Navigation Links
1 shot of gene therapy and children with congenital blindness can now see
Date:10/24/2009

and said he could see the color of his eyes. Later they played soccer together.

For children and adults in the study, functional improvements in vision followed single injections of genes that produced proteins to make light receptors work in their retinas.

The 12 subjects ranged in age from 8 to 44 years old at the time of treatment. Four of the children, aged 8 to 11, are the world's youngest individuals to receive gene therapy for a non-lethal disease (A fifth subject was 17 years old). On the other end of the age scale, the 35-year-old man and 44-year-old woman are the oldest patients to ever receive gene therapy for retinal degeneration.

For the current human trial, the research team used a vector, a genetically engineered adeno-associated virus, to carry a normal version of the gene, called RPE65, that is mutated in one form of LCA, called LCA2, that accounts for 8 to 16 percent of all LCA cases. Jeannette Bennicelli, Ph.D., in Bennett's laboratory, cloned the gene. The clinical vector production facility at Children's Hospital's Center for Cellular and Molecular Therapeutics (CCMT), directed by Fraser Wright, Ph.D., manufactured the vector.

The clinical trial brought together subjects and scientists from two continents. Five patients enrolled in the study were identified at the Department of Ophthalmology at the Second University of Naples, an institution with a long-standing program in researching inherited retinal diseases, under the supervision of Francesca Simonelli, M.D. Two children from Belgium were recruited through Ghent University Hospital, under the supervision of Bart Leroy, M.D., Ph.D. Jennifer Wellman, of the CCMT, directed all local and federal regulatory interactions for the study.

Another co-author, Edwin Stone, M.D., Ph.D., Howard Hughes Medical Institute Investigator and director of the Carver Center, a genetic testing laboratory at the University of Iowa, identified and verified several of th
'/>"/>

Contact: Karen Kreeger
karen.kreeger@uphs.upenn.edu
215-459-0544
University of Pennsylvania School of Medicine
Source:Eurekalert  

Page: 1 2 3 4 5

Related medicine news :

1. Gene Therapy Offers Hope for Severe Hereditary Eye Disease
2. Common Knee Pain May Improve With Therapy
3. Leaders in Minimally Invasive Spinal Surgery Acquire Non-Surgical Spine Therapy Pioneer
4. Mice regain ability to extend telomeres suggesting potential for dyskeratosis congenita therapy
5. Stem Cell and PRP Therapy for Orthopedic Conditions
6. Supervised Exercise May Relieve Fatigue During Chemotherapy
7. Gene Therapy Shows Promise for Parkinsons
8. Video: Philips Unveils Intelligent Sleep Apnea Therapy System to Home Healthcare Industry
9. With Hormone Therapy, Tender Breasts May Signal Cancer Risk
10. Organized phone therapy for depression found cost-effective
11. Although more older women receive breast-conserving therapy, gaps in treatment exist
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
1 shot of gene therapy and children with congenital blindness can now see
(Date:10/22/2014)... Best Cheap Hosting USA is among the most ... recently announced that iPower ( http://www.best-cheap-hosting-usa.com/go/iPower/ ) is the ... the world. , “iPower is one of the most ... various kinds of useful products for new and old ... customer service and a number of features which will ...
(Date:10/22/2014)... there are only 24 hours in a day, that doesn’t seem ... time within their busy schedules for exercise and fitness. Fortunately, one ... get in shape. Here are five ways you can reach your ... Up Your Commute , Consider riding a bike or taking the ... daily routine. If you must use your car to get to ...
(Date:10/22/2014)... MA (PRWEB) October 22, 2014 ... new solutions for the treatment of bacterial infections, microbial ... Sinskey has joined its board of directors. , ... and Engineering Systems at The Massachusetts Institute of Technology, ... MIT faculty since 1968. Dr. Sinskey also holds positions ...
(Date:10/22/2014)... 2014 (HealthDay News) -- Two sisters in high school ... Ilina and Medha Krishen use electronic stethoscopes, which ... trouble in breathing patterns or heartbeats. Ilina, a ... wanted to find a way to detect early lung ... an electronic stethoscope, Ilina recorded one breath cycle each ...
(Date:10/22/2014)... taken by Firestone officials at the company,s rubber ... spread of the disease there and could prove effective ... provides health services to about 80,000 employees, retirees, their ... Between Aug. 1 and Sept. 23, there were 71 ... That incidence rate of 0.09 percent was much lower ...
Breaking Medicine News(10 mins):Health News:Best Cheap Hosting USA Has Recently Announced The Best Cheap Web Hosting Companies In 2014 2Health News:HealthGuideMD Presents Ways to Improve Your Exercise and Fitness 2Health News:Akeso Biomedical Appoints Dr. Anthony J. Sinskey to Board of Directors 2Health News:Teen Sisters Develop Ways to Measure Lung, Heart Damage 2Health News:Tire Company Sets Standard for Ebola Care in Liberia: CDC 2
... 6 HealthSouth Corporation (NYSE: HLS ) ... a.m. CT, on Thursday, May 7, 2009, at its ... in Birmingham, Ala.HealthSouth stockholders are invited to attend the ... to a live audio webcast of the annual meeting, ...
... Health Information Exchange ComponentsCHICAGO, April 6 (HIMSS,09, ... HRS ) has been awarded the first-year option to ... the Federal Health Architect of the U.S. Department of ... CONNECT is an open-source implementation of the NHIN ...
... Portable health record sticks around as you change ... 6 While the government strives to deliver electronic ... Health Solutions, Inc. (NYSE: MHS ) and ... The companies, collaboration enables Medco,s more than 60 million ...
... Records (EHR) ImplementationROUND ROCK and PLANO, Texas, April 6 ... and Perot Systems today ... will provide fully-integrated global IT solutions that unite their ... storage and server solutions for hospitals, health systems and ...
... data transparency for quality improvement.CHICAGO, April 6 ... www.siemens.com/healthcare ) ... care with Soarian(R) Quality Measures, enabled by the REMIND(TM) Platform, ... Systems Society (HIMSS) 2009 Annual Conference and Exhibition in Chicago ...
... Booth #3974) -- Harris Corporation (NYSE: ... company, has donated $10,000 to Wounded Warrior Project (WWP), ... empower wounded warriors. The donation is being presented ... the HIMSS,09 Healthcare IT Conference and Exposition being held ...
Cached Medicine News:Health News:HealthSouth to Hold Annual Stockholder Meeting 2Health News:Harris Corporation Awarded Follow-on Contract Option to Expand CONNECT Solution for Nationwide Health Information Network 2Health News:Harris Corporation Awarded Follow-on Contract Option to Expand CONNECT Solution for Nationwide Health Information Network 3Health News:Medco and Google Keep Information and Patients Safe with Personal Prescription Records 2Health News:Medco and Google Keep Information and Patients Safe with Personal Prescription Records 3Health News:Dell and Perot Systems Announce Strategic Alliance to Provide Fully-Integrated Virtualized Healthcare Technology Solutions to Reduce Cost and Improve Patient Care 2Health News:Dell and Perot Systems Announce Strategic Alliance to Provide Fully-Integrated Virtualized Healthcare Technology Solutions to Reduce Cost and Improve Patient Care 3Health News:Dell and Perot Systems Announce Strategic Alliance to Provide Fully-Integrated Virtualized Healthcare Technology Solutions to Reduce Cost and Improve Patient Care 4Health News:Dell and Perot Systems Announce Strategic Alliance to Provide Fully-Integrated Virtualized Healthcare Technology Solutions to Reduce Cost and Improve Patient Care 5Health News:Siemens Enhances Knowledge-Driven Solutions to Support Provider Quality of Care Initiatives 2Health News:Siemens Enhances Knowledge-Driven Solutions to Support Provider Quality of Care Initiatives 3Health News:Harris Corporation Donates $10,000 to Wounded Warrior Project during HIMSS'09 Healthcare IT Conference and Exposition 2
(Date:10/22/2014)... VERNE, Calif. , Oct. 22, 2014 /PRNewswire/ ... readiness products, has developed APLS ® Body ... that minimizes the spread of bacteria and virus ... APLS Body Guard Bio features a rugged, ... finished edges that create a leak-proof inner chamber ...
(Date:10/22/2014)... , October 22, 2014 ... ), a clinical-stage pharmaceutical company focused on ... reported positive top-line clinical results from its Phase ... oral insulin capsules, to treat type 1 diabetes. The ... States under a U.S. Food and ...
(Date:10/22/2014)... Colo. , Oct. 22, 2014   Surefire ... of infusion systems designed to maximize targeted delivery of ... pleased to announce that oncologist John R. Daniels ... Comprehensive Cancer Center, and surgical oncologist Steven C. ... have joined the Company,s Scientific Advisory Board (SAB).    ...
Breaking Medicine Technology:New, State-Of-The-Art Body Bag Minimizes The Spread Of Bacteria And Virus 2Oramed Pharmaceuticals Reports Positive Top-Line Data from U.S. Phase IIa Trial with Oral Insulin in Type 1 Diabetes 2Oramed Pharmaceuticals Reports Positive Top-Line Data from U.S. Phase IIa Trial with Oral Insulin in Type 1 Diabetes 3Oramed Pharmaceuticals Reports Positive Top-Line Data from U.S. Phase IIa Trial with Oral Insulin in Type 1 Diabetes 4Surefire Medical's Scientific Advisory Board Expands, Broadens Clinical Trial Expertise 2
... Dec. 12, 2011 /PRNewswire-iReach/ -- Reagent ... a common compliance concern driving Quality Control departments ... VerGo Pharma Research proves to be an innovative ... their business practices this month.  Harness ...
... Phase III clinical trial data presented today contribute ... Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in ... have deeper levels of response compared to those ... The findings from the ENEST (Evaluating Nilotinib Efficacy ...
Cached Medicine Technology:VerGo Integrates New Solution for Tracking Compliance Dates 2Two Novartis Phase III Studies Show Twice as Many Ph+ CML Patients Achieve Deeper Levels of Response With Tasigna® Compared to Gleevec® 2Two Novartis Phase III Studies Show Twice as Many Ph+ CML Patients Achieve Deeper Levels of Response With Tasigna® Compared to Gleevec® 3Two Novartis Phase III Studies Show Twice as Many Ph+ CML Patients Achieve Deeper Levels of Response With Tasigna® Compared to Gleevec® 4Two Novartis Phase III Studies Show Twice as Many Ph+ CML Patients Achieve Deeper Levels of Response With Tasigna® Compared to Gleevec® 5Two Novartis Phase III Studies Show Twice as Many Ph+ CML Patients Achieve Deeper Levels of Response With Tasigna® Compared to Gleevec® 6Two Novartis Phase III Studies Show Twice as Many Ph+ CML Patients Achieve Deeper Levels of Response With Tasigna® Compared to Gleevec® 7Two Novartis Phase III Studies Show Twice as Many Ph+ CML Patients Achieve Deeper Levels of Response With Tasigna® Compared to Gleevec® 8Two Novartis Phase III Studies Show Twice as Many Ph+ CML Patients Achieve Deeper Levels of Response With Tasigna® Compared to Gleevec® 9Two Novartis Phase III Studies Show Twice as Many Ph+ CML Patients Achieve Deeper Levels of Response With Tasigna® Compared to Gleevec® 10Two Novartis Phase III Studies Show Twice as Many Ph+ CML Patients Achieve Deeper Levels of Response With Tasigna® Compared to Gleevec® 11Two Novartis Phase III Studies Show Twice as Many Ph+ CML Patients Achieve Deeper Levels of Response With Tasigna® Compared to Gleevec® 12Two Novartis Phase III Studies Show Twice as Many Ph+ CML Patients Achieve Deeper Levels of Response With Tasigna® Compared to Gleevec® 13Two Novartis Phase III Studies Show Twice as Many Ph+ CML Patients Achieve Deeper Levels of Response With Tasigna® Compared to Gleevec® 14Two Novartis Phase III Studies Show Twice as Many Ph+ CML Patients Achieve Deeper Levels of Response With Tasigna® Compared to Gleevec® 15Two Novartis Phase III Studies Show Twice as Many Ph+ CML Patients Achieve Deeper Levels of Response With Tasigna® Compared to Gleevec® 16
Advanced Lighting System with Multi-Lamp Design providing Surgeon Focus Control and Extended, Variable Depth of Field over greater working distances. High Intensity, Cool Beam Temperature...
... technology that precisely focuses a column ... surgical site. Patented prism based technology ... illumination, shadow control, heat management and ... PrismAlix Series is available in a ...
... SureFlex™ line of reusable and autoclavable Lithotripsy ... This revolutionary and patented termination method accepts ... In the unlikely event you do manage ... face absolutely prevents damage to your laser ...
Up-tapered output compresses NA to give low NA fiber performance on high NA fiber stock....
Medicine Products: