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."

###

Others who participated in this research include: Drs. Gabriele Toietta, Viraj P. Mane, Milton J. Finegold, Philip Ng, and Arthur L. Beaudet of BCM and Drs. Wilma S. Norona and Antony F. McDonagh of the University of California, San Francisco.


'"/>

Source:Baylor College of Medicine


Related biology news :

1. Adding Radiation Therapy To Chemotherapy Improves Survival In Patients With High-risk Breast Cancer
2. Columbia research lifts major hurdle to gene therapy for cancer
3. Combination therapy boosts effectiveness of telomere-directed cancer cell death
4. Gene therapy converts dead bone graft to new, living tissue
5. Study identifies predictors of HIV drug resistance in patients beginning triple therapy
6. New imaging method gives early indication if brain cancer therapy is effective, U-M study shows
7. Muscle-targeted gene therapy reverses rare muscular dystrophy in mice
8. New therapy for HIV/AIDS eliminates needles and excessive toxicity
9. New Treatment Rivals Chemotherapy For Lymphoma, Study Finds
10. Gene therapy for Parkinsons disease moves forward in animals
11. Pulsating ultrasound enhances gene therapy for tumors
Post Your Comments:
*Name:
*Comment:
*Email:


(Date:4/13/2016)... physicians supporting Medicaid patients in Central Florida ... telehealth thanks to a new partnership with higi.   ... can routinely track key health measurements, such as blood ... they opt in, share them with IMPOWER clinicians through ... location at no cost. By leveraging this data, IMPOWER ...
(Date:3/22/2016)... 22, 2016 According ... Market for Consumer Industry by Type (Image, Motion, ... (Communication & IT, Entertainment, Home Appliances, & ... to 2022", published by MarketsandMarkets, the market ... reach USD 26.76 Billion by 2022, at ...
(Date:3/15/2016)... York , March 15, 2016 ... market report published by Transparency Market Research "Digital Door Lock ... and Forecast 2015 - 2023," the global digital door lock ... 731.9 Mn in 2014 and is forecast to grow at ... Growth of micro, small and medium enterprises (MSMEs) across the ...
Breaking Biology News(10 mins):
(Date:5/3/2016)... MA (PRWEB) , ... May 03, 2016 , ... ... that facilitates accessibility to unique bioresearch materials from laboratories across the globe, today ... researchers scramble to increase the pace of research toward treatment and prevention measures ...
(Date:5/3/2016)... , ... May 03, 2016 , ... ... fertility clinics and IVF laboratories. A contingency of reproductive endocrinologists, including Dr. ... and women experiencing infertility and to help them build families. , Ovation Fertility ...
(Date:4/29/2016)... ... , ... The MIT bioLogic design team has won multiple A' Design ... can be applied to fabric and formed into living interfaces between body and environment. ... change. The team harvested Natto cells and applied them to fabric with custom 3D ...
(Date:4/29/2016)... CA (PRWEB) , ... April 29, 2016 , ... Summit ... (NSCF) to support the development of a patient-specific stem cell therapy for the treatment ... the lab of Dr. Jeanne Loring at The Scripps Research Institute in San Diego, ...
Breaking Biology Technology: