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
'"/>

Source:Baylor College of Medicine


Page: 1 2

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:
TAG: Gene therapy cures inherited liver disease rats

(Date:12/11/2014)... --Research and Markets , ... ) has announced the addition of the ... to their offering. One major ... technology. With continuous advances in technology, it is ... standard that meets the needs and expectations of ...
(Date:12/10/2014)...  Valencell, a leader in performance biometric data sensor ... from its licensees for highly accurate, clinically validated biometric ... coming from fitness and health sectors, but first responders/military ... is only as useful as the biometric data it ... in long-term mass consumer adoption of wearable products," said ...
(Date:12/5/2014)... 2014 Increased emphasis on product quality, ... testing and inspection technologies. The convergence of industries ... range of innovative test solutions. This trend is ... which is generally more inclined towards communication, media ... strategies of test equipment vendors will cater to ...
Breaking Biology News(10 mins):Biometrics Market in the APAC Region 2015-2019: Key Vendors are 3M Cogent, NEC, Safran and Suprema 2Wearable Technology Products Demand Highly Accurate Biometric Technology 2Emerging Technologies Enable Smarter Monitoring of Assets, Push Industrial Testing, Inspection and Monitoring Market 2Emerging Technologies Enable Smarter Monitoring of Assets, Push Industrial Testing, Inspection and Monitoring Market 3Emerging Technologies Enable Smarter Monitoring of Assets, Push Industrial Testing, Inspection and Monitoring Market 4
... -- Weill Cornell Medical College and the Columbia University ... Mortimer D. Sackler, M.D. Prize for Distinguished Achievement in ... Dr. Fernando Nottebohm for his seminal work in songbirds ... Dr. Nottebohm is currently the Dorothea L. Leonhardt ...
... but in an infectious disease, it is insidious. Witness tuberculosis, ... before bursting forth into infection. TB,s stealthy nature has made ... to control it. The World Health Organization estimates that a ... a study led by Stanford scientists has provided new insights ...
... sends the human immune system into overdrive it can ... and thrive inside our digestive system. New research suggests ... significant role in triggering the innate immune system to stay ... in the face of an infection. But exactly how ...
Cached Biology News:Sackler Prize awarded to pioneering neuroscientist 2Sackler Prize awarded to pioneering neuroscientist 3Tuberculosis strain spread by the fur trade reveals stealthy approach of epidemics 2Tuberculosis strain spread by the fur trade reveals stealthy approach of epidemics 3Tuberculosis strain spread by the fur trade reveals stealthy approach of epidemics 4Stress wrecks intestinal bacteria, could keep immune system on idle 2
(Date:12/19/2014)... 2014 In contrast to traditional LED ... various colors, Valoya uses proprietary LED technology to incorporate ... possible uniformity which is critical in low proximity installations. ... accuracy throughout its lifetime which is up to four ... , "The investment cost was clearly higher when compared ...
(Date:12/19/2014)...  Decision Resources Group finds that the ... moderately through 2023 as the aging population, increasing adoption ... biomaterials all spur procedure volumes. Growth will be particularly ... and India , where ... for a larger proportion of the population. ...
(Date:12/19/2014)... , Dec. 18, 2014 Relmada Therapeutics, Inc. ... the treatment of chronic pain, announced the results of ... "This was a transformational year in Relmada,s history ... for our company," said Sergio Traversa , chief ... have executed on key financial, human resource and clinical ...
(Date:12/17/2014)... Bellingham, Washington, USA (PRWEB) December 16, 2014 ... Kirstin Baum, manager at Philipps- Universität Marburg’s Department ... optics and photonics, in the newly released ... 2015-2016. “And never be too shy to ask ... STEM (science, technology, engineering, and mathematics) occupations ranging ...
Breaking Biology Technology:Asia Pacific Dental Biomaterial Market Will Grow Strongly Through 2023 2Asia Pacific Dental Biomaterial Market Will Grow Strongly Through 2023 3Relmada Therapeutics Announces Results of Annual Shareholders Meeting 2Network, Multitask, Persevere: SPIE Women in Optics Planner Subjects Share from Experience 2Network, Multitask, Persevere: SPIE Women in Optics Planner Subjects Share from Experience 3
... California. , Dynal : 400+ employees worldwide, 87 in ... Norway. Brown Deer, Wis. California-based ... ASA , whose U.S. operations are based in Brown ... million. , ,Invitrogen has considered the acquisition since ...
... venture capital performance in the U.S. during 2004. , ,We ... share of this investment as well as how the life science ... detailed look at how the life science industry in the Midwest ... report. , ,Still, I was able to ferret out some ...
... written about the important impact that generic pharmaceutical drugs ... Their rise and growth spurs Big Pharma to invest ... Americans not having any kind of medical health insurance ... medicines from Canada being rapidly shut by U.S. pharmaceutical ...
Cached Biology Technology:Invitrogen to buy Norwegian biotech with operations in Brown Deer 2Midwest VC investment up in 2004, but number of deals down 2Midwest VC investment up in 2004, but number of deals down 3Pharmas will be forced into generic-drug strategy 2Pharmas will be forced into generic-drug strategy 3
... The Arabidopsis 2 Oligo Microarray Kit (V2), ... growth stages, and stress response. Each microarray contains ... of The Institute for Genomic Research (TIGR) and ... contains 5 standard 1 x 3? glass ...
... Successful biomarker discovery projects require screening ... to distinguish clinically relevant data from ... Data Manager is now available to ... tracking and data flood inherent to ...
... number is a new product ... Cornings product number. If showing ... under the old Sigma-Aldrich number ... for assistance. ID clarifier: reusable ...
... ,Product Information , ... high quality, general purpose shaker with economical analog ... models in the line, the Orbit LS features ... Electronic controls are utilized for setting speed and ...
Biology Products: