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

ntually die throughout the entire brain."

When this happens, children with the disease begin exhibiting neurological symptoms, starting around age 4, including impaired muscle coordination (ataxia), involuntary twitching (myoclonus), and speech and developmental disorders. A gradual decline in visual ability follows. Affected children generally become wheelchair-bound by the ages of 4 to 6 years and eventually become bedridden.

Because the disease is fatal early in life, there are only about 200 cases of the disease in the world at a given time. Subjects from around the world were carefully selected to take part in the trial.

Neurological surgeons from NewYork-Presbyterian Hospital/Weill Cornell Medical Center, led by Drs. Mark Souweidane and Michael Kaplitt, performed the gene therapy procedure. Six tiny holes were made in the skull of each subject, and then a liquid containing the healthy CLN2 gene, within the harmless adeno-associated virus (AAV), was injected into the brain.

"Before now, we had no hope of a therapy for Batten disease, but today we can say that there is some hope," says Dr. Crystal. "These results are not just promising for sufferers of the disease, but suggest that gene therapy can work and should be studied for other neurological disorders. Each gene in our body has the potential to become a target to study for human disease."


'/>"/>

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:7/11/2014)... 2014 Global enterprises today are shifting ... day-to-day management activities. Managed services offer these global enterprises ... economic slowdown resulted into rising operational costs to companies. ... considered to be the global market driver for expanding ... global managed services market is estimated to grow from ...
(Date:7/11/2014)... (July 10, 2014) Obese and overweight firefighters ... health care providers, according to new research from ... Houston (UTHealth). , National guidelines state that health ... importance of maintaining a healthy weight. Firefighters have ... the leading cause of line-of-duty deaths in firefighters. ...
(Date:7/11/2014)... Prostate cancer patients are more likely to be diagnosed ... sooner if they also have depression, a new study ... American men who were diagnosed with localized prostate cancer ... Nearly 1,900 of the patients had been diagnosed with ... was discovered. "Men with intermediate- or high-risk prostate ...
(Date:7/11/2014)... 11, 2014 Europe is one of ... large cultivated-and hence, irrigated land area. The agricultural sector ... accounting for around 30.0% of the total water use. ... in the southern region, but far from negligible in ... accounts for over 60.0% of the water use in ...
(Date:7/11/2014)... who screened positive for a history of hazardous or ... 50 percent after receiving mobile phone text messages following ... study published online yesterday in Annals of Emergency ... Young Adult Emergency Department Patients: A Randomized Clinical Trial ... 50,000 adults ages 18 to 24 visit ERs and ...
Breaking Medicine News(10 mins):Health News:North America Managed Services Market Worth $68.65 Billion by 2018 - New Report by MicroMarket Monitor 2Health News:North America Managed Services Market Worth $68.65 Billion by 2018 - New Report by MicroMarket Monitor 3Health News:North America Managed Services Market Worth $68.65 Billion by 2018 - New Report by MicroMarket Monitor 4Health News:Depression May Make It Harder to Beat Prostate Cancer 2Health News:Europe Micro Irrigation System Market Driven by Growing Environmental Concerns and Decreasing Water Availability - New Report by MicroMarket Monitor 2Health News:Europe Micro Irrigation System Market Driven by Growing Environmental Concerns and Decreasing Water Availability - New Report by MicroMarket Monitor 3Health News:Text message medicine: Texts from the ER can reduce binge drinking 2
... , , , ... G. Komen for the Cure((R)), the world,s largest grassroots network of breast ... that its San Diego Affiliate will receive proceeds from the album release ... 6 at Anthology Supper Club, will feature Miz Mandy along with more ...
... , SANTA CLARA, Calif., July 21 ... on the a lawsuit brought by Ormco Corporation (Ormco) against Align Technology, ... California (District Court), which was tried by jury June 9 through June ... previously disclosed, on June 26, 2009, the jury delivered a verdict against ...
... ANDOVER, Mass., July 21 Hendrick Health System, a ... area, finished its implementation of Sentillion,s Tap & Go(TM) solution, ... its enterprise roll-out of Sentillion,s single sign-on solution (SSO), Hendrick ... its trauma center to use their facility access cards to ...
... , , SAVANNAH, Ga. July ... Diane Weems, MD, Chief Medical Officer of the Coastal Health ... Jenkins, Program Coordinator, Coastal Area Agency on Aging, gathered today ... their personal risk for diabetes. Together, they launched the ...
... the U.S. Surgeons General provided roadmap to take decisive action in ... , AUSTIN, Texas and TUCSON, Ariz., July ... Armstrong Foundation (LAF), cancer survivor, and champion cyclist, battles through the ... to Action on Cancer Prevention and Survivorship that Armstrong helped ...
... health at risk by avoiding disease screening, in the ... they prove their masculinity through their sexuality and sexual ... their notion of masculinity. Waverly Duck, a Post Doctoral ... in the US, argues that current leading theories of ...
Cached Medicine News:Health News:Susan G. Komen for the Cure(R), San Diego to Receive Proceeds from Miz Mandy Album Release Concert 2Health News:Susan G. Komen for the Cure(R), San Diego to Receive Proceeds from Miz Mandy Album Release Concert 3Health News:Align Technology Provides Update on Patent Litigation with Ormco Corporation 2Health News:Align Technology Provides Update on Patent Litigation with Ormco Corporation 3Health News:Align Technology Provides Update on Patent Litigation with Ormco Corporation 4Health News:Hendrick Health System Deploys Sentillion's Tap & Go Advanced Authentication Solution 2Health News:Hendrick Health System Deploys Sentillion's Tap & Go Advanced Authentication Solution 3Health News:Savannah City Leaders Launch Medicare Diabetes Screening Project 2Health News:Savannah City Leaders Launch Medicare Diabetes Screening Project 3Health News:First Anniversary of New War on Cancer Marks Many Advances 2Health News:First Anniversary of New War on Cancer Marks Many Advances 3Health News:First Anniversary of New War on Cancer Marks Many Advances 4Health News:First Anniversary of New War on Cancer Marks Many Advances 5Health News:First Anniversary of New War on Cancer Marks Many Advances 6Health News:First Anniversary of New War on Cancer Marks Many Advances 7Health News:'Go to the doctor? Only if I'm really sick...' 2
(Date:1/15/2014)... , Jan. 15, 2014 Sono-Tek Corporation (OTC BB: SOTK) ... November 30, 2013, compared to sales of $2,202,000 for the ... again, this quarter has also shown growth over the preceding ... quarter of this fiscal year. Markets that experienced sales increases ...
(Date:1/15/2014)... GRAND RAPIDS, Mich. , Jan. 15, 2014 As ... of the flu virus, select Meijer pharmacies in ... and strep throat to eligible patients, enabling Meijer pharmacists to ... under strict protocol set by a physician participating in the ...
(Date:1/15/2014)... 2014 Massachusetts General Hospital (MGH), the Broad ... announced today that they have launched a strategic ... targets and develop novel therapies for inflammatory bowel ... worldwide. The MGH-Broad-Amgen collaboration brings together scientists with ...
Breaking Medicine Technology:Sono-Tek Announces Third Quarter Results 2Sono-Tek Announces Third Quarter Results 3Sono-Tek Announces Third Quarter Results 4Meijer Pharmacy Offers Free Flu, Strep Testing as Influenza Cases Rise Across the Midwest 2Meijer Pharmacy Offers Free Flu, Strep Testing as Influenza Cases Rise Across the Midwest 3Mass. General, Broad Institute and Amgen Will Work to Discover New Drugs for Inflammatory Bowel Disease 2Mass. General, Broad Institute and Amgen Will Work to Discover New Drugs for Inflammatory Bowel Disease 3Mass. General, Broad Institute and Amgen Will Work to Discover New Drugs for Inflammatory Bowel Disease 4
... 10 Xanodyne Pharmaceuticals, Inc., an integrated specialty pharmaceutical company, ... the company on the pain management market.  The company plans to ... a range of products that address both the acute and chronic ... , , , ...
... Inc. (Nasdaq: YDNT ) today announced that its Board of Directors declared a ... to all shareholders of record on May 22, 2010 . , ... The Company also announced that its Board of Directors elected ... 2003 . He has assumed positions of increasing responsibility in the areas of finance, ...
Cached Medicine Technology:Xanodyne Announces Renewed Focus on Pain Management Market 2Xanodyne Announces Renewed Focus on Pain Management Market 3Xanodyne Announces Renewed Focus on Pain Management Market 4Young Innovations, Inc. Board of Directors Declares Quarterly Dividend and Announces Election of New Officer 2
... ARUP Laboratories is a national reference laboratory ... research and development. ARUP offers an extensive ... medical tests in clinical and anatomic pathology. ... Laboratories' clients include more than half of ...
... ARUP Laboratories is a national reference laboratory ... research and development. ARUP offers an extensive ... medical tests in clinical and anatomic pathology. ... Laboratories' clients include more than half of ...
... national reference laboratory and a worldwide leader ... offers an extensive test menu of highly ... and anatomic pathology. Owned by the University ... than half of the nation's university teaching ...
... ARUP Laboratories is a national reference laboratory ... research and development. ARUP offers an extensive ... medical tests in clinical and anatomic pathology. ... Laboratories' clients include more than half of ...
Medicine Products: