Gene Therapy Restores Sight To Blind

An anonymous reader writes "Looks like we have found a cure for genetic blindness (clinical trial — abstract — paper [PDF] — ABC News video). This gene therapy treatment increases both cone and rod photoreceptor-based vision. These engineered viruses are implanted to do our bidding to restore vision. Clinical trials on 6 children and young people proved the therapy and didn't find any notable side effects." Any blind person, especially any adapted and competent one, who wants to gain the sense of sight would be well advised to study Oliver Sachs's classic piece "To See and Not See."

Re:um...

[Anonymous Coward]

I think OP means Oliver Sacks.

Here is some more recent work

[ZuchinniOne]

http://www.wired.com/wiredscience/2009/09/colortherapy/ [wired.com]

I have a feeling this will be up for a Nobel Prize. It was seriously groundbreaking work and the entire vision science community is excited about it.

wrong paper

[scapermoya]

after a quick look at the paper linked in the article (Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy success), it is clearly not about gene therapy in humans. it is a study of the thickness of the retina in humans homozygous for a mutation in a specific retinal gene. as the title says, it is a prerequisite for gene therapy.

the actual paper, Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics, can be found here [pnas.org]. It concerns the same gene, incidentally.

Re:coloublind

[Plazmid]

Well if I recall correctly the military tried doing something like this(sans gene therapy) with fighter pilots during WWII. There was a research project to administer fighter pilots a chemical that would make their eyes sensitive to infrared light(night vision infrared not thermal infrared) so they would be better adapted to fighting at night. I don't think much became of it though. Now the only problem with doing this with gene therapy is the effects would be permanent.

Re:coloublind

[Kesch]

Are you sure it is infrared? I had heard that loss of your lens let you see further into ultraviolet (http://en.wikipedia.org/wiki/Aphakia).

Re:coloublind

[TheLink]

ttp://www.livescience.com/history/090429-military-experiment-1.html

The U.S. Navy wanted to boost sailors' night vision so they could spot infrared signal lights during World War II. However, infrared wavelengths are normally beyond the sensitivity of human eyes. Scientists knew vitamin A contained part of a specialized light-sensitive molecule in the eye's receptors, and wondered if an alternate form of vitamin A could promote different light sensitivity in the eye. They fed volunteers supplements made from the livers of walleyed pikes, and the volunteers' vision began changing over several months to extend into the infrared region. Such early success went down the drain after other researchers developed an electronic snooperscope to see infrared, and the human study was abandoned. Other nations also played with vitamin A during World War II - Japan fed its pilots a preparation that boosted vitamin A absorption, and saw their night vision improve by 100 percent in some cases.

Re:Myopia

[dltaylor]

I wish, but ...

When the rods/cones exist in the retina, and the nervous system connections to the brain, but the photo-chemical pathways inside the rods/cones are blocked, this therapy unblocks the chemical error, letting the other components work.

For myopics the damage is different. Our eyeballs are not spherical, so the lens and cornea, matched to a spherical retina surface cannot focus incoming light "incorrectly" onto our distorted retinas. our best bet is still prosthetic. Although the cornea can be hacked up to make some correction, it is not really the issue (it is for astigmatism). What we need are lenses designed for non-spherical retinas. This can emulated by glasses/contacts, but the real solution would be corrective lens implants.

Current materials are not as flexible as natural lenses, so cannot be complete replacements. However, lenses can be shaped for accurate vision at longer than reading distances, or within reading-to-desktop range. As we get older and cataracts appear, there is a stronger justification to replace the lenses, and many older adults no longer have to wear glasses due to replacement lenses. I'm really hoping that by the time I get to replacement, the materials will have been improved so that I can not only stop wearing contacts, but get rid of the reading glasses, too.

Re:coloublind

[dave420]

Well, it definitely was in Britain, when the cavity magnetron was put into use in night-fighters. It provided the first centimetric radar, capable of detecting fighters and even breached submarine periscopes, while being small enough to mount in a fighter. To explain the sudden increase in the nocturnal accuracy of the RAF, the old "carrots help you see in the dark" myth was spun, which had the added benefit of encouraging children to eat healthy food.

Re:Good news, but...

[Jaydee23]

I seem to remember that this is the kind of size for a first human study. I guess this is to make sure that the patients didn't die / develop cancer / turn into zombies. The more detailed studies will happen, but I think when you get to the human trial stage, the ethical considerations suggest a small group.

Re:Seems like an approach to tackle color blindnes

[slashchuck]

From the Abstract it seems this study was reported on July, 2008. Where is the follow up?

Re:Proved?!?!?

[MrCrassic]

Kind of troll-like, but it's breakfast time. This is the way clinical research works; it's all normal procedure. First, you test the new drug on mice. After that doesn't yield disastrous results, you go on to test it on a *handful* of people who express the condition pretty severely. After *that* works, then you test on a much larger sample size, and after that works, the drug is practically ready for mass manufacturing and production.

Re:Good news, but...

[thesandtiger]

Well, it's certainly small for a later stage clinical trial prior to deployment of the treatment, but it's about right for an early trial of efficacy.

With gene therapy you don't want to just start pumping people full of it - there have been some less than fortunate situations in the past, so limiting the initial trial is a wise choice.

Now that this demonstrates that there may be some beneficial effect without horrific side effects, they can ramp it up to a larger trial size and go from there in good conscience.

Re:Myopia

[Cellshade]

There are intraocular lenses now that allow for focusing in the same manner as a natural lens. See: http://en.wikipedia.org/wiki/Intraocular_lens#Accommodating_IOLs [wikipedia.org]