HOME >> BIOLOGY >> NEWS
Gene therapy for chicken model of childhood blindness

In this week's PLoS Medicine Susan Semple-Rowland and colleagues, from the University of Florida, report the results of gene therapy for a chicken model of Leber congenital amaurosis (LCA), one of a group of hereditary diseases that cause blindness in infants and children.

Leber congenital amaurosis (LCA) is the name of a group of hereditary diseases that cause blindness in infants and children. Changes in any one of a number of different genes can cause the blindness, which usually comes on quite soon after birth. About 20% of children with LCA have the most common type called LCA1 which is caused by defects in a gene called retinal guanylate cyclase (GUCY2D). Defects in the gene leave the light sensitive photoreceptors in the eye unable to respond to light, and so cause blindness. Loss of function of these receptor cells leads to their degeneration and eventually breakdown of the retina the light sensitive tissue at the back of the eye.

There is an animal model of LCA1, a strain of chickens with a mutation in the chicken GUCY2D gene. These chickens develop a disease very similar to that of humans. Gene therapy works by replacing a defective gene with a normal functional one, usually by packaging the normal gene into a harmless virus and injecting it into the affected area, in this case the eye.

The researchers injected a virus containing the normal gene into chicken embryos that had the abnormal gene, and allowed the chicks to hatch normally. Of seven treated chicks, six appeared to develop sight, in contrast to untreated animals. The improvement in the chicks' sight occurred despite the fact that only a relatively small percentage of the receptor cells were infected by the virus.

This study shows that in theory it is possible to treat this form of childhood blindness by gene therapy. However, before clinical trials can be considered for humans, researchers would need to demonstrate that the virus is safe in humans, and addition
'"/>

Contact: Andrew Hyde
ahyde@plos.org
44-012-234-63330
Public Library of Science
22-May-2006


Page: 1 2

Related biology news :

1. Testosterone replacement therapy: How safe for aging men?
2. Progesterone therapy and preterm birth: More evidence helps identify women who can benefit
3. Aggressive therapy best for certain AML patients
4. Under magnetic force, nanoparticles may deliver gene therapy
5. 47th Interscience Conference on Antimicrobial Agents and Chemotherapy
6. Stem cell therapy rescues motor neurons in ALS model
7. Study identifies new gene therapy tools for inherited blindness
8. Breast cancer and hormone therapy -- A looking-glass mirror?
9. Radiation therapy combined with microsurgery shows promise for curing injured spinal cord
10. Nodal status is best predictor of outcome after neoadjuvant therapy for esophageal cancer
11. Scientists follow familiar TRAIL to new cancer therapy

Post Your Comments:
*Name:
*Comment:
*Email:


(Date:1/4/2019)... ... January 03, 2019 , ... Over the ... into their drug discovery research workflows with biology ranging from single cell-type spheroids ... of in vitro studies, they create several challenges that need to be overcome ...
(Date:12/27/2018)... FRANCISCO (PRWEB) , ... December 27, 2018 , ... ... annual Microbiome Conference at JPM as part of the JP Morgan 37th Annual ... innovative scientists and physicians, and members of the investment community. , Since ...
(Date:12/20/2018)... ... December 20, 2018 , ... Human Factors and product ... a new usability/observation room. This addition will help meet the increased industry demand ... delivery devices/combination products are safe and easy to use when released to the ...
Breaking Biology News(10 mins):
(Date:1/10/2019)... Fla. (PRWEB) , ... January ... ... Geometrical Implant Co, (SAGICO), an innovative spinal implant company, announced today that ... in San Francisco, California. The annual J.P. Morgan Healthcare Conference is ...
(Date:1/7/2019)... SEONGNAM, South Korea (PRWEB) , ... January 07, ... ... has completed Phase 1 clinical trials of its Parkinson's Disease drug candidate, code-named ... disease-modifying treatment of Parkinson's Disease. The trial took place in South Korea. ...
(Date:1/4/2019)... ... January 03, 2019 , ... Calidi Biotherapeutics, Inc., ... today announced the founding of a scientific and medical advisory board (SMAB). The ... leaders in the Immuno-Oncology field. The founding members of Calidi Biotherapeutics SMAB include ...
(Date:12/18/2018)... ... December 17, 2018 , ... ABCT ... emerging biosciences ventures invited to participate in the annual program. Twelve ventures will ... plans and a professional network. All startups receive an entrepreneur coach, access to ...
Breaking Biology Technology:
Cached News: