Navigation Links
NYP/Weill Cornell gene therapy clinical trial yields promising results for Batten disease
Date:5/13/2008

NEW YORK (May 13, 2008) -- Promising results from a team of NewYork-Presbyterian Hospital/Weill Cornell Medical Center physician-scientists show that gene therapy is both safe and effective at slowing the progression of Batten disease, or Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL), a rare, genetic, degenerative neurological disorder that usually becomes fatal in children by the age of 8 to 12.

The clinical trial found that the procedure -- which involves injecting a harmless gene-bearing virus into the brain -- was not only safe, but, on the average, significantly slowed the disease progression of the subjects tested. Neurological function was assessed using a rating scale throughout an 18-month follow-up period.

"The virus is used as a Trojan horse that houses and then delivers a healthy, functional gene into the cells of the brain," says lead author Dr. Ronald Crystal, chairman of the Department of Genetic Medicine and chief of the Division of Pulmonary and Critical Care Medicine at NewYork-Presbyterian Hospital/Weill Cornell Medical Center. "The genes are incorporated within the genetic material of the cells, which are then able to produce a protein that is deficient in Batten disease."

Dr. Crystal is a world leader and pioneer in the use of gene therapy to treat a number of genetic disorders and diseases.

The results are published in the May 13 online issue of Human Gene Therapy.

The gene in question -- CLN2 -- is mutated in children with the disease, causing a deficiency in the enzyme TTP-1, which is responsible for ridding cells of the central nervous system of waste materials. Small organelles within the cell, called lysosomes, become clogged with toxic material within the neurons of the brain.

"It's like the garbage man of the cell is not able to do its job," says Dr. Crystal. "The trash keeps getting backed up inside the cell until the cells can no longer function properly and then eve
'/>"/>

Contact: Andrew Klein
ank2017@med.cornell.edu
212-821-0560
New York- Presbyterian Hospital/Weill Cornell Medical Center/Weill Cornell Medical College
Source:Eurekalert

Page: 1 2

Related medicine news :

1. Weill Cornell Medical College Announces First Commencement of Its New Medical School Graduates in Doha, Qatar, May 8, 2008
2. Weill Cornell receives funding to study creation of new elder abuse center
3. Weill Cornell team identifies potential new drug targets against hormone-dependent breast cancer
4. $50 million gift to NewYork-Presbyterian Hospital/Weill Cornell Medical Center
5. By color-coding atoms, new Cornell electron microscope promises big advance in materials analysis
6. NewYork-Presbyterian/Weill Cornell creates world-class cancer center
7. Weill Cornell receives $2.4 million in grants from Gates Foundation to fight tuberculosis
8. White House awards Weill Cornells Bruce McCandliss highest honor for early career scientists
9. The latest about male infertility and testosterone from NewYork-Presbyterian/Weill Cornell
10. Healthy restaurants help make us fat, says a new Cornell study
11. NIH selects Weill Cornell Medical College to lead new NYC translational research collaboration
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:12/24/2014)... December 25, 2014 Today, UWDress.com, the famous ... sleeve wedding gowns, and launched a site-wide wedding gown promotion. ... sleeve wedding gowns in a more unexpected way, the collection ... are popular in the global market, and they are available ... UWDress.com are offered at discounted prices, up to 75% off; ...
(Date:12/24/2014)... Dennis Thompson HealthDay Reporter ... men who have abstained from sex for one year will be ... federal policy that would reverse a 31-year ban on donations from ... Drug Administration announced Tuesday its intention to release a new draft ... blood donations from gay men. The FDA is changing its ...
(Date:12/24/2014)... Researchers say they have used human embryonic stem cells ... While this had already been done using rodent ... types of cells -- called primordial germ cells -- ... to the team at the University of Cambridge in ... one of the earliest events during early mammalian development," ...
(Date:12/24/2014)... 2014 For chic ladies, new fashionable outfits ... part of their Holiday Wish List, Angeldress.co.uk has ... ways to accent ladies’ holiday wardrobe by launching a promotion ... all over the discount of 80% off, like a cheap ... ship. The company’s chic wedding dresses are among the hottest ...
(Date:12/24/2014)... Fort Lauderdale, FL (PRWEB) December 24, 2014 ... (“ACA”) these days. With the Republican Party now in control ... shakeup begins again for the Affordable Care Act. A lawsuit ... alleges that delays of certain provisions in the ACA have ... Congress. (1) Also, a Supreme Court hearing will take place ...
Breaking Medicine News(10 mins):Health News:Buy Cheap Long Sleeve Wedding Gowns At UWDress.com 2Health News:FDA to Lift Ban on Blood Donations by Gay Men 2Health News:FDA to Lift Ban on Blood Donations by Gay Men 3Health News:Angeldress.co.uk Starts Its Big Sales by Offering Various Kinds of Cheap Clothes and Fashion Accessories 2Health News:Steven Gerst MD Discusses How, Amid Political Opposition, Court Challenges, and a New Open Enrollment Period, the Affordable Care Act still Relies on Health Informatics 2Health News:Steven Gerst MD Discusses How, Amid Political Opposition, Court Challenges, and a New Open Enrollment Period, the Affordable Care Act still Relies on Health Informatics 3
... turning to mathematics to take the guesswork out of efforts ... restore damaged body parts will have enough blood and oxygen ... published mathematical model of tissue transfer, mathematicians have shown that ... selected for transfer from one part of the body to ...
... , PROVO, Utah, July 14 Nu Skin ... Board Blake Roney was honored yesterday with the International Stevie Award ... Business Awards. The International Business Awards are the only global, all-encompassing ... on the heels of the company,s largest revenue year ever. , ...
... NEW YORK, July 14 The Empire State ... Act, which creates a national, government-run health plan to compete ... a public plan would eliminate free market efficiency and reduce ... Empire State Healthcare Coalition (the "Coalition") is not-for-profit organization formed ...
... , , CHARLESTON, S.C., ... health care system is the time it takes for a promising ... University of South Carolina (MUSC) joins 39 previously funded academic medical ... time it takes to turn laboratory discoveries into treatments for patients, ...
... you choose to say "Thank You"? , , , ... proud." , "I get teary-eyed when I think of all ... , "Oorah! As a United States Marine Corp SSgt, I am ... say thank you to our soldiers. More than you know - thank you!" , ...
... FAIRFAX, Va.The 2009 Interventional Radiology Coding Users, Guide, ... and services, is now available in both book ... available by the Society of Interventional Radiology, a ... professionals dedicated to improving public health through disease ...
Cached Medicine News:Health News:Mathematics taking guesswork out of plastic surgery tissue transfer 2Health News:Mathematics taking guesswork out of plastic surgery tissue transfer 3Health News:Mathematics taking guesswork out of plastic surgery tissue transfer 4Health News:Nu Skin Chairman Recognized With 2009 International Award for Outstanding Business Performance 2Health News:Nu Skin Chairman Recognized With 2009 International Award for Outstanding Business Performance 3Health News:The Empire State Healthcare Coalition Voices Concern Over the Affordable Health Choices Act 2Health News:MUSC Earns $20 Million Award From National Institutes of Health 2Health News:MUSC Earns $20 Million Award From National Institutes of Health 3Health News:Econo Lodge Hotels Team with Strikeouts For Troops to Help Consumers Send Personal 'Thank You' Notes to U.S. Armed Forces 2Health News:Econo Lodge Hotels Team with Strikeouts For Troops to Help Consumers Send Personal 'Thank You' Notes to U.S. Armed Forces 3Health News:New: Interventional radiology coding users' guide 2
(Date:12/24/2014)... CHAPEL HILL, N.C. , Dec. 23, 2014 /PRNewswire/ ... marketplace success. As the key link between the medical ... will grow even more in the coming years as ... relationships. According to research by benchmarking ... and MSLs can gain an important edge is by ...
(Date:12/22/2014)... , Dec. 22, 2014 Apnea Sciences ( ... offices in Aliso Viejo , California.  ... to the tremendous growth and worldwide expansion, a new ... patented SnoreRx, the anti snore cure have exceeded our ... "We are now in 23 countries and will ...
(Date:12/22/2014)... --Research and Markets ( http://www.researchandmarkets.com/research/hmlfbq/investment ) ... Analysis of the US Medical Device Sector " ... http://photos.prnewswire.com/prnh/20130307/600769 This analysis ... identifies the key trends and rationale for undertaking ... and acquisitions. The study will be ...
Breaking Medicine Technology:Role of Medical Science Liaisons Evolving in Medical Device Sector 2Investment Analysis of the US Medical Device Sector 2
... 10, 2012 uVera Diagnostics has introduced the CR3 Keyless ... screening specimen cup provides several state-of-the-art features not available ... "This is revolutionary to the drug testing industry," said Rick ... designed for ease of use for both genders. The unique ...
...  - Agreement allows development for potential treatment of ... SHPGY ), the global specialty biopharmaceutical company, ... develop and market Resolor ® (prucalopride) in the ... N.V.  Terms of the agreement have not been disclosed. ...
Cached Medicine Technology:uVera Diagnostics Offers New Drug Screening Product 2Shire Acquires US Rights to Resolor(R) (prucalopride) 2Shire Acquires US Rights to Resolor(R) (prucalopride) 3Shire Acquires US Rights to Resolor(R) (prucalopride) 4
... the first intra-operative fluorescent imaging system ... the location of the coronary arteries ... coronary artery bypass procedures. ,The patented ... to view, record, replay, print, and ...
... When myocardial infarction is ... TROPT sensitive rapid assay allows ... marker troponin T from a ... qualitative result is available within ...
The Polygraf ID multi-parametric recorder works with POLYGRAM NET analysis software to create a unique interface that combines web-style navigation, graphical presentation and intuitive design to del...
... The White Sun light source offers the industry ... Kelvin color temperature at a lower price. A ... White Sun to be used with Olympus, Storz, ... light output LED and re-settable bulb timer. The ...
Medicine Products: