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."

Comments

um...

[fyngyrz]

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."

How?

Re:um...

[Fluffeh]

Braille? Text to speech? Any one of the other multitude of ways that visually impaired people deal with daily to get through life meant for people with vision?

Not to be blunt, but seriously...

Re:um...

[edittard]

Blind people don't read slashdot. They edit it.

Myopia

[mxh83]

Could this apply to myopia too? Could it be an option to LASIK?

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:Myopia

[dltaylor]

There's a "design flaw" in the lens. Unlike bones, that have cells that both remove and replace bone, the lenses only have cells that smooth the surface by adding more material. After a few decades, the lens is too thick to be stretched for close focus, so we lose that ability, although distance vision may still be as good as when young.

Some people can tolerate a pair of replacement lenses, one near-focusable for reading and one far-focusable, between them covering the full range of vision. IIRC, the dominant eye is close-focusable. Contacts are available in the same arrangement, but, again, not everyone can tolerate them.

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

coloublind

[retech]

I'd do it for colourblindness.

"...if only you could see what I've seen with your eyes!"

Re:coloublind

[MichaelSmith]

I agree. I can hardly see into the ultraviolet and infared and I would love to be able to see microwaves...

Re:coloublind

[Nefarious Wheel]

Interestingly enough, the acrylic lenses used in cataract lens replacement therapy are a bit more transmissive in the infra red than the ones you are born with.

I haven't noticed much improvement along those lines (I haven't done any empirical studies myself) although my night vision is superb compared with how it was at any time prior to the surgery.

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:coloublind

[CarpetShark]

I would love to be able to see microwaves...

Kitchen tours. $10 a pop. Kids unwelcome.

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:coloublind

[Vahokif]

which had the added benefit of encouraging children to eat healthy food

And, incidentally, food you could get with rationing.

Re:coloublind

[RealErmine]

Chlorophyll eye drops help night vision: Article here.

Blindness

[Thanshin]

Losing sight has always been my greatest fear. I understand a lot of blind people can live perfectly fine lives, but I can't think of many worse futures. (I know the news are about genetic blindness, but still).

The day someone comes up with a way of completely bypassing the eyes, for example by perfecting the technology of connecting cameras directly to the brain, will feel as important for me as the day someone finds a way of curing all medular wounds.

It may sound stupid but one of the few reasons I've got for accumulating more money is being able to pay the medicine I hope will exist by the time my body starts failing in those kind of ways.

Re:Blindness

[Nefarious Wheel]

Losing sight has always been my greatest fear. I understand a lot of blind people can live perfectly fine lives, but I can't think of many worse futures

Agree wholeheartedly. I was blind for a year, and was cured. Once you lose your sight you would crawl through broken glass if it meant you could get your eyesight back.

I can see my wife's face, and my daughters are beautiful. Bring on science.

Re:Blindness

[outsider007]

Easy for you to say, my wife's got the face of a saint - a Saint Bernard.

Re:Blindness

[spooje]

I'm guessing it was chronic masturbation.

Re:Blindness

[apoc.famine]

Even if you call her "Pamela Handerson", it's still not screwing...

There were notable side affects

[^switch]

"All three patients showed a statistically significant increase in visual sensitivity at 30 days after treatment localized to retinal areas that had received the vector."

Well, one notable side-affect of the virus was improved vision.

Re:There were notable side affects

[Linker3000]

Better peripheral vision = Side effect!?

Re:Old news?

[I kan Spl]

It seems that you do not appreciate how long it takes to do real research.

Here is some more recent work

[ZuchinniOne]

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

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.

Rogue-like game for the blind

[EvilDingo]

My children might have an incurable genetic blindness (we haven't tested them) that causes progressive blindness. After researching a bit, I found that the blind and visually impaired can use computers quite well with screen readers, but there wasn't a lot of accessible software -- especially games. http://www.audio-games.net/ was a great resource and helped me design an accessible audio-RPG called Entombed. http://www.blind-games.com/ - Full disclaimer: my site. I think the biggest hurdle (obvious from reading some of these comments) is that there isn't a lot of awareness that the blind can navigate and use computers.

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. It concerns the same gene, incidentally.

Oblig...

[Psychotic_Wrath]

Nothing to see here move along..... wait a second...

Going blind sucks, I should know...

[frank_carmody]

I have retinitis pigmentosa which affects me in a number of different ways. At the moment it's the night blindness that's the most problematic. But as the disease is a degenerative one and as there's no way to predict (or even give a rough estimate of) the time when I will be fully blind, not a day goes past when I don't think of what it will be like to be completely in the dark. I read these stories all the time and they're all like stories on holographic storage tech: Just 5-10 more years and it'll be here for me to enjoy...

Re:Going blind sucks, I should know...

[Anonymous Coward]

Me too. I was diagnosed 25 years ago, with a predicted 10 years before I would be completely blind. The disintegration has actually been a bit slower.

Lots of things I can't do, but it doesn't hurt, and it really doesn't make life that difficult.

The only problem I have at all is when I walk in to people by accident. One one occasion, I was accused of being on drugs, and on a couple of occasions (spilling a pint in a pub, for example), people have been pretty rude and wanted to start a fight.

Honestly, without the intolerance from people, it is perfectly easy to cope. Never use your condition as an excuse, and good luck.

Another ABC News 'Miracle'

[2phar]

As usual, ABC News reaches for the M word. Nothing supernatural.. more like many years of painstaking and brilliant science.

Interesting read

[Citizen of Earth]

would be well advised to study Oliver Sachs's classic piece "To See and Not See."

Yeah, now it's 5:30 in the morning. Thanks a lot.

Good news, but...

[vegiVamp]

Is it just me, or does 6 patients seem rather few for a significant trial ?

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: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.

Then and now

[Mr2001]

People are still making plenty of text games, even more elaborate than the ones from the 80s (thanks to increased memory capacity, better tools, and evolving expectations). And indeed, they're popular with blind players who use screen readers.

Proved?!?!?

[Secret Rabbit]

Um, it takes a fuck load more than SIX kids to /PROVE/ something. SIX isn't anywhere close to statistical significance, nor does it even remotely demonstrate safety. Proven/proof are VERY big words and shouldn't be thrown around lightly. These preliminary results may be encouraging, but are FAR from proof. Especially, in the medical field.

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: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?

you are correct

[zogger]

I have the exact same situation. Even down to the not learning as much about electronics back in the day because I would miss on wire colors and resistor color bands etc. The other thing you mention, in seeing detail in the field is also correct. I can see animals and oddball stuff hidden in the bushes etc quite well. Even beat my dogs a lot when we are out walking around, because my brain doesn't think in color so much as it does shapes/lines, etc. I only see some very "loud" and brilliant basic colors, shades, etc..nope. Miss a lot. The docs said I was red green deficient. Well, heck ya! That's why I got tested in the first place...

  Anyway, I'll see the rabbit or squirrel or deer or wild turkey, etc well before they do, even close by. And other oddball things like that..for instance I can walk into a room and if a pin is on the floor, dropped accidentally, I will see it almost immediately, it just jumps out as a "wrongness" to the over all expected patterns. Coins on the sidewalk, freaking lots of them over the years. Even just the roundness of a copper penny will stand out to me laying in a green lawn.

So ya, tradeoffs, your brain compensates.