Navigation Links
One shot of gene therapy spreads through brain in animal study
Date:10/8/2007

By targeting a site in a mouse brain well connected to other areas, researchers successfully delivered a beneficial gene to the entire brainafter one injection of gene therapy. If these results in animals can be realized in people, researchers may have a potential method for gene therapy to treat a host of rare but devastating congenital human neurological disorders, such as Tay-Sachs disease.

Researchers from The Childrens Hospital of Philadelphia and the University of Pennsylvania reported their findings in the September 12 issue of the Journal of Neuroscience.

After a single injection, this technique succeeded in correcting diseased areas throughout the brain, said study leader John H. Wolfe, V.M.D., Ph.D., a neurology researcher at The Childrens Hospital of Philadelphia and a professor of pathology and medical genetics at the Penn School of Veterinary Medicine. This may represent a new strategy for treating genetic diseases of the central nervous system.

Wolfe and Penn graduate student Cassia N. Cearley performed the study in mice specially bred to have the neurogenetic disease mucopolysaccharidosis type VII (MPS VII). In people, MPS VII, also called Sly syndrome, is a rare, multisystem disease causing mental retardation and death in childhood or early adulthood.

Sly syndrome is one of a class of some 60 disorders called lysosomal storage diseases that collectively cause disabilities in about one in 5,000 births. Those diseases account for a significant share of childhood mental retardation and severe, often fatal, disabilities. In each of the lysosomal storage diseases, a defect in a specific gene disrupts the production of an enzyme that cleans up waste products from cells. Cellular debris builds up within cell storage sites called lysosomes, and the waste deposits interfere with basic cell functions. Other examples of lysosomal storage diseases are Tay-Sachs disease, Hunter disease and Pompe disease.

<
'/>"/>
Contact: John Ascenzi
Ascenzi@email.chop.edu
267-426-6055
Children's Hospital of Philadelphia
Source:Eurekalert

0

GOOD

Page: 1 2 3

Related medicine news :

1. Cancer Doctors Okays Controversial Prostate Therapy
2. Consensus on "Combination Therapy" for Breast Cancer
3. Acupuncture May Help Chemotherapy Side Effects
4. Antioxidants protect health tissue in people undergoing radiation therapy.
5. Distraction therapy found very effective in postoperative patients
6. Gene Therapy shows promise in treating Hemophilia
7. "Make AIDS Therapy affordable" - Physicians demand
8. Acupuncture superseeds massage therapy for short term relief of pain
9. Hormone replacement Therapy a headache
10. Simple Therapy
11. Ovarian cancer in relationship with Hormone therapy
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:12/17/2009)... 17, 2009) Researchers from the La Jolla Institute for ... the world,s most dangerous infectious diseases tuberculosis, malaria ... federally-funded set of projects seeking to make new inroads ... Institute received four project awards totaling $18.8 million from ... part of the National Institutes of Health, to fund ...
(Date:12/17/2009)... Stem cells that could one day provide therapeutic options ... from the tissue of the umbilical cord, just as ... to treat some blood disorders, said University of Pittsburgh ... of the Journal of Biomedicine and Biotechnology ... expanded to greater number, are remarkably stable and might ...
(Date:12/17/2009)... , , THURSDAY, Dec. 17 (HealthDay News) -- Among teenage ... least one of three common sexually transmitted infections -- ... becoming sexually active, a new study has found. , ... also found that it was common for these patients ... within four to six months after treatment of the ...
(Date:12/17/2009)... Therapure Biopharma to perform fill/finish of nimotuzumab , ... ... Wellner, President and CEO of Therapure Biopharma Inc. and David ... the signing of an agreement under which Therapure Biopharma will ... monoclonal antibody licensed to YM’s majority-owned Canadian subsidiary, CIMYM BioSciences ...
(Date:12/17/2009)... - Procedure Provides Efficient Mechanism to Pursue ... QC, Dec. 17 /PRNewswire-FirstCall/ - Labopharm Inc. ... announced it has initiated the regulatory approval ... Europe under the Decentralized Procedure (DCP). The ... a company to simultaneously pursue regulatory approval ...
Breaking Medicine News(10 mins):Health News:NIH awards La Jolla Institute $18.8 million for major infectious disease study 2Health News:NIH awards La Jolla Institute $18.8 million for major infectious disease study 3Health News:NIH awards La Jolla Institute $18.8 million for major infectious disease study 4Health News:NIH awards La Jolla Institute $18.8 million for major infectious disease study 5Health News:Umbilical cord could be new source of plentiful stem cells, say Pitt researchers 2Health News:STDs Common Among Sexually Active Teen Girls in Cities 2Health News:Therapure Biopharma Inc. and YM BioSciences Inc. Announce Partnership 2Health News:Therapure Biopharma Inc. and YM BioSciences Inc. Announce Partnership 3Health News:Therapure Biopharma Inc. and YM BioSciences Inc. Announce Partnership 4Health News:Labopharm Initiates Regulatory Approval Process for Twice-Daily Tramadol-Acetaminophen in Europe 2Health News:Labopharm Initiates Regulatory Approval Process for Twice-Daily Tramadol-Acetaminophen in Europe 3
... Students are protesting against the ban put on gay men ... Gordon University expressed the desire that they wanted the ban ... about gay men that are now defunct., ,As part ... young people to give blood “because I can’t”. ,Those ...
... The avian flu scare has changed the fortunes of the mutton ... severely hit in the past few days. They are under the ... red meat have gone up in a dramatic fashion. // ... late last year after chicken shop owners launched a malicious campaign ...
... has found that hormonal changes due to aging in older ... younger ones.Researchers at Vrije University Medical Centre in the Netherlands ... more than one egg, in the older women. // ... women and found multiple follicles - the sacs in the ...
... London – Investors and Financial Markets are simply at a ... on the financial market. // ,"We just don't ... Ip, a senior economist with Morley Fund Management in London. ... financial markets, are pretty clueless to its repercussions, clearly fueled ...
... key receptor that could be a potential target for drugs ... //The condition is sudden in onset and is a life-threatening ... triggers the sense of sight, smell and taste (G-protein coupled ... the body's own fluid gets trapped in the lungs and ...
... study researchers might be able to isolates molecule that ... ,The revolutionary study has been done independently by ... and they have identified a master protein that shows ... ,Science (Feb 17) features the two studies ...
Other Medicine News:Health News:Mutton And Beef Sellers Rule The Roost In Kerala 2
(Date:12/17/2009)... Medical, a privately-held aesthetic medical device company, today ... line of credit facility with Western Technology Investment ... be used to fund commercial support for Artefill®, ... the market today, as well as to expand ... Medical entered into a multi-million dollar line of ...
(Date:12/17/2009)... LOS ANGELES, Dec. 17 Instacare Corp. ... of life-saving and life-enhancing prescription drugs and diagnostics ... ill, and a leading developer and provider of ... and products announced today that as part of ... Board has determined to add two new Directors ...
(Date:12/17/2009)... (Nasdaq: ANDS ) today announced preliminary results from a ... first dose cohort, 200 mg bid, in an ongoing Phase ... ribavirin (SOC) in HCV patients. 56% of patients receiving ... at week 4, known as R apid V ... patients receiving placebo plus SOC. ,, ANA598 was ...
Breaking Medicine Technology:Suneva Medical Signs Multi-Million Dollar Line of Credit Agreement with Western Technology Investment 2Suneva Medical Signs Multi-Million Dollar Line of Credit Agreement with Western Technology Investment 3Instacare Corp. Board of Directors Sets Growth Plans for 2010 2ANA598 Demonstrates Positive 4-Week Results at 200 mg BID 2ANA598 Demonstrates Positive 4-Week Results at 200 mg BID 3ANA598 Demonstrates Positive 4-Week Results at 200 mg BID 4ANA598 Demonstrates Positive 4-Week Results at 200 mg BID 5ANA598 Demonstrates Positive 4-Week Results at 200 mg BID 6ANA598 Demonstrates Positive 4-Week Results at 200 mg BID 7Groups are key to good health 56765 1Groups are key to good health 56765 2Groups are key to good health 56765 3Energy experts helping Australian households reduce carbon emissions 9863 1Energy experts helping Australian households reduce carbon emissions 9863 2Precision Therapeutics Named to Inc Magazines List of Fastest Growing Companies 13983 1Precision Therapeutics Named to Inc Magazines List of Fastest Growing Companies 13983 2
Designed to fit a variety of 250 L and 300 ul multi-channel pipettors, this tip offers a secure fit and extra space for repeat pipetting....
Hydrophobic PE filter without self-sealing additives, permits sample recovery after accidental over-pipetting. All filters are sterile (to USP, DAB, Ph.Eur. standards) and PCR clean (free from human ...
... are ideal for PCR ... or for any work ... occur. They are designed ... infectious materials, labelled samples, ...
1000 L, Racked, Pre-sterilized, 100 tips/rack Recommended for 500 and 1000 L pipettes....
Medicine Products: