Navigation Links
Gene therapy safety trial for childhood blindness under way
Date:11/19/2007

GAINESVILLE, Fla. Three decades have passed since gene therapy pioneer William W. Hauswirth, Ph.D., and his colleagues at the University of Florida began work on a virus that could safely deliver corrective genes into living animals.

Its been six years since a multi-university team used gene therapy to give sight to puppies born with a defect that causes blindness.

Now the gene-transfer technique is being tested for safety in people in a phase 1 clinical research study conducted by the University of Pennsylvania and the University of Florida with support from the National Eye Institute of the National Institutes of Health.

A young adult with a form of hereditary blindness called Leber congenital amaurosis type 2, or LCA2, received an injection of trillions of replacement genes into the retina of one eye this month, making the volunteer one of the first people in the world to undergo the procedure. Shalesh Kaushal, M.D., Ph.D., an assistant professor of ophthalmology at UF, performed the gene transfer.

The volunteer was discharged last week from the General Clinical Research Center at UF.

In all, six adults and then three children between the ages of 8 and 17 will undergo the gene-transfer procedure at UF over the next year or more before safety data are fully evaluated. Names are not being disclosed for privacy reasons. Potential risks are discussed with prospective participants as part of an extensive screening and informed consent process.

This is the first study of its kind to investigate inherited blindness, said Barry J. Byrne, M.D., Ph.D., a professor of molecular genetics and microbiology and director of UFs Powell Gene Therapy Center. The accomplishment reflects a great deal of work and dedication on the part of Dr. Hauswirth, as well as many other scientists and physicians, including Samuel G. Jacobson, M.D. Ph.D., professor of ophthalmology of the University of Pennsylvania, and literally dozens
'/>"/>

Contact: John Pastor
jdpastor@ufl.edu
352-273-5815
University of Florida
Source:Eurekalert

Page: 1 2 3

Related medicine news :

1. Vision restoration therapy shown to improve brain activity in brain injured patients
2. HIV therapy in pregnancy-data support WHO recommendations
3. Family-based treatment more effective than supportive psychotherapy in treating bulimia
4. Trial to Test Gene Therapy for Angina in Women
5. One of the Largest Post-WHI Physician Surveys Shows More Education is Needed: Patient Misinformation About Hormone Therapy Remains High
6. Aromatherapy Gift Line Sheds Humorous Light on Modern Therapy
7. Chemotherapy may be culprit for fatigue in breast cancer survivors
8. Atlanta Falcons Physical Therapy Centers Set to Launch This Fall
9. New Drug No Substitute for Standard Blood-Clot Therapy
10. Stem Cell Therapy Disappoints Against Rare Kidney Ailment
11. Nucletron Announces Management Buy-Out to More Effectively Meet the Demands of Its Customers and the Radiation Therapy Community
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:7/9/2014)... most common type of lung cancer, researchers have uncovered ... in forming tumors. The new knowledge may expand treatments ... changes already are available or are in clinical trials. ... from The Cancer Genome Atlas (TCGA), including researchers at ... Medical School and other institutions, studied tumors from 230 ...
(Date:7/9/2014)... in policing injection drug users in Russia might ... , A study, conducted by researchers from Boston ... collaboration with St. Petersburg Pavlov State University, sought ... the health outcomes of a cohort of HIV-positive ... Those who were arrested by police were more ...
(Date:7/9/2014)... There has been a sharp increase in the ... using MyChart, the online, interactive service that allows patients ... healthcare providers, schedule appointments, and renew prescriptions. , Over ... MyChart each year increased five-fold, while the ... 10-fold, according to a study by Dr. David ...
(Date:7/9/2014)... and have a reputation for being highly toxic, but when ... being being found to offer potential health benefits in a ... dementia. A new compound (AP39), designed and made at the ... by targeting delivery of very small amounts of the substance ... Scientists in Exeter have already found that the compound protects ...
(Date:7/9/2014)... prescription labels handed out by pharmacists may be ... medication, according to new research by the University ... the Blind)., The study, published recently in the ... on prescription medications dispensed by pharmacies do not ... By simply following recommended guidelines for font size, ...
Breaking Medicine News(10 mins):Health News:Lung cancer study hints at new treatments 2Health News:BU researchers relate arrests with HIV risk environment 2Health News:MyChart use skyrocketing among cancer patients, UT Southwestern study finds 2Health News:MyChart use skyrocketing among cancer patients, UT Southwestern study finds 3Health News:Rotten egg gas holds key to healthcare therapies 2Health News:Most prescription labels fail to meet guidelines, risking dosage errors 2
... French . Robot-assisted surgery dramatically improves outcomes ... researchers at the Jewish General Hospital,s Lady Davis Institute ... post-operative complications and shorter hospital stays, robotic procedures also ... late 2010 in a series of studies in ...
... , WEDNESDAY, Jan. 12 (HealthDay News) -- People who have ... after receiving the results, whether they are positive or negative, ... or get screening tests that might modify that risk, despite ... in the Jan. 13 issue of the New England ...
... of Science (AAAS) has elevated four Dartmouth professors to the ... Dartmouth Medical School. Medical School faculty members Charles ... MD, join Dartmouth biologist C. Robertson McClung among the 504 ... eight other Dartmouth researchers to have earned the distinction. ...
... Jan. 12 (HealthDay News) -- Research in rats suggests that ... by persistent ringing or other noises in the ears. ... a result of hearing loss. In this study, U.S. ... variety of sound tones over an extended period of time. ...
... cancer with a relatively high degree of accuracy when applied ... cancer, according to a study in the January issue of ... was performed at the University of Washington and Seattle Cancer ... with 592 patients who were recently diagnosed with breast cancer ...
... Scientists are reporting a scientific basis for the long-standing belief ... other countries can prevent and treat cataracts, a clouding of ... of vision loss worldwide. Their study appears in Inorganic ... Wu, Fu-Yung Huang, Shih-Hsiung Wu and colleagues note that eye ...
Cached Medicine News:Health News:Robotic surgery of 'tremendous benefit' to patients, say JGH researchers 2Health News:Consumer Genetic Testing May Not Boost Users' Anxiety 2Health News:Consumer Genetic Testing May Not Boost Users' Anxiety 3Health News:Dartmouth Medical School faculty named fellows of the American Association for the Advancement of Science 2Health News:Animal Study Finds Nerve Stimulation May Thwart Tinnitus 2
(Date:1/15/2014)... , Jan. 15, 2014  In an unprecedented effort to curb ... in ambulances and other transport vehicles, an advanced and portable UV ... personnel for the first time. In order to ... these deadly pathogens, West Palm Beach Fire ...
(Date:1/15/2014)... Jan. 15, 2014  Echo Therapeutics, Inc. (Nasdaq: ... Symphony® CGM System as a non-invasive, wireless continuous glucose ... , Executive Chairman and Interim CEO of Echo Therapeutics, ... Annual Equity Conference. Mr. Doman will ...
(Date:1/15/2014)... 2014 Reportlinker.com announces that a new ... catalogue: Insulin Pump ... http://www.reportlinker.com/p01957812/Insulin-Pump-Market--Forecast-–-Worldwide-Analysis.html#utm_source=prnewswire&utm_medium=pr&utm_campaign=Medical_Equipment_and_Supply ... Insulin Pumps are gaining strong acceptance ...
Breaking Medicine Technology:Superbugs No Longer Ride In Rescue Vehicles 2Echo Therapeutics to Present at the Noble Financial Capital Markets' Tenth Annual Equity Conference 2Echo Therapeutics to Present at the Noble Financial Capital Markets' Tenth Annual Equity Conference 3Insulin Pump Market & Forecast - Worldwide Analysis 2Insulin Pump Market & Forecast - Worldwide Analysis 3Insulin Pump Market & Forecast - Worldwide Analysis 4Insulin Pump Market & Forecast - Worldwide Analysis 5Insulin Pump Market & Forecast - Worldwide Analysis 6Insulin Pump Market & Forecast - Worldwide Analysis 7Insulin Pump Market & Forecast - Worldwide Analysis 8Insulin Pump Market & Forecast - Worldwide Analysis 9Insulin Pump Market & Forecast - Worldwide Analysis 10Insulin Pump Market & Forecast - Worldwide Analysis 11Insulin Pump Market & Forecast - Worldwide Analysis 12Insulin Pump Market & Forecast - Worldwide Analysis 13Insulin Pump Market & Forecast - Worldwide Analysis 14Insulin Pump Market & Forecast - Worldwide Analysis 15Insulin Pump Market & Forecast - Worldwide Analysis 16Insulin Pump Market & Forecast - Worldwide Analysis 17
... revised Concepts in Clinical Pharmacokinetics , a web-based ... Georgia. Developed in cooperation with the American Society of ... Continuing Education and College of Pharmacy, the new Online ... fundamental principles of absorption, distribution, metabolism, and the elimination ...
... preeminent law office of Nurenberg, Paris, Heller & ... Johnson & Johnson,s artificial joint replacement company DePuy ... damages resulting from the company,s now-recalled hip replacement ... District of Ohio, Eastern Division) ...
Cached Medicine Technology:Newly Revised Concepts in Clinical Pharmacokinetics Web-Based Continuing Education Course Offered by the University of Georgia 2Newly Revised Concepts in Clinical Pharmacokinetics Web-Based Continuing Education Course Offered by the University of Georgia 3DePuy Orthopaedics Accused of Deceiving Hip Replacement Patients 2DePuy Orthopaedics Accused of Deceiving Hip Replacement Patients 3
... is a national reference laboratory and a ... development. ARUP offers an extensive test menu ... in clinical and anatomic pathology. Owned by ... include more than half of the nation's ...
... reference laboratory and a worldwide leader in ... an extensive test menu of highly complex ... anatomic pathology. Owned by the University of ... half of the nation's university teaching hospitals ...
... 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 ...
... Laboratories is a national reference laboratory and ... and development. ARUP offers an extensive test ... tests in clinical and anatomic pathology. Owned ... clients include more than half of the ...
Medicine Products: