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posted by mrpg on Friday December 22 2017, @03:30PM   Printer-friendly
from the science-to-the-rescue dept.

First step toward CRISPR cure of Lou Gehrig's disease

University of California, Berkeley scientists have for the first time used CRISPR-Cas9 gene editing to disable a defective gene that causes amyotrophic lateral sclerosis, or Lou Gehrig's disease, in mice, extending their lifespan by 25 percent.

[...] The mice were genetically engineered to express a mutated human gene that in humans causes about 20 percent of all inherited forms of the disease and about 2 percent of all cases of ALS worldwide. Though the genetic cause is not known for all cases of ALS, all are accompanied by the premature death of motor neurons in the brain stem and spinal cord. The neurons allow the brain to control muscles, so loss of this connection means loss of muscle control.

[...] The UC Berkeley research team used a virus that Schaffer's team engineered to seek out only motor neurons in the spinal cord and deliver a gene encoding the Cas9 protein into the nucleus. There, the gene was translated into the Cas9 protein, a molecular scissors that cut and disabled the mutant gene responsible for ALS.

In this case, Cas9 was programmed to knock out the mutated gene SOD1 (superoxide dismutase 1). The onset or start of the disease was delayed by almost five weeks, and mice treated by the gene therapy lived about a month longer than the typical four-month lifespan of mice with ALS. Healthy mice can live a couple of years.

Lou Gehrig's disease = amyotrophic lateral sclerosis (ALS).

In vivo genome editing improves motor function and extends survival in a mouse model of ALS (open, DOI: 10.1126/sciadv.aar3952) (DX)

Previously: New Therapy Halts Progression of Lou Gehrig's Disease in Mice


Original Submission

Related Stories

New Therapy Halts Progression of Lou Gehrig's Disease in Mice 18 comments

Researchers at Oregon State University announced today that they have essentially stopped the progression of amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease, for nearly two years in one type of mouse model used to study the disease - allowing the mice to approach their normal lifespan.

The findings, scientists indicate, are some of the most compelling ever produced in the search for a therapy for ALS, a debilitating and fatal disease, and were just published in Neurobiology of Disease.

"We are shocked at how well this treatment can stop the progression of ALS," said Joseph Beckman, lead author on this study, a distinguished professor of biochemistry and biophysics in the College of Science at Oregon State University, and principal investigator and holder of the Burgess and Elizabeth Jamieson Chair in OSU's Linus Pauling Institute.

In decades of work, no treatment has been discovered for ALS that can do anything but prolong human survival less than a month. The mouse model used in this study is one that scientists believe may more closely resemble the human reaction to this treatment, which consists of a compound called copper-ATSM.

Copper delivery to the CNS by CuATSM effectively treats motor neuron disease in SODG93A mice co-expressing the copper-chaperone-for-SOD (DOI: 10.1016/j.nbd.2016.01.020)


Original Submission

2017: Gene Therapy's Milestone Year 4 comments

In a milestone year, gene therapy is finding a place in medicine

After decades of hope and high promise, this was the year scientists really showed they could doctor DNA to successfully treat diseases. Gene therapies to treat cancer and even pull off the biblical-sounding feat of helping the blind to see were approved by U.S. regulators, establishing gene manipulation as a new mode of medicine.

Almost 20 years ago, a teen's death in a gene experiment put a chill on what had been a field full of outsized expectations. Now, a series of jaw-dropping successes have renewed hopes that some one-time fixes of DNA, the chemical code that governs life, might turn out to be cures. "I am totally willing to use the 'C' word," said the National Institutes of Health's director, Dr. Francis Collins.

[...] The advent of gene editing — a more precise and long-lasting way to do gene therapy — may expand the number and types of diseases that can be treated. In November, California scientists tried editing a gene inside someone's body for the first time using a tool called zinc finger nucleases for a man with a metabolic disease. It's like a cut-and-paste operation to place a new gene in a specific spot. Tests of another editing tool called CRISPR to genetically alter human cells in the lab may start next year. "There are a few times in our lives when science astonishes us. This is one of those times," Dr. Matthew Porteus, a Stanford University gene editing expert, told a Senate panel discussing this technology last month.

Previously: Gene Therapy Cure for Sickle-Cell Disease
Gene Therapy to Kill Cancer Moves a Step Closer to Market
U.S. Human Embryo Editing Study Published
FDA Approves a Gene Therapy for the First Time
Gene Editing Without CRISPR -- Private Equity Raises $127 Million
FDA Committee Endorses Gene Therapy for a Form of Childhood Blindness
FDA Approves Gene Therapy for Non-Hodgkin's Lymphoma
Gene Therapy and Skin Grafting for Junctional Epidermolysis Bullosa
Gene Therapy for Spinal Muscular Atrophy Type 1
Biohackers Disregard FDA Warning on DIY Gene Therapy
CRISPR Used to Epigenetically Treat Diseases in Mice
Gene Therapy Showing Promise for Hemophilia B
Gene Therapy for Retinal Dystrophy Approved by the FDA
CRISPR Treatment for Some Inherited Forms of Lou Gehrig's Disease Tested in Mice


Original Submission

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  • (Score: 2) by aiwarrior on Friday December 22 2017, @03:40PM (4 children)

    by aiwarrior (1812) on Friday December 22 2017, @03:40PM (#613223) Journal

    Is it safe to say that all all known and identified genetic disorders will be curable in the near future?

    • (Score: 2) by fishybell on Friday December 22 2017, @04:25PM (1 child)

      by fishybell (3156) on Friday December 22 2017, @04:25PM (#613239)

      Will be curable? Probably.

      Will be cured? Definitely not. The perceived ethical quandaries will cause at least many parts of the world (cough, USA) to lag behind.

      • (Score: 2, Insightful) by Anonymous Coward on Friday December 22 2017, @06:17PM

        by Anonymous Coward on Friday December 22 2017, @06:17PM (#613271)
        > The perceived financial quandaries of cure vs. treat will cause at least many parts of the world (cough, USA) to lag behind.
    • (Score: 0) by Anonymous Coward on Friday December 22 2017, @04:35PM

      by Anonymous Coward on Friday December 22 2017, @04:35PM (#613242)

      No, not at all. You will see a continuing stream of press releases that imply that though. These people amazingly still do not use high school level scientific techniques:

      No blinding was used to perform measurements.
      [...]
      measurements were not performed in a blinded manner.
      [...]
      The mice were provided with wet mashed food in their cages at the first sign of hindlimb paralysis and were henceforth monitored daily.

      See, once they thought they saw a sign of disease ("first sign" is something not even defined in the paper), they started treating the mice differently. Also, they say motor function and survival were improved due to less motor neurons dying, but never show us the plot of # motor neurons vs survival. This is basic stuff.

      There is no reason to think this is anything more than "biased people report something that is likely to help their careers". Whatever, reading this junk is bad for my health, I need to stop.

    • (Score: 4, Funny) by takyon on Friday December 22 2017, @06:11PM

      by takyon (881) <reversethis-{gro ... s} {ta} {noykat}> on Friday December 22 2017, @06:11PM (#613268) Journal

      *In mice

      Mice have it made.

      --
      [SIG] 10/28/2017: Soylent Upgrade v14 [soylentnews.org]
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