the drug development process."