Navigation Links
Erasing a genetic mutation
Date:3/30/2014

d 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.

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.
'/>"/>

Contact: Sarah McDonnell
s_mcd@mit.edu
617-253-8923
Massachusetts Institute of Technology
Source:Eurekalert

Page: 1 2 3

Related biology news :

1. Genetic mutations warn of skin cancer risk
2. Drilling into the trends in genetics and epigenetics of aging and longevity
3. Faster genetic testing method will likely transform care for patients with breast cancer
4. Genetics can explain why infections can trigger rheumatoid arthritis
5. Certain genetic variants may put bladder cancer patients at increased risk of recurrence
6. MRI reveals genetic activity
7. UT Southwestern ob/gyn researchers studying genetic factors in premature births
8. Researchers issue state-of-the-state on genetic-based testing & treatment for breast cancer
9. TGen-led study discovers genetic cause of rare type of ovarian cancer
10. Genetic testing may help select women with ER+ breast cancer for extended hormone therapy
11. Genetic test could improve colon cancer screening
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:8/22/2014)... , August 22, 2014 /PRNewswire/ ... http://www.researchandmarkets.com/research/mtm27l/biometrics_market ) has announced the addition ... America 2014-2018"  report to their offering.  ... , ,Biometrics is a technology that ... based on their physical or behavioral ...
(Date:8/22/2014)... leading source of information for businesses and professionals, has ... in its 2014 list of "some of the best ... the list, the company analyzed citation data over 11 ... had enormous impact. , The following UC Riverside researchers ... greatest number of highly cited papers in one of ...
(Date:8/22/2014)... Research presented at the 29th Annual International Papillomavirus ... Hera Therapeutics Inc. combats three types of human ... of all cervical cancer. , When tested in ... replication of HPV-16, HPV-18 and HPV-11 cells, according ... Ph.D., who presented the findings at the Seattle ...
Breaking Biology News(10 mins):Biometrics Market in North America 2014-2018: Key Vendors are 3M Cogent, Cross Match, NEC and Safran SA 2Biometrics Market in North America 2014-2018: Key Vendors are 3M Cogent, Cross Match, NEC and Safran SA 35 UC Riverside scientists among world's most influential scientific minds 2
... tap the vast, unexplored reaches of the earth,s microbial ... DOE Joint Genome Institute (DOE JGI) has announced its ... undertake in the coming year. The 44 projects, culled ... Program (CSP), represent over 60 billion nucleotides of data ...
... in Spanish . Most of the functions ... proteins, which recognise their binding partner by affinity features localized ... however, the most complicated to study from the perspective of ... interaction is brief and occurs through a large section of ...
... trials to find a successful vaccine against footrot in ... funding of $663,000 from Australian Wool Innovation (AWI) ... at Monash University,s Australian Research Council Centre of Excellence ... of Sydney. Chief investigator Professor Julian Rood said ...
Cached Biology News:US Department of Energy Joint Genome Institute announces new genome sequencing projects 2US Department of Energy Joint Genome Institute announces new genome sequencing projects 3US Department of Energy Joint Genome Institute announces new genome sequencing projects 4US Department of Energy Joint Genome Institute announces new genome sequencing projects 5US Department of Energy Joint Genome Institute announces new genome sequencing projects 6US Department of Energy Joint Genome Institute announces new genome sequencing projects 7Scientists reveal the key mechanisms for affinity between transient binding proteins 2Footrot vaccine closer than ever 2
(Date:8/22/2014)... August 22, 2014 Today the Academy of ... United States Court of Appeals for the D.C. Circuit challenging ... published in the Federal Register on June 25, 2014. ... contrary to the text and intent of the FAA Modernization ... Congress’ specific prohibition on any new rules or regulations regarding ...
(Date:8/22/2014)... BIOREM Inc. (TSXV: BRM) ("Biorem" or "the Company") today announced results for ... complete 2014 second quarter financial statements and MD&A have been filed on ... 30, , Six-months ended June 30, ( in CDN$,000 except ... , 2014 , 2013 Revenue , 2,459 ... profit , 56 , 1,485 , 385 ...
(Date:8/22/2014)... have discovered how the receptors responsible for contractions in ... new study, published today in The FASEB Journal ... regulates the cycle of all cells in the body. ... biological clock and its control are weakened in ageing ... physiology and ageing. , While currently much ...
(Date:8/22/2014)... (August 22nd), in the scientific journal PLoS ... establish the effect of serotonin on sensitivity to ... optical techniques., "Serotonin is a small molecule known ... brain functions, from the control of sleep and ... This neurotransmitter is also popularly thought to contribute ...
Breaking Biology Technology:AMA Files Petition for Review of FAA’s Interpretive Rule 2Biorem Reports 2014 Second Quarter Results 2Biorem Reports 2014 Second Quarter Results 3Treatment for overactive bladder and irritable bowel syndrome advanced through pioneering research 2From happiness to pain: Understanding serotonin's function 2
... Third Wave Technologies Inc. (Nasdaq: TWTI) today announced ... assays to RIKEN in Japan for use in various large ... Wave has been supplying its Invader SNP assays to Dr. ... Medicine at RIKEN, director of the Genome Center ...
... hit the road , ,MILWAUKEE - Ask any successful ... and you will get one answer: SALES. Despite this, sales ... in business school curricula today. What They Dont Teach You ... the Kellogg Alumni Club of Chicago titled Creating, Growing and ...
... between U.S. white collar workers and those in India is ... ,IT Manager ,U.S: $55,000 ,India: $8,500 , ,Accountant ,U.S.: $41,000 ... States were almost solely in the manufacturing sector, as more ... other Latin American countries to cut costs. Not to mention ...
Cached Biology Technology:Sales Strategies for the High-Tech Entrepreneur 2Sales Strategies for the High-Tech Entrepreneur 3Sales Strategies for the High-Tech Entrepreneur 4Sales Strategies for the High-Tech Entrepreneur 5Outsourcing Offshore - Wisconsin Companies Sending Work Abroad 2Outsourcing Offshore - Wisconsin Companies Sending Work Abroad 3
... • Small design specifically for western blots ... are specifically designed for staining western blots. ... 25 mm) of the disposable tray requires ... significant cost savings to the user. Pour ...
... pLivSelect is a direct ... recombinant identification. Recombinant selection ... survival, eliminating the need ... This quick and inexpensive ...
... Signal Enhancer does for enzyme-/substrate-based blotting what intensifying ... the signal up to 10-fold (or one order ... Western Blot Signal Enhancer membrane treatment is a ... to your current Western blotting protocol. The result ...
... through columns is a well-known separation technique ... applications are in DNA/RNA extraction in microbiology, ... purification in chemical or analytical laboratories. ... Genesis series instruments for smaller scales. The ...
Biology Products: