Scientists recode DNA to help the blind see

Last week British doctors injected lab modified DNA into the eyes of a blind man to restore his sight.  The groundbreaking surgery would not have been possible just a few years ago.

The 29-year-old man has Retinitis pigmentosa (RP), a rare inherited eye disorder. Patients suffer a slow deterioration in vision before ultimately losing sight altogether. The condition is the leading cause of blindness among young Britons and it is irreversible.

At least that used to be the case.

In the New Gilded Age, researchers are working with a new set of rules. After the human genome was successfully sequenced in 2003 it was just a matter of time before scientists started tinkering with the building blocks of life to fix medical disorders.

RP is the result of a single faulty gene, choroideremia.  The defect causes the cells in the retina that detect light to slowly die.  The initial symptoms are poor night and peripheral vision as rods deteriorate.  Eventually the cones fail too, leading to the complete loss of central, detailed and color vision.

In theory, it could all be fixed with a good copy of the bad gene.

Researchers at the NHS, University of Oxford and Nightstar worked for several years under the guidance of Robert MacLaren, professor of ophthalmology at the University of Oxford.  Their lone goal was to develop the gene therapy that would fix the bad gene.

Tinkering with genetic code is complex, even with the latest advances in gene editing.  Changing one gene often leads to unintended consequences for others.  Still, the team persisted.

“Changing the genetic code is always undertaken with great caution, but the new sequence we are using has proven to be highly effective in our laboratory studies,” MacLaren told The Daily Telegraph.

So far, so good.  The first patient is now at home recovering.  The initial results are encouraging but MacLaren acknowledges it could take several years before there is conclusive evidence the deterioration has ceased.

Researchers may have already won.  Gene therapy is moving out of the labs and into the operating rooms. Patients are seeing real breakthrough results.

The BBC reports a French children’s hospital used gene therapy to treat a teenage boy afflicted with sickle cell disease.  Researchers modified the genetic instructions in his bone marrow, resulting in the production of healthy red blood cells.  After 15 months there is still no sign the disease has returned.

Such results are a game-changer for the health care business. In addition to offering real hope for the ill, it resets the rules for pharmaceutical and biotechnology companies, heath care providers and insurers.

This is ultimately what the New Gilded Age is all about.  It’s a reset.  Old rules no longer apply because new technologies bring new business models, new possibilities.

Best Wishes,

Jon D. Markman

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Comments 4

  1. Michael March 22, 2017

    At first it says “into the eyes of a blind man to restore his sight”, but later it says “it could take several years before there is conclusive evidence the deterioration has ceased” Which is it ? Can it make the blind see, or just stop or slow deterioration of present state? Huge difference. It doesn’t say that it can make dead rods and cones regenerate. That would be awesome.

    Reply

  2. FJ Botha March 23, 2017

    I have a blind friend who apparently got the same eye defect. Who can I contact to see if they can help him?
    I live in South-Africa.

    Thank you in advance.

    Reply

  3. Suzie March 23, 2017

    That is fantastic

    Reply

  4. Steven March 25, 2017

    Jon
    Love your research especially when it comes to science. I have an issue I’m hoping you can help me with. I used to have a full head of brown hair, but now it’s turning white! I pull out the white hairs but more seems to grow! Is there any truth to the theory that the more you pull out the more white will come back, and is there anything I can do to reverse or at least stop the white hair from taking over?
    Thanks,
    Steven

    Reply