Navigation Links
Gene Therapy Cures Inherited Liver Disease In Rats

A single dose of gene-virus combination cured rats of a inherited liver disease in which lack of a gene causes the accumulation of bilirubin –which, untreated, results in jaundice and brain damage, said researchers at Baylor College of Medicine in a report in the Proceedings of the Natural Academy of Sciences.

"This is the first time this disease (Crigler-Najjar syndrome) has been completely cured long term with a single injection in an adult animal," said Dr. Brendan Lee, associate professor of molecular and human genetics and a Howard Hughes Medical Investigator at Baylor College of Medicine.

Crigler-Najjar syndrome is currently treated by placing the person under special UV lights. It is an unwieldy and time-consuming treatment.

While Lee does not think that cure would be possible in all adults with this technique, he does think it poses a real promise for long-term alleviations of the toxic symptoms of these kinds of diseases. The treatment could be repeated when needed.

The treatment used a specially developed adenovirus to carry the gene into the animal's cells. This viral vector, as it is called, was manipulated so that it minimized toxic side effects. This disease involved a lack of a gene that is found in the part of the cell where detoxification occurs. It points the way to using gene therapy to cure and/or treat a host of other disease that occur because of the lack of genes in this area of the cell.

The viral vector itself is important because it has no long term effect. It does not become part of the genetic machinery of the cell and poses no risk of causing cancer, said Lee.

"This approach would be applicable to many diseases where you are trying to put something back, targeting the liver," he said.

He is hesitant to predict when the treatment can be tried in humans. While the viral vector is fairly benign long term, he would like to see one developed that minimizes the body's immune response to it immediately right after it is injected into the body.

"Fifteen years ago, the goal was to get some correction of the problem," he said. "Most effects were transient. Now with these vectors, long-term correction with no long-term toxicity is possible. We need to find a way to avoid short term toxicity."


'"/>

Source:Baylor College Of Medicine


Related biology news :

1. Adding Radiation Therapy To Chemotherapy Improves Survival In Patients With High-risk Breast Cancer
2. Novel Therapy Tested in Mice Could Chase Away Cat Allergies
3. Gene Therapy For Parkinsons Disease Moves Forward In Animals
4. Rush Physicians Using Gene Therapy For Heart Patients With Moderate To Severe Chest Pains Who Do Not Benefit From Other Treatments
5. Pathogen-Mimicking Vaccine As Strategy For Cancer Therapy
6. Discovery Promises Simpler Therapy for Sickle Cell Disease
7. Antiretroviral Therapy May Prevent HIV Transmission From Breastfeeding Mothers To Infants
8. HIV Therapy Greatly Extends Life of Key T Cells, Scientists Find
9. Menopause Symptoms May Come Back After Stopping Menopausal Hormone Therapy
10. Newly Discovered Role for Heart Response Enzyme May Yield Better Heart Failure Therapy
11. Finding Cures For Tropical Diseases: Is Open Source An Answer?
Post Your Comments:
*Name:
*Comment:
*Email:


(Date:6/22/2016)... 2016 On Monday, the Department of Homeland ... share solutions for the Biometric Exit Program. The Request ... Protection (CBP), explains that CBP intends to add biometrics ... the United States , in order to deter ... Logo - http://photos.prnewswire.com/prnh/20160622/382209LOGO ...
(Date:6/16/2016)... FRANCISCO , June 16, 2016 ... size is expected to reach USD 1.83 ... by Grand View Research, Inc. Technological proliferation and ... banking applications are expected to drive the market ... ) , The development of advanced ...
(Date:6/9/2016)... -- Paris Police Prefecture ... to ensure the safety of people and operations in several ... tournament Teleste, an international technology group specialised in ... that its video security solution will be utilised by ... safety across the country. The system roll-out is scheduled for ...
Breaking Biology News(10 mins):
(Date:6/24/2016)... Raleigh, NC (PRWEB) , ... June 24, 2016 , ... ... find the most commonly-identified miRNAs in people with peritoneal or pleural mesothelioma. Their findings ... here to read it now. , Diagnostic biomarkers are signposts in the blood, ...
(Date:6/23/2016)... , June 23, 2016 A person commits a ... crime scene to track the criminal down. An ... Food and Drug Administration (FDA) uses DNA evidence to track ... Sound far-fetched? It,s not. The FDA has increasingly ... support investigations of foodborne illnesses. Put as simply as possible, ...
(Date:6/23/2016)... --  EpiBiome , a precision microbiome engineering company, today ... from Silicon Valley Bank (SVB). The financing will allow ... drug development efforts, as well as purchase additional lab ... been an incredible strategic partner to us – one ... provide," said Dr. Aeron Tynes Hammack , EpiBiome,s ...
(Date:6/23/2016)... NEWPORT BEACH, Calif. , June 23, 2016 /PRNewswire/ ... offering new biological discoveries to the medical community, has ... and co-founder Matthew Nunez . "We ... provide us with the capital we need to meet ... funding will essentially provide us the runway to complete ...
Breaking Biology Technology: