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

Philadelphia, Pa., Oct. 24, 2009 Born with a retinal disease that made him legally blind, and would eventually leave him totally sightless, the nine-year-old boy used to sit in the back of the classroom, relying on the large print on an electronic screen and assisted by teacher aides. Now, after a single injection of genes that produce light-sensitive pigments in the back of his eye, he sits in front with classmates and participates in class without extra help. In the playground, he joins his classmates in playing his first game of softball.

His treatment represents the next step toward medical science's goal of using gene therapy to cure disease. Extending a preliminary study published last year on three young adults, the full study reports successful, sustained results that showed notable improvement in children with congenital blindness.

The study, conducted by researchers from the University of Pennsylvania School of Medicine and the Center for Cellular and Molecular Therapeutics at The Children's Hospital of Philadelphia, used gene therapy to safely improve vision in five children and seven adults with Leber's congenital amaurosis (LCA). The greatest improvements occurred in the children, all of whom are now able to navigate a low-light obstacle courseone result that the researchers call "spectacular."

"This result is an exciting one for the entire field of gene therapy," said Katherine A. High, M.D., co-first author of the study and the director of the Center for Cellular and Molecular Therapeutics, the facility that sponsored the clinical trial at The Children's Hospital of Philadelphia. High, an investigator of the Howard Hughes Medical Institute and a past president of the American Society of Gene Therapy, has been a pioneer in translational and clinical studies of gene therapy for genetic disease. "This study reports dramatic results in restoring vision to patients who previously had no options for treatment," said High.
'/>"/>

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)... of Prussia, PA (PRWEB) October 22, 2014 ... is recognized as an industry leader with over 25 ... award winning team leader in the areas related to ... addition, Mr. Carlson is an Editorial Advisory Board member ... Healthcare Council Educator. Blue Fin Group is a ...
(Date:10/22/2014)... Salamon HealthDay Reporter , TUESDAY, ... vitro fertilization (IVF) are only about half as likely as ... technique, new research indicates, and the racial disparity persists even ... 31 percent of white patients became pregnant after IVF, compared ... than 4,000 IVF cycles over two years to tease out ...
(Date:10/22/2014)... HealthDay Reporter TUESDAY, Oct. ... in vitro fertilization (IVF) -- the incubation of embryos in ... device inside the vagina, new research suggests. Scientists from ... an INVOcell, might sharply cut costs for pricey IVF procedures ... accessible to those who don,t live near big-city assisted reproduction ...
(Date:10/22/2014)... HealthDay Reporter TUESDAY, Oct. 21, ... Ebola while working for NBC News in Liberia has ... isolation unit at Nebraska Medical Center in Omaha, where he had ... A blood test confirmed by the U.S. Centers for Disease ... to Providence, R.I., NBC News reported Tuesday night. ...
(Date:10/20/2014)... October 20, 2014 Allegheny General Hospital’s ... that enables physicians to repair a damaged mitral valve ... MitraClip system, developed by Abbott Vascular , ... progressive and life-threatening condition in which the heart’s mitral ... flow backward from the heart’s left ventricle into the ...
Breaking Medicine News(10 mins):Health News:Rich Carlson and Marc Duey to Hold Workshop at CBI’s Specialty Pharmacy and Distribution Networks Conference 2Health News:Black Women Fare Worse With Fertility Treatments, Study Says 2Health News:Black Women Fare Worse With Fertility Treatments, Study Says 3Health News:Experimental Infertility Treatment Seems Effective, Cheaper 2Health News:Experimental Infertility Treatment Seems Effective, Cheaper 3Health News:Experimental Infertility Treatment Seems Effective, Cheaper 4Health News:U.S. Cameraman Treated for Ebola 'Free' of the Virus 2Health News:U.S. Cameraman Treated for Ebola 'Free' of the Virus 3Health News:Allegheny General Hospital Introduces New, Beating-Heart Procedure for Mitral Valve Repair 2Health News:Allegheny General Hospital Introduces New, Beating-Heart Procedure for Mitral Valve Repair 3
... Omission leaves them without treatment for blood-borne diseases, researchers ... medical students get stuck by needles, putting them at ... alert hospital authorities, which increases their risk of infection, ... 699 surgery residents at 17 medical centers and found ...
... study, athletes had ,younger, immune cells than sedentary, healthy adults ... shown that exercise can help ward off heart disease and ... may be found within cells themselves. , , Endurance ... chromosomes that protect the cell -- in their white blood ...
... , LOS ANGELES, Nov. 30 ... woman, and brings with it a host of uncomfortable symptoms that ... weight gain , night sweats and lethargy are just some ... menopause signs and symptoms can show up a full ten ...
... , Sponsored by Merck, ... active participants in their HIV treatment decisions , ARLINGTON, ... the #1 HIV/AIDS resource online, today launched My Health Tracker, ... to save, track and analyze their medical data. The tool, ...
... ... A new poll released today by Latino Decisions, the ... New Mexico (UNM-RWJF Center), and impreMedia, shows a widespread consensus among the ... support for expansion of coverage. For the first time, health care tops ...
... , CHARLTON, Mass., Nov. 30 Harrington Physician ... Practice physicians Martin Devine, Leah Doret, Elizabeth Fuller and Monika ... esteemed and popular Charlton-based physicians, coming from the Fallon Clinic, ... HealthCare at Charlton medical office building under construction at 10 ...
Cached Medicine News:Health News:Med Students Often Fail to Report Needlestick Injuries 2Health News:Exercise Guards White Blood Cells Against Aging 2Health News:Exercise Guards White Blood Cells Against Aging 3Health News:Anti-Aging Institute of California Offers New Natural Solution for Menopause Symptoms 2Health News:TheBody.com Launches My Health Tracker, Enabling HIV-Positive Patients to Track Lab Results, Medications and Supplements 2Health News:New Survey Shows Overwhelming Support Among Latinos for Health Care Reform That Includes Public Option 2Health News:New Survey Shows Overwhelming Support Among Latinos for Health Care Reform That Includes Public Option 3Health News:New Survey Shows Overwhelming Support Among Latinos for Health Care Reform That Includes Public Option 4Health News:New Survey Shows Overwhelming Support Among Latinos for Health Care Reform That Includes Public Option 5Health News:New Survey Shows Overwhelming Support Among Latinos for Health Care Reform That Includes Public Option 6Health News:New Survey Shows Overwhelming Support Among Latinos for Health Care Reform That Includes Public Option 7Health News:New Survey Shows Overwhelming Support Among Latinos for Health Care Reform That Includes Public Option 8Health News:New Survey Shows Overwhelming Support Among Latinos for Health Care Reform That Includes Public Option 9Health News:Family Practice Physicians Martin Devine, Leah Doret, Elizabeth Fuller and Monika Mathur Join Harrington Physician Services in Charlton 2Health News:Family Practice Physicians Martin Devine, Leah Doret, Elizabeth Fuller and Monika Mathur Join Harrington Physician Services in Charlton 3
(Date:10/22/2014)... 22, 2014 Oramed Pharmaceuticals ... clinical-stage pharmaceutical company focused on the development of ... clinical results from its Phase IIa clinical trial ... to treat type 1 diabetes. The trial was conducted ... under a U.S. Food and Drug Administration (FDA) ...
(Date:10/22/2014)... , Oct. 22, 2014   Surefire Medical ... infusion systems designed to maximize targeted delivery of embolization ... to announce that oncologist John R. Daniels , ... Cancer Center, and surgical oncologist Steven C. Katz ... joined the Company,s Scientific Advisory Board (SAB).    ...
(Date:10/22/2014)... Oct. 22, 2014 Provectus Biopharmaceuticals, Inc. (NYSEMKT: ... its novel investigational drug for cancer, is designed for ... side effects. Its oncology focus is on melanoma, breast ... phase 2 trials of PV-10 as a therapy for ... for atopic dermatitis and psoriasis. This ...
Breaking Medicine Technology:Oramed 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 2Provectus Biopharmaceuticals Looks at the Asian Markets: Analyst Coverage by Small Cap Traders 2
... July 19, 2011 Valeant Pharmaceuticals International, Inc. (NYSE: ... that its subsidiary, Valeant International (Barbados) SRL ("VIB"), has ... for the Canadian marketing and distribution rights to Zuacta™, ... be used in conjunction with oral COX-2 inhibitors or ...
... CITY, N.Y., July 19, 2011 Innovation, makers of ... unveil a new pharmacy automation product, PharmASSIST OPTIx™, at ... NV on July 21-23 and at the NACDS Pharmacy ... PharmASSIST OPTIx is an optical counting and imaging technology ...
Cached Medicine Technology:Valeant Pharmaceuticals Announces Distribution Agreement for Zuacta™ in Canada 2Innovation to Unveil New Pharmacy Automation Product at Key Pharmacy Industry Trade Shows 2
The Vitek 2 is a fully automated bacteriology system that performs bacterial identification and susceptibility testing analysis using a standard inoculum. It detects resistance and helps combat bacte...
IDS Rapid Yeast Plus, a microbial identification system....
IDS Rapid NH, a microbial identification system....
The Cogent Microbial Luminescence System (MLS) is a superior Rapid microbial detection system for UHT product screening combining proven technology of ATP-bioluminescence with unsurpassed hardware in...
Medicine Products: