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

e disease-causing mutations in the study subjects.

In April 2008, the current study team published encouraging preliminary results in the New England Journal of Medicine regarding three young adults, the first to receive gene therapy in this current clinical trial. Those subjects showed improvements in their visual function in both objective vision tests and subjective reports by the patients themselves. Patients who could only detect hand movements gained the ability to read lines on an eye chart.

After the first group of three young adults was treated safely, the study team extended gene therapy to five children from the United States, Italy and Belgium, in addition to four other adults. Because animal studies conducted by Bennett and colleagues had shown that visual improvement was age-dependent, the researchers tested the hypothesis that younger human subjects would receive greater benefits from the treatment. "LCA is a progressive disease, so if a treatment was possible, it was plausible to intervene before damage to the retina was severe," said Bennett.

In all, 12 patients received the gene therapy via a surgical procedure performed by Maguire starting in October 2007 at The Children's Hospital of Philadelphia. For each subject, Maguire injected the therapeutic genes into the eye with poorer function. There were three patient cohorts, receiving low, middle and high doses. No serious adverse events occurred in any of the test subjects.

Starting two weeks after the injections, all 12 subjects reported improved vision in dimly lit environments in the injected eye. An objective measurement, which measures how the eye's pupil constricts, showed that all the subjects were able to detect significantly more light after treatment and also showed greater light sensitivity in each patient's treated eye compared to the untreated eye. In addition, before treatment, nine patients had nystagmus, an involuntary movement of the eyes that is
'/>"/>

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:12/25/2014)... York (PRWEB) December 25, 2014 Product ... DePuy ASR hip recall ( http://www.consumerinjurylawyers.com/DePuy/ ) continue to ... According to an Order issued in the U.S. District ... Court will convene an open conference on January 7, ... United States Courthouse in West Palm Beach, Florida. The ...
(Date:12/25/2014)... December 25, 2014 The short film “Color ... created in honor of a true 9/11 hero, Welles Remy ... festivals including the 24 hours of Nuremberg International Short Film ... , Actress, screenwriter and director Luciana Lagana ... film together with her husband, Gregory Graham - ...
(Date:12/25/2014)... Miami, FL (PRWEB) December 25, 2014 When ... often find themselves in a frenzy to get rid of ... classroom. But families in Coral Gables now have the solution ... its second salon clinic location, Lice Troopers provides full spectrum ... in the Coral Gable area, including Sunset, Key Biscayne, Brickell, ...
(Date:12/25/2014)... THURSDAY, Dec. 25, 2014 (HealthDay News) -- Overeating is common ... you eat in moderation, an expert says. "Don,t arrive ... up calories if you know you,ll be attending a party, ... Jill Ashbey-Pejoves, lead dietitian at Northern Westchester Hospital in Mount ... better to eat healthy during the day and even to ...
(Date:12/25/2014)... 25, 2014 Thousands of transvaginal mesh ... in a number of multidistrict litigations currently underway in ... Liebhard LLP reports. According to an Order issued on ... a Joint Status Conference in all of the proceedings ... been directed to submit a proposed agenda to the ...
Breaking Medicine News(10 mins):Health News:Federal Court Overseeing DePuy ASR Hip Lawsuit Set to Convene Conference in January, Bernstein Liebhard LLP Reports 2Health News:Federal Court Overseeing DePuy ASR Hip Lawsuit Set to Convene Conference in January, Bernstein Liebhard LLP Reports 3Health News:The True 9/11 Hero Story “Color of Honor” Premieres in Germany and Bangladesh: Luciana Lagana Plays the Mother of the Lead and Co-Produces with Blaise Dolcemaschio. 2Health News:The True 9/11 Hero Story “Color of Honor” Premieres in Germany and Bangladesh: Luciana Lagana Plays the Mother of the Lead and Co-Produces with Blaise Dolcemaschio. 3Health News:Coral Gables Florida Gets Ready to Be Lice-Free with Lice Troopers 2Health News:Federal Court Overseeing Thousands of Transvaginal Mesh Lawsuits Set to Convene Joint Status Conference for All Litigations, Bernstein Liebhard LLP Reports 2Health News:Federal Court Overseeing Thousands of Transvaginal Mesh Lawsuits Set to Convene Joint Status Conference for All Litigations, Bernstein Liebhard LLP Reports 3Health News:Federal Court Overseeing Thousands of Transvaginal Mesh Lawsuits Set to Convene Joint Status Conference for All Litigations, Bernstein Liebhard LLP Reports 4
... victims of a bite from the Brazilian wandering spider (Phoneutria ... in blood pressure, they also sport an uncomfortable erection. ,And ... Viagras. Scientists say they have figured out the chemical that ... , “The erection is a side effect that everybody who ...
... which the cancer cells ensure that new blood vessels feed ... discovered// that this can be blocked by a drug commonly ... ,New Research at Johns Hopkins have discovered that in mice ... placebo. "We were surprised, to say the least, that itraconazole ...
... hay fever for the loss of millions of hours of ... 600 people with hay fever symptoms, including sneezing, watery eyes ... hour of work per week during peak hay fever season. ... may seem small, consider that 20 to 50 million Americans ...
... serious injury and affect people of all ages. The general ... , if any, instruction on ladder use and safety. ... of the American Journal of Preventive Medicine and conducted by ... Children’s Hospital, more than 2.1 million individuals were treated in ...
... UK community survey reveals that more than one in three HIV ... be published in the journal Sexually// Transmitted Infections . ... men say that they do the same. ,The findings ... saunas in London, Manchester, and Brighton between 2003 and 2004, involved ...
... A simple dietary changes could be helpful to improve ... ,Female sexual dysfunction (FSD) includes a variety of disorders ... sexual activity, problems with orgasm or pain during sexual ... even the most loving relationships. It is also an ...
Cached Medicine News:Health News:Brazil Spider Offers Hope for Those Suffering from Erectile Dysfunction 2Health News:Hay Fever Can Send Work Productivity Down the Drain 2Health News:Hay Fever Can Send Work Productivity Down the Drain 3Health News:Ladder-related Injuries on the Rise in U.S 2Health News:Condom Promotion-Crucial To HIV/AIDS Prevention 2Health News:Condom Promotion-Crucial To HIV/AIDS Prevention 3Health News:Simple Changes in Diet Could Improve Sex Life of Women 2
(Date:12/24/2014)... ResMed (NYSE: RMD ) today announced its victory ... manufacturer BMC Medical Co., Ltd. The International ... infringe ResMed,s patents, and entered an order prohibiting BMC from ... United States : , iVolve nasal mask ... pillows mask , iVolve full face mask ...
(Date:12/24/2014)...  The American Diabetes Association is recommending a ... with diabetes and that all people with diabetes ... in keeping with recent changes to guidelines for ... of Cardiology (ACC) and American Heart Association (AHA). ... changes to the Association,s annual revised Standards of ...
(Date:12/22/2014)... MONMOUTH JUNCTION, N.J. , Dec. 22, 2014 ... immunotherapy company marketing its CytoSorb® blood purification technology ... cardiac surgery patients in 28 countries worldwide, today ... Di Russo , MD, FACS, as its Senior ... 2015. Dr. Di Russo ...
Breaking Medicine Technology:ResMed Wins Patent Infringement Lawsuit Against Chinese Manufacturer BMC Medical 2New Standards of Care Provide Guidelines for Statin Use for People with Diabetes to Prevent Heart Disease 2New Standards of Care Provide Guidelines for Statin Use for People with Diabetes to Prevent Heart Disease 3New Standards of Care Provide Guidelines for Statin Use for People with Diabetes to Prevent Heart Disease 4CytoSorbents Appoints Dr. Gregory B. Di Russo as Senior Vice President of Clinical Development 2CytoSorbents Appoints Dr. Gregory B. Di Russo as Senior Vice President of Clinical Development 3CytoSorbents Appoints Dr. Gregory B. Di Russo as Senior Vice President of Clinical Development 4CytoSorbents Appoints Dr. Gregory B. Di Russo as Senior Vice President of Clinical Development 5
... , CAMBRIDGE, Mass. , Aug. 4 Cytel Inc., ... ®: the very first industrial strength software package for adaptive dose-finding studies based on ... The ... attempts to identify the optimal dose level.  , ...
... 3 OmniComm Systems, Inc. (OTC: OMCM), (the ... that Webb & Company, P.A. ("Webb"), a South-Florida ... the Company,s new independent auditors for the fiscal year ... Company,s previous independent auditors, Greenberg & Company, LLC ("Greenberg"), ...
Cached Medicine Technology:Cytel Announces Compass® - The Only Professional Adaptive Trial Software Package for Bayesian or Frequentist Dose-Finding Studies 2Cytel Announces Compass® - The Only Professional Adaptive Trial Software Package for Bayesian or Frequentist Dose-Finding Studies 3OmniComm Systems Announces Auditor Change 2OmniComm Systems Announces Auditor Change 3
... 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 ...
... 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 ...
... a national reference laboratory and a worldwide ... ARUP offers an extensive test menu of ... clinical and anatomic pathology. Owned by the ... more than half of the nation's university ...
... 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 ...
Medicine Products: