Navigation Links
Penn receives prestigious national award for breakthrough in gene therapy
Date:4/19/2013

PHILADELPHIA A gene therapy study focused on finding a cure for a rare congenital blinding disease has been recognized as one of the ten most outstanding clinical research projects of the year by the Clinical Research Forum (CRF). The study, led by Jean Bennett, MD, Phd, F.M. Kirby professor of Ophthalmology at the University of Pennsylvania School of Medicine, and carried out in collaboration with Penn Medicine's Albert M. Maguire, MD, and Katherine A. High, MD at The Children's Hospital of Philadelphia (CHOP), has been presented with the Distinguished Clinical Research Achievement Award, the second highest given in the CRF's Annual Top 10 Clinical Research Achievement Awards. CRF award winners are cited as the most compelling examples of scientific innovation that results from the nation's investment in clinical research that can benefit human health and welfare.

The results of the most recent phase of the study for Leber's Congenital Amaurosis (LCA) at CHOP have led to the first Phase 3 gene therapy study in the United States and the first Phase 3 gene therapy study in the world for a non-lethal disorder. The team of researchers hopes that the studies could lead to the first approved gene therapy product in the United States.

"The data from our study has already been used to develop additional clinical trials for other blinding diseases," said Bennett. "There are two things that I think are really going to be important from this work: one, that we'll move forward with this particular disease and get approval for the drug that we've been developing, and two, that this could ultimately lead to approved treatments for other currently untreatable conditions."

Published in 2012, the winning studies are the latest in a long tradition of notable health advances that have occurred through clinical research such as eliminating polio, reducing the mortality of AIDS, and improving cancer survival rates that were propelled by combined in
'/>"/>

Contact: Katie Delach
katie.delach@uphs.upenn.edu
215-349-5964
University of Pennsylvania School of Medicine
Source:Eurekalert

Page: 1 2

Related biology news :

1. Peihua Jiang receives Ajinomoto Award for Young Investigators in Gustation from AChemS
2. Forsyth Institute receives $4.1 million grant for new center
3. Einstein receives $12 million grant to develop device for preventing HIV infection
4. Wayne State vision restoration technology receives Notice of Allowance for US patent app
5. BU researcher receives highest honor from the National Birth Defects Prevention Network
6. Geneticist Svante Pääbo receives the $500,000 Gruber Genetics Prize
7. Artificial retina receives FDA approval
8. NTU Provost receives prestigious Imperial College fellowship joining the ranks of top UK scientists
9. Xenon Receives a Milestone Payment for Marketing Approval of Glybera in Europe
10. Brainwave "Balancing" Research Receives $1 Million Grant From The Susanne Marcus Collins Foundation, Inc.
11. Celladon Corporation Receives Notice of Allowance from United States Patent and Trademark Office
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:9/18/2014)... of the fish world, are the ideal animal in ... They,ve moved from the ocean into tens of thousands ... time changing their skeleton to adapt to the new ... of sticklebacks now have turned up one of the ... simple change in that gene,s regulation in a freshwater ...
(Date:9/17/2014)... From accessible and affordable health care to reproductive ... depend on the ability of people to identify ... openly and respectfully, and find the most defensible ... practices to support these societal conversations?, The Hastings ... of Bioethical Issues have teamed up to publish ...
(Date:9/17/2014)... started in 2000 as an EC-FP5 four-year project, delivering ... steady progress and successful participations in several EC projects, ... Observation Network project (EU BON), to increase the ... and to extend the general dissemination of the Fauna ... has applied its novel e-Publishing tools to prepare ...
Breaking Biology News(10 mins):Counting fish teeth reveals regulatory DNA changes behind rapid evolution, adaptation 2Counting fish teeth reveals regulatory DNA changes behind rapid evolution, adaptation 3Why bioethics literacy matters 2Why bioethics literacy matters 3Contributions on Fauna Europaea: Data papers as innovative model on expert involvement 2
... to find a DNA-based test to determine the sex ... sturgeon genome contains trematode genes that didn,t originally belong ... causes a sexually transmitted disease in humans. Genetics ... found the parasite and pathogen genes while analyzing DNA ...
... have developed a rapid, high-throughput screening method for prevention ... Munkongdee et al, "Rapid diagnosis of α-thalassemia by melting ... The Journal of Molecular Diagnostics . α-Thalassemia ... in the production of a component of hemoglobin. ...
... A flyover of Belize,s thick jungles has revolutionized archaeology ... by one of the most-studied ancient civilizations -- the ... NASA-funded research project in April 2009 that collected the ... days. Aboard a Cessna 337, LiDAR (Light Detection ...
Cached Biology News:Lake sturgeon have genes from parasite, signs of human STD 2Space technology revolutionizes archaeology, understanding of Maya 2Space technology revolutionizes archaeology, understanding of Maya 3
(Date:9/18/2014)... MA (PRWEB) September 18, 2014 ... Molecule Sequencing (tSMS™) technology, announced today the closing ... of investment, led by Genomic Diagnostic Technologies, will ... and diagnostic applications. , Founded in March 2013 ... and Bioinformatics Analyst at Helicos BioSciences, SeqLL has ...
(Date:9/18/2014)... Artificial sweeteners, promoted as aids to weight loss and ... intolerance and metabolic disease; and they do it in ... of the gut microbiota the substantial population of ... of experiments in mice and humans, were published today ... Eran Elinav of the Weizmann Institute,s Immunology Department, who ...
(Date:9/18/2014)... The quest for the ultimate memory device for computing ... at The City College of New York led by ... exhibit both magnetic and ferroelectric properties. , Combining both ... can occur between them and for the devices that ... Combining these two properties in a single material, however, ...
(Date:9/18/2014)... September 18, 2014 September 24 marks ... MX. ASEA is the manufacturer of the world's first ... care product, RENU28. , The Mexico City event ... Duvan Botero, and ASEA Field Leader Shawn Catmull from ... PhD in Atomic/Medical Physics from the University of Utah ...
Breaking Biology Technology:SeqLL Closes Series-A Funding Round for Expansion of Single Molecule Sequencing 2Gut bacteria, artificial sweeteners and glucose intolerance 2Gut bacteria, artificial sweeteners and glucose intolerance 3Oxides discovered by CCNY team could advance memory devices 2Announcing the ASEA Mexico Grand Opening Tour Beginning September 18th 2
... , High-level expression of difficult heterologous proteins ... has created variants of the Epicurian Coli ... of strains, which contain extra copies of the E. ... for a high-level,expression of proteins that are difficult to ...
... primary libraries with new plasmid library construction,kits , ... Barry,Neiditch Alan Greener John C. Bauer ... II,pcmv-script and pAD-GAL4-2.1 XR library ... into Epicurian Coli ,XL10-Gold ...
... , , , ,Over 40% of Stratagenes GeneConnection cDNA ... estimated to be full-length clones , Jason Goncalves ... GeneConnection discovery cDNA clone collection YY ... many ,different human tissues and has identified thousands of ...
Cached Biology Technology:Codon Bias-Adjusted BL21 Derivatives for Protein Expression 2Codon Bias-Adjusted BL21 Derivatives for Protein Expression 3Codon Bias-Adjusted BL21 Derivatives for Protein Expression 4Codon Bias-Adjusted BL21 Derivatives for Protein Expression 5Codon Bias-Adjusted BL21 Derivatives for Protein Expression 6Codon Bias-Adjusted BL21 Derivatives for Protein Expression 7Generate High-Quality, Directional Plasmid cDNA Libraries 2Generate High-Quality, Directional Plasmid cDNA Libraries 3Generate High-Quality, Directional Plasmid cDNA Libraries 4Generate High-Quality, Directional Plasmid cDNA Libraries 5Generate High-Quality, Directional Plasmid cDNA Libraries 6Generate High-Quality, Directional Plasmid cDNA Libraries 7The GeneZoo or the GeneJungle? 2The GeneZoo or the GeneJungle? 3The GeneZoo or the GeneJungle? 4The GeneZoo or the GeneJungle? 5The GeneZoo or the GeneJungle? 6The GeneZoo or the GeneJungle? 7The GeneZoo or the GeneJungle? 8The GeneZoo or the GeneJungle? 9The GeneZoo or the GeneJungle? 10
Compound(s) tested through a broad panel of 38 ligand binding and enzyme assays targeting the Central Nervous System. Used to determine selectivity and potential Neurological liabilities of compound...
... Clean-up Maxi Kit provides a fast and ... SDS, Triton X-100 and other detergents ... chromatography using Norgens patented protein resin as ... of processing 0.25 - 8 mg of ...
... Proteolytic degradation is critical to the ... and regulatory proteins as important and ... metabolism, heat shock and stress response, ... receptors and ion channels, cell cycle ...
... for the identification of potential biomarkers in urine, ... diseases. Due to the fact that urine can ... an attractive alternative to blood plasma as a ... Urine Protein Concentration Micro Kit provides a fast ...
Biology Products: