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posted by martyb on Sunday March 08 2020, @09:21PM   Printer-friendly
from the seeing-is-believing dept.

Arthur T Knackerbracket has found the following story:

In a world first, CRISPR, the powerful gene-editing tool that can cut and paste DNA, has been used inside the human body for the first time. Scientists at the Casey Eye Institute at Oregon Health & Science University, Portland, have administered a new CRISPR-based medicine to treat an inherited form of blindness, according to the two biotech companies which make the treatment.

"This dosing is a truly historic event -- for science, for medicine, and most importantly for people living with this eye disease," said Cynthia Collins, president and CEO of Editas Medicine, a gene-editing company headquartered in Massachusetts.

The first patient in the trial received a dose of the experimental drug, called AGN-151587, via an injection in the eye. The idea is that it delivers the gene-editing tool CRISPR directly to cells in the eye which are affected by the genetic disease. CRISPR is able to find its way into those cells and correct the gene -- a cut-and-paste scenario that sees a tiny DNA edit made to remove the mutation.

Importantly, the CRISPR edit is permanent, which means patients may only need a single dose and be set for life.

The trial is expected to enroll 18 patients in total and will look at different doses of the experimental drug, refining how much is necessary to achieve the goal of reversing blindness -- without any side effects. Information about the first patient is scant, with researchers staying silent on patient information and when the surgery officially occurred.

From the press release:

AGN-151587 (EDIT-101) is an experimental medicine delivered via sub-retinal injection under development for the treatment of Leber congenital amaurosis 10 (LCA10), an inherited form of blindness caused by mutations in the centrosomal protein 290 (CEP290) gene. The BRILLIANCE clinical trial is a Phase 1/2 study to evaluate AGN-151587 for the treatment of patients diagnosed with LCA10 and is the world's first human study of an in vivo, or inside the body, CRISPR genome editing medicine. The trial will assess the safety, tolerability, and efficacy of AGN-151587 in approximately 18 patients with LCA10.

[...] "Currently patients living with LCA10 have no approved treatment options. For years, Allergan has had an unwavering commitment to advancing eye care treatments. With the first patient treated in this historic clinical trial, we mark a significant step in advancing the AGN-151587 clinical program and move closer to our goal of developing a game-changing medicine for LCA10 patients," said Brent Saunders, Chairman and CEO, Allergan.

I have a friend who has gone mostly blind in her mid-20s from a combination of genetic predisposition and environmental factors. Different circumstances than these, but I hope that the more is learned along the way, the more that can be done to alleviate vision loss.


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  • (Score: 2) by fishybell on Monday March 09 2020, @12:44AM (3 children)

    by fishybell (3156) on Monday March 09 2020, @12:44AM (#968355)

    I always loved this example: picking the eye color of your baby.

    If this is supposed to elicit a response down the slippery slope of "we're so preoccupied with whether or not we could, we didn't stop to think if we should" type badness, what exactly would be bad about choosing the eye color of a baby? There are so many better arguments like "manual selection of genes might lead to a lack of genetic diversity."

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  • (Score: 0) by Anonymous Coward on Monday March 09 2020, @02:00AM

    by Anonymous Coward on Monday March 09 2020, @02:00AM (#968378)

    I would go for resistance to diabetes, heart disease, asthma, obesity, alcoholism, psychiatric predilections, cancer, even hair loss first.

  • (Score: 0) by Anonymous Coward on Monday March 09 2020, @04:52AM

    by Anonymous Coward on Monday March 09 2020, @04:52AM (#968420)

    Aren't dark color eyes better for vision? If so wouldn't you be giving your child worse vision by picking a lighter color like blue or green.

    If people give their children uncommon traits for aesthetic purposes it's possible that those uncommon traits are uncommon exactly because natural selection selected against them. It's possible that those uncommon traits have disadvantages associated with them which is why they are uncommon. Natural selection has been selecting various traits for a long time, what's true and tested, and for us to think we know better is something we should be careful about.

  • (Score: 2) by Bot on Monday March 09 2020, @08:04AM

    by Bot (3902) on Monday March 09 2020, @08:04AM (#968441) Journal

    What is inherently bad is assigning control.
    You would trust the same system that gave you windows and systemd, put radios in every CPU, and soon RFID in every fucking product, you included, to determine the genetic traits of your child. It's so insane that I am sure you haven't worked out all the implications.

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    Account abandoned.