Navigation Links
Gene therapy cures canines of inherited form of day blindness, Penn veterinary researchers say
Date:4/21/2010

PHILADELPHIA - Veterinary ophthalmology researchers from the University of Pennsylvania have used gene therapy to restore retinal cone function and day vision in two canine models of congenital achromatopsia, also called rod monochromacy or total color blindness.

Achromatopsia is a rare autosomal recessive disorder with an estimated prevalence in human beings of about 1 in 30,000 to 50,000. It primarily affects the function of the cone photoreceptors in the retina and serves as a representative model for other more common inherited retinal disorders affecting cones. Cone function is essential for color vision, central visual acuity and most daily visual activities, which underlines the importance of the newly developed treatment.

The treatment cured younger canines regardless of the mutation that caused their achromatopsia. It was effective for the 33 months of the study and most likely is permanent; however, researchers also observed a reproducible reduction in the cone therapy success rate in dogs treated at 54 weeks of age or older.

The successful therapy in dogs was documented by the restoration of the cone function using electroretinography and by objective measure of day vision behavior. The behavioral results suggest that inner retinal cells and central visual pathways were able to usefully process the input from the recovered cones.

The results represent the second successful cone-directed gene replacement therapy in achromatopsia animal models and the first outside of mouse models. The gene therapy targets mutations of the CNGB3 gene, the most common cause of achromatopsia in humans. Achromatopsia-affected dogs represent the only natural large animal model of CNGB3-achromatopsia.

The results hold promise for future clinical trials of cone-directed gene therapy in achromatopsia and other cone-specific disorders.

"The successful restoration of visual function with recombinant adeno-associated virus-mediated gene replacement therapy has ushered in a new era of retinal therapeutics," said Andrs M. Komromy, assistant professor of ophthalmology at the Penn School of Veterinary Medicine and lead author of the study.

Many vision-impairing disorders in humans result from genetic defects, and, to date, mutations have been identified in ~150 genes out of ~200 mapped retinal disease loci. This wealth of genetic information has provided fundamental understanding of the multiple and specialized roles played by photoreceptors and the retinal pigment epithelium in the visual process and how mutations in these genes result in disease. Together with the development of gene-transfer technologies, it is now possible to realistically consider the use of gene therapy to treat these previously untreatable disorders.


'/>"/>

Contact: Jordan Reese
jreese@upenn.edu
215-573-6604
University of Pennsylvania
Source:Eurekalert

Related biology news :

1. A new radiation therapy treatment developed for head and neck cancer patients
2. St. Jude finds factors that accelerate resistance to targeted therapy in lymphoblastic leukemia
3. UC health news: molecular pathway may predict chemotherapy effectiveness
4. MIT works toward safer gene therapy
5. Intravenous gene therapy protects normal tissue of mice during whole-body radiation
6. Gene, stem cell therapy only needs to be 50 percent effective to create a healthy heart
7. Fourth Annual International Conference on Cell Therapy for Cardiovascular Diseases
8. Safe and effective therapy discovered for patients with protein-losing enteropathy
9. Ireland Cancer Center researchers advance stem cell gene therapy
10. Dolphin therapy a dangerous fad, Emory researchers warn
11. Cancer and arthritis therapy may be promising treatment for diabetes
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:1/28/2016)... -- Synaptics (NASDAQ: SYNA ), a leading developer of human ... December 31, 2015. --> --> ... 2 percent compared to the comparable quarter last year to $470.5 ... $35.0 million, or $0.93 per diluted share. ... quarter of fiscal 2016 grew 9 percent over the prior year ...
(Date:1/25/2016)...  Glencoe Software, the world-leading supplier of image data ... provide the data management solution OMERO Plus for the ... Photo - http://photos.prnewswire.com/prnh/20160125/325328LOGO ... Phenotypic analysis measures the characteristics and behavior of ... states such as health and disease, the presence or ...
(Date:1/21/2016)... --> ... report "Emotion Detection and Recognition Market by Technology (Bio-Sensors, NLP, ... Voice Recognition and Others), Services, Application Areas, End ... published by MarketsandMarkets, the global Emotion Detection and ... Billion by 2020, at a CAGR of 31.9%, ...
Breaking Biology News(10 mins):
(Date:2/4/2016)... , Feb. 4, 2016 Sinovac Biotech Ltd. ... leading provider of biopharmaceutical products in China ... its board of directors received on February 4, 2016 ... from a consortium comprised of PKU V-Ming ( ... Co., Ltd., CICC Qianhai Development ( Shenzhen ...
(Date:2/3/2016)... 2016  Discovery Laboratories, Inc. (NASDAQ: DSCO ... KL4 surfactant therapies for respiratory diseases, today announced ... inducement award as a component of employment compensation ... President and Chief Executive Officer.  The award was ... 1, 2016 and granted as an inducement material ...
(Date:2/3/2016)... ... February 03, 2016 , ... Aerocom, a world-leading supplier ... the North American healthcare market. , Aerocom Healthcare, LLC will be based in ... will provide new pneumatic tube systems or expand existing systems within the U.S. ...
(Date:2/3/2016)... Feb. 3, 2016  Today, Symphony Technology Group (STG) ... , a leading provider of primary research and analytics-based ... IMS Health , a global information and technology services ... and technologies will be integrated into IMS Health to ... market research capabilities. ...
Breaking Biology Technology: