Navigation Links
'Smart' genetic therapy helps the body to heal itself

New approaches to genetic disease, based on cells' own ability to correct themselves, will be outlined today (Monday 8 May 2006) at the annual conference of the European Society of Human Genetics in Amsterdam, The Netherlands. Instead of replacing a faulty gene (commonly referred to as gene therapy) these new 'smart' approaches make use of the therapeutic potential that already exists in human biology.

Professor Brunhilde Wirth, from the Institute of Human Genetics, University of Cologne, Germany, will describe to the conference her team's work on developing therapies for patients with spinal muscular atrophy (SMA). This is a relatively common inherited disease in humans and the leading cause of death in infants, with an incidence of about 1 in every 6,000 newborns. Due to degeneration of the motor neurons in the spinal cord patients develop muscle weakness and atrophy of the legs, arms and trunk.

In patients with SMA the survival motor neuron genes (SMN1) is deleted, but they all carry a copy gene (SMN2). However, this only produces about 10% of the correct protein; insufficient to prevent the diseases. The severity of the SMA is influenced by the number of SMN2 genes, which normally vary between one and four ?the more copies there are the better the patient does.

Professor Wirth's team identified a drug ?valproate, used successfully in epilepsy treatment for decades ?that is able to increase SMN protein levels 2-4 fold in cell lines derived from SMA patients. Later, they showed that valproate also raised SMN levels in neuronal tissues such as cultured brain slices derived from epilepsy patients after surgery, as well as motor neuron cultures derived from rat embryos.

"The next step was to try this therapy in patients", said Professor Wirth. "In a first pilot trial, we enrolled ten parents of SMA patients who were treated with valproate for four months. We saw that valproate significantly increased SMN levels in blo
'"/>

Source:European Society of Human Genetics


Page: 1 2 3 4

Related biology news :

1. Smart nanoprobes light up disease
2. Its not all genetic: Common epigenetic problem doubles cancer risk in mice
3. Scientists detect probable genetic cause of some Parkinsons disease cases
4. Mouse brain tumors mimic those in human genetic disorder
5. Scientists identify genetic pathways essential to RNA interference
6. Researchers trace evolution to relatively simple genetic changes
7. Key mechanism in genetic inheritance during cell division identified
8. Scientists identify genetic pathways essential to RNA interference
9. MUHC scientists describe genetic resistance to rampant virus
10. Mosaic mouse technique offers a powerful new tool to study diseases and genetics
11. Montreal researchers probe the genetic basis of memory
Post Your Comments:
*Name:
*Comment:
*Email:
TAG: Smart genetic therapy helps the body heal itself

(Date:2/24/2015)... Research and Markets ( http://www.researchandmarkets.com/research/sxwl43/global_2d_gesture ... "Global 2D Gesture Recognition Market 2015-2019" report ... forecast the Global 2D Gesture Recognition market to ... period 2014-2019 The increased demand for ... of the major trends in the market. The ...
(Date:2/19/2015)... .19, 2015 Research and Markets ... the "Military Electro-Optical / Infrared Systems Market ... Platform - Forecast to 2020" report to ... systems market is expected to reach $16.35 billion ... This report segments the military electro-optical/infrared ...
(Date:2/13/2015)... 2015 ACT Genomics Co., Ltd., a ... transform cancer genomic information into precision diagnosis and ... raised US$ 8 million in the its first private ... Taipei, Taiwan , ACT Genomics has developed ... aim to implement next generation sequencing (NGS) and ...
Breaking Biology News(10 mins):Global 2D Gesture Recognition Market 2015-2019 with Cognitec Systems, CogniVue, eyeSight Mobile Technologies & PointGrab Dominating 2Military Electro-Optical / Infrared Systems (Image Intensification, Laser, Infrared) Market - Forecast to 2020: Featuring Profiles of the Market Leaders on this $16 Billion market 2Military Electro-Optical / Infrared Systems (Image Intensification, Laser, Infrared) Market - Forecast to 2020: Featuring Profiles of the Market Leaders on this $16 Billion market 3ACT Genomics Raises $8 Million in its First Private Funding Round 2
... Dates in Press Release, TAMPA, Fla., Feb. ... a leader in electro-stun technologies announced,today that as ... Taser International (Nasdaq: TASR ) has noticed ... Systems, Inc. (Federal Case,CV07-0042-PHX-MHM) of Taser,s intent to ...
... interested in natural history but it also challenges those people ... life on Earth. Thats the view of Dr Robert Lambert, ... of Nottingham, who has just returned from the Antarctic in ... Antarctica Tour Operators (IAATO). Dr Lambert, who is a ...
... University of Missouri scientist Ray Semlitsch studies creatures most ... at night and thrive in the shallow, cool, wet ... A collaborative study, with MU graduate student Bill Peterman, ... biomass (total mass of an organism in an area) ...
Cached Biology News:Taser Notices Intent to Serve Subpoena for Information on Old NIJ Tests in Taser v. Stinger 2Taser Notices Intent to Serve Subpoena for Information on Old NIJ Tests in Taser v. Stinger 3Observing sustainable tourism in Antarctica 2Observing sustainable tourism in Antarctica 3Salamanders, headwater streams critical in food chain 2
(Date:3/3/2015)... 03, 2015 Crown Bioscience, Inc., a ... launched a new Life Science division specializing in in ... , The first products released include in vivo ... soon to be released in vivo grade monoclonal antibodies ... expand their life science portfolio with additional antibody products ...
(Date:3/3/2015)... 3, 2015  RegeneRx Biopharmaceuticals, Inc. (OTCQB: RGRX) ("the ... journal, Expert Opinion on Biological Therapy , Dr. ... Zhenggang Zhang , at the Henry Ford Hospital in ... beta 4 (TB4) has the capacity to promote CNS ... more post-injury, leading to neurological recovery in each case.  ...
(Date:3/3/2015)... New Jersey (PRWEB) March 03, 2015 ... has announced today that it has acquired The ... Terms of the transaction were not announced. , ... services to the energy sector including oil and ... energy, and fossil and nuclear power sectors. The ...
(Date:3/3/2015)... Research and Markets ( http://www.researchandmarkets.com/research/gxqhg9/gene_therapy ... new report "Gene Therapy - Technologies, Markets ... Gene therapy technologies are described in ... therapy with genetically modified vectors. Gene therapy is ... routes of administration as well as targeted gene ...
Breaking Biology Technology:Crown Bioscience Launches Life Science Product Catalog 2Researchers at Henry Ford Hospital Propose Thymosin Beta 4 as Novel Way to Treat Neural Injury and Neurodegenerative Diseases 2Researchers at Henry Ford Hospital Propose Thymosin Beta 4 as Novel Way to Treat Neural Injury and Neurodegenerative Diseases 3Gene Therapy Market Report 2014-2024 - Technologies, Markets and Companies 2Gene Therapy Market Report 2014-2024 - Technologies, Markets and Companies 3
... Bay, Wis. - Whenever the title executive vice president ... an established part of management. So it is with Judy ... National following a career that she thought was winding ... Green Bay-based provider of global transportation, logistics, and intermodal services, ...
... students are no longer interested in technology careers? , ... Area Technical College has won first place in ... beating four teams from Minnesota, and later advanced to ... third place. , ,The competitions were open to students ...
... a series of articles on raising capital from investors. This installment ... the time when raising equity capital, the offering terms are summarized ... When dealing with angel investors , it is typical ... prospective angel investors will want to negotiate the initial term sheet, ...
Cached Biology Technology:CIO Leadership Series: Judy Lemke, Schneider National 2CIO Leadership Series: Judy Lemke, Schneider National 3CIO Leadership Series: Judy Lemke, Schneider National 4CIO Leadership Series: Judy Lemke, Schneider National 5Capital-raising term sheets for angels and venture capitalists 2Capital-raising term sheets for angels and venture capitalists 3Capital-raising term sheets for angels and venture capitalists 4Capital-raising term sheets for angels and venture capitalists 5