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:7/30/2014)... July 30, 2014 Ticket Down ... Bayern Munich vs. Chivas Guadalajara friendly exhibition match at ... World Cup, the one team that was left standing as ... made it through the world’s largest football competition and even ... their way to the cup. Germany was led by seven ...
(Date:7/30/2014)... Ticket Down is a reputable source for affordable ... on Saturday, August 2nd. Two of the most popular club ... American soil on Aug. 2 when Manchester United and Real Madrid ... of the University of Michigan is also known as Michigan Stadium. ... American sports and has also hosted numerous other monumental events in ...
(Date:7/30/2014)... (HealthDay News) -- It can happen in an instant: A ... computer monitor and gets critically injured when the furniture tips ... should look around their homes and imagine what could tip ... is better than it becoming a reality," Dr. Alex Rosenau, ... a college news release. Between 2009 and 2011, roughly ...
(Date:7/30/2014)... MA (PRWEB) July 30, 2014 ... of authentication, access management and secure communications solutions ... Imprivata OneSign ® is the most widely selected ... McKesson Paragon or Horizon electronic medical records (EMR) ... increase of about 11 percent in the last ...
(Date:7/30/2014)... KY (PRWEB) July 30, 2014 DRE ... pleased to announce the launch of its Medical ... online resource for medical professionals and equipment vendors. , ... shows and conventions in the United States and around ... the calendar, and that number is always growing. , ...
Breaking Medicine News(10 mins):Health News:Bayern Munich vs. Chivas Guadalajara Tickets Red Bull Arena: Ticket Down Slashes Ticket Prices for Chivas Guadalajara vs. Bayern Munich in Harrison, NJ 2Health News:Bayern Munich vs. Chivas Guadalajara Tickets Red Bull Arena: Ticket Down Slashes Ticket Prices for Chivas Guadalajara vs. Bayern Munich in Harrison, NJ 3Health News:Manchester United vs. Real Madrid Tickets Ann Arbor: Ticket Down Slashes Ticket Prices for Real Madrid vs. ManU Guinness International Champions Cup Match 2Health News:Manchester United vs. Real Madrid Tickets Ann Arbor: Ticket Down Slashes Ticket Prices for Real Madrid vs. ManU Guinness International Champions Cup Match 3Health News:Manchester United vs. Real Madrid Tickets Ann Arbor: Ticket Down Slashes Ticket Prices for Real Madrid vs. ManU Guinness International Champions Cup Match 4Health News:Tip-Over Furniture Can Kill Kids 2Health News:Imprivata Achieves 51 Percent Single Sign-on Market Share Amongst U.S. Hospitals Using McKesson Paragon or Horizon EMR Systems, According to HIMSS Analytics 2Health News:DRE Launches Online Interactive Medical Trade Show Calendar 2
... Chilcott Limited (Nasdaq: WCRX ) announced today that ... Conference on Wednesday, June 10, 2009 at 10:20 a.m. ET. ... and Chief Executive Officer and Paul Herendeen, Executive Vice President ... will be webcast live for all interested parties at ...
... , , WOONSOCKET, R.I., June 8 ... the fight against amyotrophic lateral sclerosis (ALS). Since 2002, CVS/pharmacy ... advance ALS research. To date, CVS/pharmacy has raised more than ... at a time -- to support the funding of new ...
... Proven Solution to Help Employees Lose Weight, Be Healthy and Save ... (Nasdaq: NTRI ), a leading provider of weight management ... its Nutrisystem Works(TM) program to companies and health plans ... care costs. The obesity epidemic continues to rise and studies ...
... Includes New Weight Management Genetic Test that Provides Evidence-Based ... , , WALTHAM, Mass., June 8 ... announced today the introduction of its new Inherent Health(TM) ... first-of-its-kind test for weight management that identifies an individual,s ...
... for Fourth Annual National Conference with Key Legislators, Policy Makers ... Risk Youth , , LOS ANGELES and WASHINGTON, June ... one of the premier non-profit organizations working with at-risk and ... Action Network" announces celebrities and legislators lending their voice the ...
... THAT STOCKHOLDERS VOTE FOR PROVIDENCE ,S DIRECTOR NOMINEES ... Service Corporation (Nasdaq: PRSC ) today announced that ... and risk management consulting firm to the global financial community, ... on Providence,s WHITE proxy card for ...
Cached Medicine News:Health News:Video: CVS/pharmacy Kicks Off 8th Annual ALS Fundraising Campaign in Stores Nationwide 2Health News:Video: CVS/pharmacy Kicks Off 8th Annual ALS Fundraising Campaign in Stores Nationwide 3Health News:New Nutrisystem Works(TM) Offers Corporate America New Weapon in the Fight Against Obesity 2Health News:New Nutrisystem Works(TM) Offers Corporate America New Weapon in the Fight Against Obesity 3Health News:New Nutrisystem Works(TM) Offers Corporate America New Weapon in the Fight Against Obesity 4Health News:Interleukin Genetics Launches Inherent Health Brand of Genetic Tests Offering Clear and Personalized Insights to Wellness 2Health News:Interleukin Genetics Launches Inherent Health Brand of Genetic Tests Offering Clear and Personalized Insights to Wellness 3Health News:Interleukin Genetics Launches Inherent Health Brand of Genetic Tests Offering Clear and Personalized Insights to Wellness 4Health News:Interleukin Genetics Launches Inherent Health Brand of Genetic Tests Offering Clear and Personalized Insights to Wellness 5Health News:Children Uniting Nations, Russell Simmons, Mos Def, David Archuleta, Terrence Howard, Gabrielle Union, Vivica Fox, Elise Neal, and Shawn King - Visit Nation's Capitol to Promote Student Mentoring and Change for Child Welfare 2Health News:RiskMetrics Recommends That Providence Service Corporation Stockholders Vote for Providence's Director Nominees on the White Proxy Card 2Health News:RiskMetrics Recommends That Providence Service Corporation Stockholders Vote for Providence's Director Nominees on the White Proxy Card 3Health News:RiskMetrics Recommends That Providence Service Corporation Stockholders Vote for Providence's Director Nominees on the White Proxy Card 4Health News:RiskMetrics Recommends That Providence Service Corporation Stockholders Vote for Providence's Director Nominees on the White Proxy Card 5Health News:RiskMetrics Recommends That Providence Service Corporation Stockholders Vote for Providence's Director Nominees on the White Proxy Card 6
(Date:7/30/2014)... ALEXANDRIA, Va. , July 30, 2014 /PRNewswire-USNewswire/ ... of Managed Care Pharmacy (AMCP) last evening expressed ... developed by the Joint Commission of Pharmacy Practitioners ... other member organizations over a two-year period to ... process for pharmacists.  "Pharmacists are ...
(Date:7/30/2014)... 30, 2014  El Camino Hospital, a leader in ... utilizing the Leaf Patient Monitoring system to help prevent ... of patients susceptible to bed sores. The ... study of the system, which showed dramatic improvement in ... regularly is the one of the most effect ways ...
(Date:7/30/2014)... 30, 2014   Unique Pizza and Subs ... Pink: UPZS), a Delaware Corporation, is pleased to ... Larasan Pharmaceutical Corporation ("Larasan"), a nature-based pharmaceutical company that ... agreement, US-based Larasan will work with Unique to ... through Larasan,s vast distribution network throughout ...
Breaking Medicine Technology:AMCP Endorses JCPP "Pharmacists' Patient Care Process" 2El Camino Hospital Utilizes Cutting-Edge Leaf Patient Monitor System to Help Reduce Risk of Pressure Ulcers 2El Camino Hospital Utilizes Cutting-Edge Leaf Patient Monitor System to Help Reduce Risk of Pressure Ulcers 3Unique Pizza and Subs Announces Asian Distribution Agreement with Larasan Pharmaceutical Corporation 2Unique Pizza and Subs Announces Asian Distribution Agreement with Larasan Pharmaceutical Corporation 3
... 15, 2011 Patient Safety Technologies, Inc. (the "Company", ... its third quarter of 2011 ended September 30th, 2011. ... the third quarter of 2011, the number of institutions ... the Company lost no customers.  This compares to approximately ...
... (NYSE: BSX ) commends the leadership of Senator ... Kerry (D-MA) for their introduction today of the Patient Access ... The legislation is a thoughtful and well-timed effort to ...  It seeks to improve the ability of companies like Boston ...
Cached Medicine Technology:Patient Safety Technologies Reports Third Quarter 2011 Results 2Patient Safety Technologies Reports Third Quarter 2011 Results 3Patient Safety Technologies Reports Third Quarter 2011 Results 4Patient Safety Technologies Reports Third Quarter 2011 Results 5Patient Safety Technologies Reports Third Quarter 2011 Results 6Patient Safety Technologies Reports Third Quarter 2011 Results 7
... 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 ...
... 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 ...
... 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 ...
... 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 ...
Medicine Products: