• HOME
  • NEWS
    • BIOENGINEERING
    • SCIENCE NEWS
  • EXPLORE
    • CAREER
      • Companies
      • Jobs
    • EVENTS
    • iGEM
      • News
      • Team
    • PHOTOS
    • VIDEO
    • WIKI
  • BLOG
  • COMMUNITY
    • FACEBOOK
    • FORUM
    • INSTAGRAM
    • TWITTER
  • CONTACT US
Saturday, March 6, 2021
BIOENGINEER.ORG
No Result
View All Result
  • Login
  • HOME
  • NEWS
    • BIOENGINEERING
    • SCIENCE NEWS
  • EXPLORE
    • CAREER
      • Companies
      • Jobs
        • Lecturer
        • PhD Studentship
        • Postdoc
        • Research Assistant
    • EVENTS
    • iGEM
      • News
      • Team
    • PHOTOS
    • VIDEO
    • WIKI
  • BLOG
  • COMMUNITY
    • FACEBOOK
    • FORUM
    • INSTAGRAM
    • TWITTER
  • CONTACT US
  • HOME
  • NEWS
    • BIOENGINEERING
    • SCIENCE NEWS
  • EXPLORE
    • CAREER
      • Companies
      • Jobs
        • Lecturer
        • PhD Studentship
        • Postdoc
        • Research Assistant
    • EVENTS
    • iGEM
      • News
      • Team
    • PHOTOS
    • VIDEO
    • WIKI
  • BLOG
  • COMMUNITY
    • FACEBOOK
    • FORUM
    • INSTAGRAM
    • TWITTER
  • CONTACT US
No Result
View All Result
Bioengineer.org
No Result
View All Result
Home NEWS Bioengineering

Erasing a genetic mutation

Bioengineer by Bioengineer
April 21, 2014
in Bioengineering, EDITOR'S CHOICE
1
Share on FacebookShare on TwitterShare on LinkedinShare on RedditShare on Telegram

Researchers reverses a liver disorder in mice by correcting a mutated gene.Using a new gene-editing system based on bacterial proteins, MIT researchers have cured mice of a rare liver disorder caused by a single genetic mutation.

Erasing a genetic mutation

Illustration: Christine Daniloff

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 Biotechnology paper.

“This work shows that CRISPR can be used successfully in adults, and also identifies several of the challenges that will need to be addressed moving forward to the development of human therapies,” says Charles Gersbach, an assistant professor of biomedical engineering at Duke University who was not part of the research team. “In particular, the authors note that the efficiency of gene editing will need to improve significantly to be relevant for most diseases and other delivery methods need to be explored to extend the approach to humans. Nevertheless, this work is an exciting first step to using modern gene-editing tools to correct the devastating genetic diseases for which there are currently no options for affected patients.”

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.

Wen Xue, a senior postdoc at the Koch Institute, is also a lead author of the paper. Other authors are Institute Professor Phillip Sharp; Tyler Jacks, director of the Koch Institute; postdoc Sidi Chen; senior postdoc Roman Bogorad; Eric Benedetti and Markus Grompe of the Oregon Stem Cell Center; and Victor Koteliansky of the Skolkovo Institute of Science and Technology.

The research was funded by the National Cancer Institute, the National Institutes of Health, and the Marie D. and Pierre Casimir-Lambert Fund.

Story Source:

The above story is based on materials provided by MIT News Office, Anne Trafton.

Share12Tweet8Share2ShareShareShare2

Related Posts

blank

Robo-fish

September 19, 2016
blank

Mice born from ‘tricked’ eggs

September 17, 2016

UCLA researchers use stem cells to grow 3-D lung-in-a-dish

September 16, 2016

Sixteen MIT grad students named Siebel Scholars for 2017

September 16, 2016

Leave a Reply Cancel reply

Your email address will not be published.

This site uses Akismet to reduce spam. Learn how your comment data is processed.

POPULAR NEWS

  • IMAGE

    Terahertz accelerates beyond 5G towards 6G

    668 shares
    Share 267 Tweet 167
  • People living with HIV face premature heart disease and barriers to care

    84 shares
    Share 34 Tweet 21
  • Global analysis suggests COVID-19 is seasonal

    39 shares
    Share 16 Tweet 10
  • HIV: an innovative therapeutic breakthrough to optimize the immune system

    36 shares
    Share 14 Tweet 9

About

We bring you the latest biotechnology news from best research centers and universities around the world. Check our website.

Follow us

Tags

Climate ChangecancerMaterialsCell BiologyChemistry/Physics/Materials SciencesBiologyTechnology/Engineering/Computer ScienceInfectious/Emerging DiseasesPublic HealthEcology/EnvironmentMedicine/HealthGenetics

Recent Posts

  • “Magic sand” might help us understand the physics of granular matter
  • Study reveals how egg cells get so big
  • Survey identifies factors in reducing clinical research coordinator turnover
  • New ‘split-drive’ system puts scientists in the (gene) driver seat
  • Contact Us

© 2019 Bioengineer.org - Biotechnology news by Science Magazine - Scienmag.

No Result
View All Result
  • Homepages
    • Home Page 1
    • Home Page 2
  • News
  • National
  • Business
  • Health
  • Lifestyle
  • Science

© 2019 Bioengineer.org - Biotechnology news by Science Magazine - Scienmag.

Welcome Back!

Login to your account below

Forgotten Password?

Create New Account!

Fill the forms below to register

All fields are required. Log In

Retrieve your password

Please enter your username or email address to reset your password.

Log In