Navigation Links
Gene therapy treatment extends lives of mice with fatal disease, MU study finds
Date:7/16/2012

COLUMBIA, Mo. A team of University of Missouri researchers has found that introducing a missing gene into the central nervous system could help extend the lives of patients with Spinal Muscular Atrophy (SMA) the leading genetic cause of infantile death in the world.

SMA is a rare genetic disease that is inherited by one in 6,000 children who often die young because there is no cure. Children who inherit SMA are missing a gene that produces a protein which directs nerves in the spine to give commands to muscles.

The MU team, led by Christian Lorson, professor in the Department of Veterinary Pathobiology and the Department of Molecular Microbiology and Immunology, introduced the missing gene into mice born with SMA through two different methods: intravenously and directly into the mice's central nervous systems. While both methods were effective in extending the lives of the mice, Lorson found that introducing the missing gene directly into the central nervous system extended the lives of the mice longer.

"Typically, mice born with SMA only live five or six days, but by introducing the missing SMN gene into the mice's central nervous systems, we were able to extend their lives 10-25 days longer than SMA mice who go untreated," said Lorson, who works in the MU Bond Life Sciences Center and the College of Veterinary Medicine. "While this system is still not perfect, what our study did show is that the direct administration of the missing gene into the central nervous system provides some degree of rescue and a profound extension of survival."

There are several different types of SMA that appear in humans, depending on the age that symptoms begin to appear. Lorson believes that introducing the missing gene through the central nervous system is a way to potentially treat humans regardless of what SMA type they have.

"This is a treatment method that is very close to being a reality for human patients," Lorson said. "Cl
'/>"/>

Contact: Nathan Hurst
hurstn@missouri.edu
573-882-6217
University of Missouri-Columbia
Source:Eurekalert

Page: 1 2

Related medicine news :

1. KRAS gene mutation and amplification status affects sensitivity to antifolate therapy
2. Antibody therapy prevents gastrointestinal damage following radiation exposure in mice
3. Chemotherapy proves life-saving for some leukemia patients who fail induction therapy
4. Immunotherapy for elderly cancer patients finds new promise in drug combination
5. Therapy exploits addiction of leukemia cells
6. Intensity Modulated Radiation Therapy optimal for localized prostate cancer
7. Adding drug to standard chemotherapy provides no survival benefit for older lung cancer patients
8. Study Casts Doubt on Value of Pricey Prostate Cancer Therapy
9. 19th century therapy for Parkinsons disease may help patients today
10. New Psoriasis Drugs Not Much Better Than Standard Therapy, Study Finds
11. Xenotransplantation as a therapy for type 1 diabetes
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:7/11/2014)... 2014 Netmail, a ... its unique Netmail Platform, today announced that it ... (WPC), which will be held in Washington’s George ... During the show, Netmail will officially launch the ... that consolidates email security, archiving, encryption, eDiscovery, storage, ...
(Date:7/10/2014)... consuming a supplement of omega 3 fatty acids had ... fats and omega 6 fatty acids, according to Duke ... Annals of the Rheumatic Diseases (10.1136/annrheumdis-2014-205601) on July ... just obesity may contribute to worsening osteoarthritis. , ... significant role than mechanical factors in the link between ...
(Date:7/10/2014)... (PRWEB) July 10, 2014 One week ... Bangladesh Worker Safety announced today it will extend compensation ... four months’ wages for workers displaced as a result ... date, Alliance inspections have resulted in the closure of ... all five cases, raising immediate safety concerns. In May, ...
(Date:7/10/2014)... Best Drug Rehabilitation , which offers treatment ... during a stay in rehab can make a ... is successful, has launched an inspiring “Success Stories” ... , The new Success Stories section contains both ... Rehabilitation graduates who wanted to help others draw ...
(Date:7/10/2014)... widespread across the United States, due in large to ... of clinical data that will become available for research ... experts in healthcare have become increasingly focused on clinical ... purpose of gleaning insights that have the potential to ... that is known as big data. ...
Breaking Medicine News(10 mins):Health News:Netmail Heads to Washington, D.C. to Demonstrate New Release of Netmail 5.3 at WPC 2014 July 13-17, 2014 2Health News:Netmail Heads to Washington, D.C. to Demonstrate New Release of Netmail 5.3 at WPC 2014 July 13-17, 2014 3Health News:Omega 3 fatty acids lessen severity of osteoarthritis in mice 2Health News:Alliance for Bangladesh Worker Safety to Double Compensation for Displaced Factory Workers; Members Vote to Extend Wage Benefits from Two to up to Four Months 2Health News:Best Drug Rehabilitation Launches New Success Stories Section of Website 2Health News:Six cases where big data can reduce healthcare costs 2Health News:Six cases where big data can reduce healthcare costs 3Health News:Six cases where big data can reduce healthcare costs 4
... fever cases in Singapore, which is currently battling an outbreak, ... soar, the city-state's environment minister said Monday . ... told parliament there were 3,597 dengue fever cases in the ... in the same period last year. ,He said ...
... a.k.a. Mel B is getting back into shape for the ... birth to a baby girl ., ,The singer said that ... schedule has paid off really well., ,I've been working out ... off. I feel great," the Mirror quoted her, as saying., ...
... 'PizzaExpress' has been named the most vegan-friendly food chain by ... the animal rights group for its cheese-free pizzas, the Scotsman ... recognised by PETA for 'animal-friendly progress'. ,The ... the first restaurant was opened in Wardour Street in London ...
... flowing in two of China's longest rivers -- the Yangtze ... change linked to the contraction of wetlands, warned the ... at the Chinese Academy of Sciences (CAS) as saying after ... wetlands on the Tibetan Plateau had shrunk by over 10 ...
... for researchers have isolated a chemical called bisdemethoxycurcumin , ... found in turmeric root that may help the ... , ,Researchers state that the chemical may help the ... the plaques found in Alzheimers disease. ,, ,The ...
... minister in charge of womens affairs has announced that the federal ... . ,Minister for Women and Child Development Renuka Chowdhury ... who are thrown out of their homes due to their HIV ... could be self-reliant." , ...
Cached Medicine News:Health News:India Plans Hospices for HIV Positive Women 2
(Date:7/10/2014)... 10, 2014   LabStyle Innovations Corp . ... announced the appointment of Professor Richard B. ... Board of Directors. Prof. Stone brings ... to LabStyle as an entrepreneur, venture capitalist public, ... governance expert. He currently serves on the board ...
(Date:1/15/2014)... ANTONIO , Jan. 15, 2014 Most osteoporosis ... according to a recent online survey* sponsored by Mission ... among the National Osteoporosis Foundation online support community, revealed ... take medication that comes in a form other than ...
(Date:1/15/2014)... 2014  Celsion Corporation (the "Company") (NASDAQ: CLSN ... institutional investors to purchase an aggregate of approximately $15 ... registered direct offering, led by a dedicated health care ... agreements with these investors pursuant to which the Company ...
Breaking Medicine Technology:LabStyle Innovations Welcomes Richard B. Stone to its Board of Directors 2LabStyle Innovations Welcomes Richard B. Stone to its Board of Directors 3LabStyle Innovations Welcomes Richard B. Stone to its Board of Directors 4Mission Pharmacal Conducts Online Survey of Osteoporosis Patients 2Mission Pharmacal Conducts Online Survey of Osteoporosis Patients 3Celsion Corporation Announces $15 Million At-The-Market Registered Direct Offering 2Celsion Corporation Announces $15 Million At-The-Market Registered Direct Offering 3
... , , NEW YORK, Nov. 20 ... today contributing to a transformation in American healthcare through an ... clinical care and fundamentally bending the cost curve downward -- ... alike. , The company today explained how the elements in ...
... , ... , Reportlinker.com announces that a new market ... India Medical Equipment Market Analysis and Forecasts ... , Description , India Medical ...
Cached Medicine Technology:Medco Reforming Healthcare with 'Smarter Medicine'; Wired, Transformational Pharmacy Model Advances Clinical Quality and Lowers Cost 2Medco Reforming Healthcare with 'Smarter Medicine'; Wired, Transformational Pharmacy Model Advances Clinical Quality and Lowers Cost 3Medco Reforming Healthcare with 'Smarter Medicine'; Wired, Transformational Pharmacy Model Advances Clinical Quality and Lowers Cost 4Medco Reforming Healthcare with 'Smarter Medicine'; Wired, Transformational Pharmacy Model Advances Clinical Quality and Lowers Cost 5Reportlinker Adds India Medical Equipment Market Analysis and Forecasts to 2015 2Reportlinker Adds India Medical Equipment Market Analysis and Forecasts to 2015 3Reportlinker Adds India Medical Equipment Market Analysis and Forecasts to 2015 4Reportlinker Adds India Medical Equipment Market Analysis and Forecasts to 2015 5Reportlinker Adds India Medical Equipment Market Analysis and Forecasts to 2015 6Reportlinker Adds India Medical Equipment Market Analysis and Forecasts to 2015 7Reportlinker Adds India Medical Equipment Market Analysis and Forecasts to 2015 8Reportlinker Adds India Medical Equipment Market Analysis and Forecasts to 2015 9Reportlinker Adds India Medical Equipment Market Analysis and Forecasts to 2015 10Reportlinker Adds India Medical Equipment Market Analysis and Forecasts to 2015 11Reportlinker Adds India Medical Equipment Market Analysis and Forecasts to 2015 12Reportlinker Adds India Medical Equipment Market Analysis and Forecasts to 2015 13
... a completely automated instrument that offers Rapid ... 6 hour same day turnaround), random or ... It is used for bacterial and yeast ... and has a complete data management system. ...
IDS Rapid One, a microbial identification system....
The Cogent Microbial Luminescence System (MLS) is a superior Rapid microbial detection system for UHT product screening combining proven technology of ATP-bioluminescence with unsurpassed hardware in...
... SkanWasher 300 is an ... designed to give reliable, ... cross-contamination between wells, and ... to plate. The washer ...
Medicine Products: