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:4/17/2014)... April issue of Experimental Biology and Medicine ... Rex Gaskins and Paul Kenis in the Institute ... the University of Illinois Urbana-Champaign describe their recent ... underlie a variety of cell functions including energy ... crucial roles in regulating normal cellular behavior, redox ...
(Date:4/17/2014)... in the April issue of Immunity , Kevin ... Medical Research, says it,s time to take a fresh ... sepsis, which kills millions worldwide every year, including ... released into the bloodstream to fight an injury or ... for maintaining good health without inflammation, wounds and ...
(Date:4/17/2014)... military personnel who served in Iraq and Afghanistan ... injury (TBI) were compared to military personnel without ... Differences in measures of overall disability, cognitive function, ... are reported in an article in Journal ... Ann Liebert, Inc., publishers. The article is available ...
(Date:4/17/2014)... YORK, NY (April 16, 2014) The cause of ... a new study proposes that neurons may be mistaken ... immune system, similar to the way autoimmune diseases like ... the body,s cells. The study was published April 16, ... a new, and likely controversial, idea in Parkinson,s disease; ...
(Date:4/17/2014)... BostonResearchers from Boston University School of Medicine (BUSM) ... alcohol consumption in an experimental model. The findings, ... and Alcohol Abuse , may lead to more ... alcohol is one of the leading causes of ... significant negative economic impact by limiting the productivity ...
Breaking Medicine News(10 mins):Health News:Live cell imaging reveals distinct alterations of subcellular glutathione potentials 2Health News:Feinstein Institute researcher publishes new perspective on sepsis 2Health News:Long-term effects of battle-related 'blast plus impact' concussive TBI in US military 2Health News:Is Parkinson's an autoimmune disease? 2Health News:BUSM researchers find anti-seizure drug may reduce alcohol consumption 2
... in a novel way to design molecules that ... for breast cancer research. Scientists from the Albany ... showed that they were indeed potential anti-breast cancer ... paper detailing the research, Computational Design and Experimental ...
... helps patients with severe depression who don't respond to ... the earlier stages of the illness, according to ... ,"Electroconvulsive therapy has been shown to be a highly ... severe depression, bipolar disorder, and schizophrenia," writes Dr. Stephen ...
... women's separatist outfit, Dukhtaran-e-Milat (DeM) Monday opposed introduction of ... no such plans. ,Addressing a media conference ... oppose tooth and nail the nefarious Indian designs to ... introduction of sex education in schools." ,Andrabi, ...
... obviously thrilled with their landmark discovery of hemangioblasts or ... , According to the company,the stem cells made from ... and arteries of mice and rats, and appear to start ... blood vessel precursor cells out of the stem cells. The ...
... a deadly parasitic disease transmitted by the tiny sand fly. ... the scourge. ,According to C.P. Thakur, a former central ... over 100,000 people, mostly the poor, are suffering from this disease ... dying in thousands and silently. It is unlike when dozens died ...
... of Australia has advised to avoid using cups while feeding ... ,Traditionally, hospitals and parents have used bottles or ... some cases, hospitals have turned to cup feeding, in which ... placed beneath the upper lip. ,Anndrea Flint, a ...
Cached Medicine News:Health News:Researchers Identifies Molecules That is Effective in Fighting Against Breast Cancer 2Health News:Despite Being Effective Electroconvulsive Therapy is Underused for Severe Depression 2Health News:Hemangioblasts Aiding Eye Damage Repair 2Health News:Bihar Under the Grip of Kala Azar 2
(Date:1/15/2014)... 15, 2014 AARP Foundation today announced it has established ... from the severe cold weather that has gripped much of ... those in need; so to support these emergency relief efforts ... $250,000, which could mean up to $500,000 in aid. The ...
(Date:1/15/2014)... Texas , Jan. 15, 2014  Humberto C. Antunes,  ... Achievement Award" from the Journal of Drugs in ... & Clinical Conference (ODAC). The event is January 17-20, 2014, at ... Florida . The ODAC is ...
(Date:1/15/2014)... , Jan. 15, 2014 MultiCell Technologies, Inc. ... a U.S. provisional patent application concerning composition of matter, ... achieve targeted tumor cell death.  This ... noncoding double stranded RNA molecules (VSRNAs) which interfere with ...
Breaking Medicine Technology:AARP Foundation Establishes Winter Relief Fund for Older Adults Suffering from the Severe Cold Weather 2AARP Foundation Establishes Winter Relief Fund for Older Adults Suffering from the Severe Cold Weather 3Galderma's global leader honored with a "Lifetime in Dermatology Achievement Award" by Journal of Drugs in Dermatology 2Galderma's global leader honored with a "Lifetime in Dermatology Achievement Award" by Journal of Drugs in Dermatology 3Galderma's global leader honored with a "Lifetime in Dermatology Achievement Award" by Journal of Drugs in Dermatology 4MultiCell Technologies Files Patent Application Covering New Class of RNA Therapeutics 2MultiCell Technologies Files Patent Application Covering New Class of RNA Therapeutics 3
... detects the presence of Hemoglobin S ... of reconstituted solubility buffer and 20l of ... cell hemoglobin control. Each test kit contains ... two buffer dispensing caps and two vials ...
... Streck Cytometry Concentrate is for use ... by flow cytometry. Each 70ml bottle of ... fluid with distilled water. Compared to Cubitainers, ... usable space and saves money without compromising ...
... is an assayed control for monitoring immunophenotyping ... accuracy and reproducibility of all the steps ... red blood cell lysis. Reference values for ... B lymphocytes, granulocytes, monocytes, and NK cells. ...
Use Streck-Sheath to count and classify white blood cells on the Abbott CELL-DYN® 3000, 3500 and 3700. The shelf life is two years from date of manufacture...
Medicine Products: