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Genetic medicine: cure the blind with one shot

Sick with a severe genetic disease that took away his ability to see, he was treated in the last two years with advanced genetic medicine that gave him a chance for a normal life.

The three researchers from the Children's Hospital of Philadelphia and the University of Pennsylvania
The three researchers from the Children's Hospital of Philadelphia and the University of Pennsylvania

Some children zigzag. Unlucky children. Children born with genetic diseases that cause them to be disabled, blind or mentally retarded. Even with all the efforts of conventional medicine, it is very difficult to provide a cure for such children, because they were born with the disease present in each of their cells, in the form of one or more defective genes. Each gene is responsible for the production of a different protein, and if one of the genes is damaged as a result of a mutation, then the enzyme under its supervision will not be produced in the child's cells. And if the same enzyme is responsible, an act of Satan, for the absorption of light in the eyes, then the child with the defective gene will not have the light of his eyes.

The solution is found in the ancient Jewish proverb: "Give me a fish - and I will eat." Teach me to fish - and I won't be hungry all my life." The children's problem is that their cells do not know how to 'fish'. Their defective gene does not contain the correct manufacturing instructions for the enzyme. The best way to help them would be to teach the cells to produce the correct enzyme, and for this it is necessary to insert the correct gene into them. For this, a particularly powerful treatment is needed: actual genetic engineering. Such is the case of child Z, sick with a severe genetic disease that took away his ability to see, but was treated in the last two years with advanced genetic medicine that gave him a chance for a normal life.

Child Z was born with a genetic disease called LCA, which causes retinal degeneration. He was declared blind in the eyes of the law, and the only way he could study was at the back of the classroom, with a large electronic screen hanging in front of his eyes and next to him the teaching assistant reading the study material to him. The turning point occurred when he joined the human trial group in the first large-scale genetic medicine treatment for LCA. Twelve subjects, between the ages of eight and forty-four, received a single injection of genetically engineered viruses. Those viruses carried within them the gene responsible for producing light-sensitive receptors in the eye. The same gene is also present in patients, but in its defective state it is unable to produce normal receptors. The viruses infected the cells in the eye with the normal gene, causing them to start producing the light receptor that was missing in the patients.

The improvement in the condition of the patients happened quickly. Two weeks after the injections, all dozen subjects reported an improvement in the ability to see in the eye into which the viruses were injected. An objective test, designed to measure how much the pupil of the eye is able to contract in response to light, showed that all the subjects were indeed able to detect light better after the treatment, and that there was a higher sensitivity to light in the treated eye, compared to the untreated eye.

The video attached to the study demonstrates the dramatic improvement in the eyesight of child Z, when he is asked to go through an obstacle course with a cover over one of his eyes. When the eye that received the treatment is covered, the child is almost unable to locate the obstacles. On the other hand, when the eye is covered and the viruses have been injected into it, the child goes through a similar path relatively easily, even under low light. The treatment seems to help children in particular, as some adults did not improve their results on the obstacle course, and those who did did so to a lesser extent than children.

Despite the impressive success, especially in children, there is still no complete certainty about the safety of the treatment. At the same time, it is living proof of the ability of genetic medicine to treat diseases that were incurable until now. As the technology improves, we will surely be exposed to additional treatments for other hereditary and genetic diseases.

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12 תגובות

  1. Anonymous:
    right.
    In my opinion, it should have been written "the eye that has not been injected with viruses" (or instead of "covered" it should have been written "visible")

  2. : "On the other hand, when the viruses were injected into the blindfold, the child goes through a similar path relatively easily, even under low light." What does that mean……………?????????????.

  3. Is it possible to program the viruses to die after infecting the last cells in the eye? For example, by adding an enzyme that will shorten their chromosome in each division at such a rate that after infecting a number of cells they will no longer be able to divide?

  4. I would exercise caution in relation to genetic Hinduism in general and the use of viruses in particular, because you never know how the viruses will react with the body in the long run. And in addition, I really hope that there is no possibility of infection, since perhaps infecting a healthy person could have serious consequences. In short, I hope the research team was very, very responsible...

  5. For number 4 - facing more difficult things, when it is not a risk but treatment is not scary in the end, look for vitrectomy on Youtube

    The question is why does a child get an advantage and an adult does not?
    Is there any "home" learning of genes?

  6. magnificent,
    If there is something I will agree to contribute to, it will usually be this type of research, which treats the problem from the root...

  7. An injection into the eye?? How can a normal person (and another child!) be able to withstand such a thing?? This is madness!

  8. Amazing! Absolutely amazing!
    I can't wait for what the future brings. I can literally imagine a world without diseases and disabilities.
    The blind will see, the crippled will walk, and the crippled will feel.
    Thanks Roy for another interesting article!
    By the way: "In contrast, when the eye is covered and the viruses have been injected into it, the child goes through a similar path relatively easily, even under low light." - Shouldn't it be the eye that hasn't been treated?

  9. If possible for the sake of good order call the process gene healing or gene therapy, not genetic. This is because the use of the word genetic speaks of the inheritance of a trait, which is not the case with this treatment. Thanks Oren

  10. This kid sounds a bit German, maybe you will call him "Bernard Joachim" (the name is saved in the system) for the purpose of the article, that way it will be clearer.

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