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> We injected 4-week-old rd12 mice subretinally with LV-ABE-A5, LV-ABE-A6 or LV-ABE-NT

This was a key detail I wasn’t able to find in the linked article, which was when the treatment was being applied.

They aren’t doing gene editing on embryos, but actually applying the treatment to living mice.



It's cool but similar things have already been done in humans.


I had this done in 2017 as part of a trial for a gene therapy for X-Linked Juvenile Retinoschisis.

https://clinicaltrials.gov/ct2/show/NCT02317887

I had an adverse reaction to the vector and have had eight surgeries since to save some of the sight in the eye they injected.


This is off-topic, but can someone educate me on how to read clinicaltrails.gov? When looking at a trial page, it's hard for me to tell the result of the trial. Usually it just says "Completed" with "No results posted". Also, it doesn't show which phase the study is in, with no history of the previous phases.


Phase is listed, this trial was phase 1 and 2 (2a in particular according to the paper). The summary usually contains motivating background information about why they’re taking this approach.

The results haven’t been posted directly, but there is a publication linked that has some of the results so far.

A lot of this stuff is not rigidly structured - with a mix between recording actual data, references to actual data, or just having the study design if the trial is still early.


Thanks for sharing. That must've been a huge disappointment to come out of the study with that and very difficult to deal with. I'm awaiting the start of trials for blue-cone (x-linked achromatopsia). I've been very uncertain of how badly I wish to participate.




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