Corporate Banner
Satellite Banner
Technology
Networks
Scientific Communities
 
Become a Member | Sign in
Home>News>This Article
  News
Return

Gene Repair Technique Could Have Many Applications

Published: Tuesday, August 13, 2013
Last Updated: Tuesday, August 13, 2013
Bookmark and Share
Using human pluripotent stem cells and DNA-cutting protein from meningitis bacteria, researchers have created an efficient way to target and repair defective genes.

Published today in the Proceedings of the National Academy of Sciences, the team's findings demonstrate that the novel technique is much simpler than previous methods and establishes the groundwork for major advances in regenerative medicine, drug screening, and biomedical research.

Principal investigator James A. Thomson, co-director of biology at UCSB's Center for Stem Cell Biology and Engineering and professor in the campus's Department of Molecular, Cellular and Developmental Biology, said the discovery holds many practical applications, including paving a new route for correcting genetic disorders. Thomson is also director of regenerative biology at the Morgridge Institute, serves as the James Kress Professor of Embryonic Stem Cell Biology at the University of Wisconsin–Madison, and is a John D. MacArthur professor at UW–Madison's School of Medicine and Public Health.

According to the paper's lead author, Zhonggang Hou of the Morgridge Institute's regenerative biology team, the technique has the potential to repair any genetic defect, including those responsible for some forms of breast cancer, Parkinson's, and other diseases. "The fact that it can be applied to human pluripotent stem cells opens the door for meaningful therapeutic applications," said Hou.

The research team focused on Neisseria meningitidis bacteria because it is a good source of the Cas9 protein needed for precisely cleaving damaged sections of DNA. Using different types of small RNA molecules, the research team was able to guide this protein, engendering the careful removal, replacement, or correction of problem genes. "This represents a step forward from other recent technologies built upon proteins, such as zinc finger nucleases and transcription activator-like effector nucleases," said Yan Zhang of Northwestern University, second author of the paper.

These previous gene correction methods required engineered proteins to help with the cutting. The researchers said scientists can synthesize RNA for the new process in as little as one to three days, compared with the weeks or months needed to engineer suitable proteins.

"Human pluripotent stem cells can proliferate indefinitely and they give rise to virtually all human cell types, making them invaluable for regenerative medicine, drug screening, and biomedical research," Thomson said. "This collaboration has taken us further toward realizing the full potential of these cells because we can now manipulate their genomes in a precise, efficient manner."

Erik Sontheimer, another principal investigator and the Soretta and Henry Shapiro Research Professor of Molecular Biology in Northwestern's department of molecular biosciences, Center for Genetic Medicine, and the Robert H. Lurie Comprehensive Cancer Center of Northwestern University, said the team's results also offer hopeful signs about the safety of the technique.

"A major concern with previous methods involved inadvertent or off-target cleaving, raising issues about the potential impact in regenerative medicine applications," said Sontheimer. "Beyond overcoming the safety obstacles, the system's ease of use will make what was once considered a difficult project into a routine laboratory technique, catalyzing future research."

Also contributing to the study, which was supported by funding from the National Institutes of Health, the Wynn Foundation, and the Morgridge Institute for Research, were Nicholas Propson, Sara Howden, and Li-Fang Chu from the Morgridge Institute for Research.


Further Information
Access to this exclusive content is for Technology Networks Premium members only.

Join Technology Networks Premium for free access to:

  • Exclusive articles
  • Presentations from international conferences
  • Over 2,500+ scientific posters on ePosters
  • More Than 3,700+ scientific videos on LabTube
  • 35 community eNewsletters


Sign In



Forgotten your details? Click Here
If you are not a member you can join here

*Please note: By logging into TechnologyNetworks.com you agree to accept the use of cookies. To find out more about the cookies we use and how to delete them, see our privacy policy.

Related Content

Ultrafast DNA Diagnostics
New technology developed by UC Berkeley bioengineers promises to make a workhorse lab tool cheaper, more portable and many times faster by accelerating the heating and cooling of genetic samples with the switch of a light.
Monday, August 03, 2015
Scientists Create CRISPR/Cas9 Knock-In Mutations in Human T Cells
In a project spearheaded by investigators at UC San Francisco, scientists have devised a new strategy to precisely modify human T cells using the genome-editing system known as CRISPR/Cas9.
Tuesday, July 28, 2015
Simple Technology Makes CRISPR Gene Editing Cheaper
University of California, Berkeley, researchers have discovered a much cheaper and easier way to target a hot new gene editing tool, CRISPR-Cas9, to cut or label DNA.
Friday, July 24, 2015
Printed "Smart Cap" Detects Spoiled Food
It might not be long before consumers can just hit “print” to create an electronic circuit or wireless sensor in the comfort of their homes.
Tuesday, July 21, 2015
Growing Spinal Disc Tissue
Scientists develop new method for growing spinal disc tissue in the lab for combating chronic back pain.
Friday, July 03, 2015
Delivering Drugs to the Right Place
Thomas Weimbs has developed a targeted drug delivery method that could potentially slow the progression of polycystic kidney disease.
Monday, June 29, 2015
The Deep Carbon Cycle
Over billions of years, the total carbon content of the outer part of the Earth—in its upper mantle, crust, oceans and atmospheres—has gradually increased, scientists report.
Tuesday, June 23, 2015
Designing New Pain Relief Drugs
Researchers have identified the molecular interactions that allow capsaicin to activate the body’s primary receptor for sensing heat and pain, paving the way for the design of more selective and effective drugs to relieve pain.
Thursday, June 11, 2015
Engineers Crack DNA Code of Autoimmune Disorders
Researchers have identified an unexpectedly general set of rules that determine which molecules can cause the immune system to become vulnerable to the autoimmune disorders lupus and psoriasis.
Wednesday, June 10, 2015
Genetic Markers for Detecting and Treating Ovarian Cancer
Custom bioinformatics algorithm identifies human mRNAs that distinguish ovarian cancer cells from normal cells and provide new therapeutic targets
Wednesday, May 27, 2015
Researchers Reverse Bacterial Resistance to Antibiotics
Evidence continues to surface that supports the premise that antibiotics which have been out of use could still be effective in treating drug-resistant bacteria.
Friday, May 08, 2015
Industry-Sponsored Academic Inventions Spur Increased Innovation
Analysis questions assumption that corporate support skews science toward inventions that are less useful than those funded by the government or non-profit organizations.
Monday, March 24, 2014
May the Cellular Force be With You
Like tiny construction workers, cells sculpt embryonic tissues and organs in 3D space.
Friday, December 13, 2013
Grant Supports Creation of Patient-Derived Stem Cell Lines
Researchers have received a two-year, $600,000 grant from the National Institute on Aging to develop and study patient-derived stem cell lines.
Thursday, December 12, 2013
Prostate Cancer Stem Cells are a Moving Target
Researchers have discovered how prostate cancer stem cells evolve as the disease progresses, a finding that could help point the way to more highly targeted therapies.
Friday, December 06, 2013
Scientific News
The Changing Tides of the In Vitro Diagnostics Market
With the increasing focus in personalized medicine, diagnostics plays a crucial role in patient monitoring.
LaVision BioTec Reports on the Neuro Research on the Human Brain After Trauma
Company reports on the work of Dr Ali Ertürk from the Institute for Stroke and Dementia Research at LMU Munich.
NIH Study Shows No Benefit of Omega-3 Supplements for Cognitive Decline
Research was published in the Journal of the American Medical Association.
Less May Be More in Slowing Cholera Epidemics
Mathematical model shows more cases may be prevented and more lives saved when using one dose of cholera vaccine instead of recommended two doses.
Investigating the Vape
Expert independent review concludes that e-cigarettes have potential to help smokers quit.
NIH Launches Human RSV Study
Study aims to understand infection in healthy adults to aid development of RSV medicines, vaccines.
Researchers Discover Synthesis of a New Nanomaterial
Interdisciplinary team creates biocomposite for first time using physiological conditions.
Poor Survival Rates in Leukemia Linked to Persistent Genetic Mutations
For patients with an often-deadly form of leukemia, new research suggests that lingering cancer-related mutations – detected after initial treatment with chemotherapy – are associated with an increased risk of relapse and poor survival.
Flu Remedies Help Combat E. coli Bacteria
Physiologists from the University of Zurich have now discovered why the intestinal bacterium Escherichia coli (E. coli) multiplies heavily and has an inflammatory effect.
Marijuana Genome Unraveled
A study by Canadian researchers is providing a clearer picture of the evolutionary history and genetic organization of cannabis, a step that could have agricultural, medical and legal implications for this valuable crop.
Scroll Up
Scroll Down
Skyscraper Banner

Skyscraper Banner
Go to LabTube
Go to eposters
 
Access to the latest scientific news
Exclusive articles
Upload and share your posters on ePosters
Latest presentations and webinars
View a library of 1,800+ scientific and medical posters
2,500+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
3,700+ scientific videos
Close
Premium CrownJOIN TECHNOLOGY NETWORKS PREMIUM FREE!