Gene therapy to cure disease and possibly aging

by Comatose 11 Replies latest jw friends

  • Comatose

    This article is amazing. There have been other recent articles about reversing aging in mice. Science is the solution.

    The findings, described in the March 30 issue of Nature Biotechnology, offer the first evidence that this gene-editing technique, known as CRISPR, can reverse disease symptoms in living animals. CRISPR, which offers an easy way to snip out mutated DNA and replace it with the correct sequence, holds potential for treating many genetic disorders, according to the research team.

    "What's exciting about this approach is that we can actually correct a defective gene in a living adult animal," says Daniel Anderson, the Samuel A. Goldblith Associate Professor of Chemical Engineering at MIT, a member of the Koch Institute for Integrative Cancer Research, and the senior author of the paper.

    The recently developed CRISPR system relies on cellular machinery that bacteria use to defend themselves from viral infection. Researchers have copied this cellular system to create gene-editing complexes that include a DNA-cutting enzyme called Cas9 bound to a short RNA guide strand that is programmed to bind to a specific genome sequence, telling Cas9 where to make its cut.

    At the same time, the researchers also deliver a DNA template strand. When the cell repairs the damage produced by Cas9, it copies from the template, introducing new genetic material into the genome. Scientists envision that this kind of genome editing could one day help treat diseases such as hemophilia, Huntington's disease, and others that are caused by single mutations.

    Scientists have developed other gene-editing systems based on DNA-slicing enzymes, also known as nucleases, but those complexes can be expensive and difficult to assemble.

    "The CRISPR system is very easy to configure and customize," says Anderson, who is also a member of MIT's Institute for Medical Engineering and Science. He adds that other systems "can potentially be used in a similar way to the CRISPR system, but with those it is much harder to make a nuclease that's specific to your target of interest."

    Disease correction

    For this study, the researchers designed three guide RNA strands that target different DNA sequences near the mutation that causes type I tyrosinemia, in a gene that codes for an enzyme called FAH. Patients with this disease, which affects about 1 in 100,000 people, cannot break down the amino acid tyrosine, which accumulates and can lead to liver failure. Current treatments include a low-protein diet and a drug called NTCB, which disrupts tyrosine production.

    In experiments with adult mice carrying the mutated form of the FAH enzyme, the researchers delivered RNA guide strands along with the gene for Cas9 and a 199-nucleotide DNA template that includes the correct sequence of the mutated FAH gene.

    Using this approach, the correct gene was inserted in about one of every 250 hepatocytes -- the cells that make up most of the liver. Over the next 30 days, those healthy cells began to proliferate and replace diseased liver cells, eventually accounting for about one-third of all hepatocytes. This was enough to cure the disease, allowing the mice to survive after being taken off the NCTB drug.

    "We can do a one-time treatment and totally reverse the condition," says Hao Yin, a postdoc at the Koch Institute and one of the lead authors of the Nature Biotechnologypaper.

    To deliver the CRISPR components, the researchers employed a technique known as high-pressure injection, which uses a high-powered syringe to rapidly discharge the material into a vein. This approach delivers material successfully to liver cells, but Anderson envisions that better delivery approaches are possible. His lab is now working on methods that may be safer and more efficient, including targeted nanoparticles.

  • Iamallcool

    (bookmarking) for my JW parents. I will print the link for them not this website for sure.

  • snare&racket

    yeah this website is poor in it's filter for good / pseudo science.... I know it quotes a journal, but that doesn't mean diddly squat, every paper needs appraising individually.

    Honestly, this is very premature. I wasn't going to say anything only lamallcool said about sending it to his parents. This is exciting science and I have been awaiting this exact technologu for some time, (since my genetics class before med school) & I truly hope they have what they say they have.

    But science has already devloped cures for disease.... it long ago proved WT not only wrong but irrelevant. Watchtower has never so much as raised money for medical research or even someomes trestment, never mind brought cures to the world. Meanwhile... science has. Simply look at the stats for pre and post vaccinations, sometjing that Jehovah's Witnesses were against in the very recent past.

    Cures for disease, fighting off of death, removal of pain, increased quality of life..... all things watchtower PROMISE, but it remains behind a curtain that nobody but 8 men in NY have been granted ti see behind and understsnd, you just need to follow their instructions to one day have it!

    Cures for disease, fighting off of death, removal of pain, increased quality of life..... all things available today, now and free for all to know, via science.

    A JW I know said no to an amazing new treatment recently, because she wanted to wait for Jehovah.... there are no words to convey the sense of disgust at the blood spilt, lives lost & wasted, treatment refused, science rebuked and doctrines died for.....all to protect the ego of 8 men in New York who have gone too far & who carry too much responsibility to now admit their human, ignorant uneducated status.

  • Comatose

    Snare I visit several science sites a day trying to keep up and learn. is there one or two you would recommend that are good?

  • mynameislame

    Nanotechnology looks promising as well. They are talking within 20-30 years.

    Search for Nanotechnology live forever

    Better save your pennies though. I doubt it will be cheap.

  • snare&racket

    No easy answer really, journals and keeping an eye on reputable sources in different specialities. Real science is often boring and never shouted from the roof tops until proven. Hypothesis and hyperbole gets clicks and sells newspapers, so anywhere doing that usually has a different motive.

    All science papers end up in journals, some have s web presence but maybe get a subscription, Nature for example or New Scientist.

    For succinct fun science at regular updates, the facebook page called "I fucking love science" (pardon the name) is great and fun and references the new stories they signpost.

    But yeah, I use my monthly medical journal subscription, I may also pick up bits and bobs and test them out after seeing it on a news site, heard in a podcast etc. But I don't have one source perse. (Remember when Watchtower was our one source...kinda dangerous hey ;) jokes aside ...for the same reason it isn't best done with science.)

  • John_Mann

    There's already a gene therapy in the market called Glybera.

    It's the most expensive medicine in the world.

  • All or nothing
    All or nothing

    60 minutes tonight about crispr

  • Brokeback Watchtower
  • Vidiot

    One of the things about the 21st Century I like the most...

    ...advances in science and medicine that Biblical literalists - and especially End-Timers - all have been insisting could only occur miraculously in their post-apocalyptic utopia.

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