Navigation Links
New gene editing method shows promising results for correcting muscular dystrophy
Date:8/14/2014

may get greater clinical benefit."

Although the genetic cause of DMD has been known for nearly 30 years, there are no treatments that can cure the condition. Duchenne muscular dystrophy breaks down muscle fibers and replaces them with fibrous and/or fatty tissue causing the muscle to gradually weaken.

DMD affects an estimated 1 in 3,600𔃄,000 male births in the United States, according to the Centers for Disease Control (CDC). Left untreated, those with DMD eventually require use of a wheelchair between age 8 and 11, and have a life expectancy of 25 years. Initial symptoms include difficulty running and jumping, and delays in speech development. DMD can be detected through high levels of a protein called creatine kinase as it leaks into the blood stream, and is confirmed by genetic testing.

Genome editing through the CRISPR/Cas9 system is not currently feasible in humans. However, it may be possible, through advancements in technology, to use this technique to develop therapies for DMD in the future, Dr. Olson said.

"At the moment, we still need to overcome technical challenges, in particular to find better ways to deliver CRISPR/Cas9 to the target tissue and to scale up," Dr. Olson said. "But in the future we might be able to use this technique therapeutically, for example to directly target and correct the mutation in muscle stem cells and muscle fibers."

Added Chengzu Long, a graduate student in the Olson lab: "We are working on a more clinically feasible method to correct mutations in adult tissues, and have already made some progress."

The research, published online in the journal Science, is the inaugural paper from UT Southwestern's newly established Hamon Center for Regenerative Science and Medicine, made possible earlier this year by a $10 million endowment gift from the Hamon Charitable Foundation. The Center's goal is to understand the basic mechanisms for tissue and organ formation, and then
'/>"/>

Contact: Russell Rian
russell.rian@utsouthwestern.edu
214-648-3404
UT Southwestern Medical Center
Source:Eurekalert  

Page: 1 2 3

Related biology news :

1. Rapid method of assembling new gene-editing tool could revolutionize genetic research
2. Programmable RNA complex could speed genome editing in the lab
3. Powerful gene-editing tool appears to cause off-target mutations in human cells
4. Unprecedented control of genome editing in flies promises insight into human development, disease
5. New method of DNA editing allows synthetic biologists to unlock secrets of a bacterial genome
6. Global regulator of mRNA editing found
7. New insight into an emerging genome-editing tool
8. Genome editing goes hi-fi
9. A new genome editing method brings the possibility of gene therapies closer to reality
10. Splice-switching oligonucleotide therapeutics is new method for editing gene transcript
11. New 3-D stem cell culture method published in JoVE
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
New gene editing method shows promising results for correcting muscular dystrophy
(Date:6/24/2015)... Calif. , June 24, 2015  Synaptics ... of human interface solutions, today announced that Sharp ... to provide secure authentication for its latest flagship ... reached more than 200 million shipments of its ... Sharp reinforces Synaptics, strength, scalability and leadership in ...
(Date:6/23/2015)... June 23, 2015   Valencell, a ... results of a recent study that illustrates its ... wrist during activity. In a study conducted at ... along with the Apple Watch against a chest ... rate during activity. The study demonstrated that Valencell,s ...
(Date:6/17/2015)... and HILDEN, Germany , June 17, 2015 ... Frankfurt Prime Standard: QIA) today launched new Investigator ® ... laboratories in the United States . The ... analyze multiple key genomic markers (short tandem repeats or STRs) ... Sensor to evaluate the quality of DNA in each sample, ...
Breaking Biology News(10 mins):Synaptics Brings Natural ID Technology to Sharp's Newest Flagship Smartphone 2Valencell Delivers Breakthrough Heart Rate Accuracy on the Wrist 2Valencell Delivers Breakthrough Heart Rate Accuracy on the Wrist 3QIAGEN Launches New Genetic Fingerprinting Kits for U.S. Forensic Labs 2QIAGEN Launches New Genetic Fingerprinting Kits for U.S. Forensic Labs 3QIAGEN Launches New Genetic Fingerprinting Kits for U.S. Forensic Labs 4QIAGEN Launches New Genetic Fingerprinting Kits for U.S. Forensic Labs 5
... It,s two inches long, grows on wood, and is shaped ... Phallus drewesii , has been discovered on the African island ... journal Mycologia . The mushroom is named after ... Sciences, and is described in the July/August issue by Professor ...
... - Psychologists at the University of Pennsylvania have identified ... which causes people to ignore vital, obvious information in ... the modern, evolved mind and may also play a ... gain. Researchers who focus on the cognitive aspects ...
... of researchers led by Dr. Jayakrishna Ambati at the University ... macular degeneration (AMD), the leading cause of blindness in older ... shows strong potential as a means for both the early ... were reported in an article published online Sunday by the ...
Cached Biology News:New species of phallus-shaped mushroom named after California Academy of Sciences scientist 2'Shortcuts' of the mind lead to miscalculations of weight and caloric intake, says Penn study 2'Shortcuts' of the mind lead to miscalculations of weight and caloric intake, says Penn study 3Major breakthrough in early detection and prevention of AMD 2Major breakthrough in early detection and prevention of AMD 3
(Date:7/2/2015)... ... July 02, 2015 , ... ... of surgical instruments in Orthopedics, announced today the acquisition of Turner Medical, ... largest provider of surgical instruments to the Spine Industry. The acquisition of Turner ...
(Date:7/1/2015)... BURLINGTON, Mass. , July 1, 2015 /PRNewswire/ ... three most frequently cited advantages of Otezla are ... favorable safety profile, as reported by surveyed rheumatologists. ... and launched for the treatment of active psoriatic ... . The current standard of care includes conventional ...
(Date:7/1/2015)... July 1, 2015   Neuraltus Pharmaceuticals, Inc. ... of groundbreaking drugs to treat neurodegenerative diseases such ... Robert G. Miller , M.D., Director of the ... California Pacific Medical Center in San Francisco, has been ... $1.5 million to help fund a Phase 2 ...
(Date:7/1/2015)... ... July 01, 2015 , ... A newly launched business ... to lower the barriers to innovation in life sciences R&D. The Pistoia Alliance President’s ... cash prizes of US$15,000 and a period of expert mentorship from a senior industry ...
Breaking Biology Technology:In'Tech Medical Acquires Turner Medical to Become a World Leader in Orthopedic Contract-Manufacturing 2In'Tech Medical Acquires Turner Medical to Become a World Leader in Orthopedic Contract-Manufacturing 3Surveyed Rheumatologists Report That Otezla's Oral Administration is its Single Greatest Advantage Over Currently Available Treatment Options 2Surveyed Rheumatologists Report That Otezla's Oral Administration is its Single Greatest Advantage Over Currently Available Treatment Options 3Investigator to Receive $1.5 Million Grant from the ALS Association to Help Fund Confirmatory Phase 2 Study of Neuraltus Pharmaceuticals' NP001 2Investigator to Receive $1.5 Million Grant from the ALS Association to Help Fund Confirmatory Phase 2 Study of Neuraltus Pharmaceuticals' NP001 3Investigator to Receive $1.5 Million Grant from the ALS Association to Help Fund Confirmatory Phase 2 Study of Neuraltus Pharmaceuticals' NP001 4Pistoia Alliance Launches Startup Challenge 2Pistoia Alliance Launches Startup Challenge 3
... a leading,provider of automated screening solutions for ... DPI, a contract research organization with,leading expertise ... utilize,Cellectricon,s Dynaflow(R)HT, a fully automated patch clamp ... capacity, particularly,for fast ligand-gated ion channels. , ...
... April 27 /PRNewswire-Asia-FirstCall/ -- China Biologic,Products, Inc. (OTC ... of the leading plasma-based pharmaceutical companies in the ... Company received an order,from one of the largest ... valued at $5.3 million to be sold under ...
... -- China Medical,Technologies, Inc. (the "Company") (Nasdaq: ... that develops, manufactures and markets advanced,in-vitro diagnostic products, ... its Bladder Cancer FISH Probe (the "Probe") from ... the People,s Republic of China. , ...
Cached Biology Technology:Cellectricon and BioFocus DPI Advance High Throughput ion Channel Drug Discovery 2China Biologic Products Receives $5.3 Million Order from Largest Biopharmaceutical Distributor in India 2China Biologic Products Receives $5.3 Million Order from Largest Biopharmaceutical Distributor in India 3
Goat polyclonal to Duffy / FY / DARC...
... The Centra-CL5R multi-purpose centrifuge offers ... a large assortment of rotor options, ... of laboratory applications., Maximum Versatility , ... 21,000 xg) angle rotors or high-volume (4 ...
... a fully licensed, high throughput, water bath thermal ... to 24 microplates. This equates to 9,216 reactions ... A robotic arm rapidly transfers a basket of ... ramping times that are considerably faster than those ...
Proliferin (C-14)...
Biology Products: