StemCell Therapy

A few doctors performing an eye procedure.

Portra/Getty

For the first time in the world, CRISPR, the powerful gene editing tool that can cut and paste DNA, has been used inside a human body. Scientists at the Casey Eye Institute at Oregon Health & Science University, Portland, have administered a CRISPR-based drug to treat a form of congenital blindness, according to two biotech companies that created the treatment.

This dose is truly a landmark event for science, medicine, and most of all for people suffering from eye disease, said Cynthia Collins, president and CEO of Editas Medicine, a Massachusetts-based gene editing company.

This state-of-the-art clinical trial aims to test an experimental treatment for the congenital condition known as Leber 10 congenital amaurosis. The disease is caused by a faulty gene that results in blindness from birth or during the first months of life. The evil affects one in every 40,000 children born. There is currently no approved treatment option.

The first patient in the clinical trial received a dose of the experimental drug called AGN-151587 through an injection into the eye. The idea is that the drug takes the CRISPR tool directly to the cells of the eye that are affected by the evil eye. CRISPR is able to find its way into those cells and correct the gene by editing the DNA to remove the mutation.

The editing done by CRISPR is permanent, which means that patients need a single dose of the drug.

The clinical trial is expected to involve 18 patients in total and will analyze the use of different doses of the drug, to refine how much is needed to reach the goal of reversal of blindness without causing side effects. There is little information about the first patient of this treatment; it is not known when the procedure took place.

CRISPR has also been at the center of the controversy regarding gene editing. In November 2018, Chinese scientist He Jiankui revealed that there was created the first human embryos edited with CRISPR and that resulted in the birth of twin girls. The scientific community was outraged by Hes work, which bypassed various ethical and regulatory approvals.

Scientists have been working to refine the ability of gene editing tools like CRISPR so that they can edit DNA accurately and effectively. In October of last year, a team of researchers from Harvard University presented a cutting-edge technology known as base editing the editing of bases.

Playing:Watch this:

The 5 geek movies we liked the most in 2019

5:38

The rest is here:
CRISPR is used for the first time within the human body - The Bulletin Time

This entry was posted on September 15, 2020 at 10:55 am and is filed under Blindness. You can follow any responses to this entry through the RSS 2.0 feed. Both comments and pings are currently closed. |