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Spanish scientists develop New treatment method for Wilson disease by gene Therapy

Scientists at the Center for Applied Medical Research (CIMA) at the University of Navarra (Spain) have designed a promising gene therapy method to treat Wilson’s disease, a rare pathology caused by the lack of or malfunction of a gene.
With this promising method Dr. Gloria González Aseguinolaza, director of the Gene Therapy Program at CIMA, and her team have designed VTX801, a viral vector that corrects an advanced stage of Wilson’s disease in animals.
And, “it consists in applying a single injection whose effects may last up to seven years, which is, up to now, the follow-up time of a patient treated with gene therapy”.
However, the potential of this therapeutic strategy opens the doors to other treatments with gene therapy, as, by changing the defective gene, we may be able to address diseases with a similar cause”.
“Our strategy goes to the root of the disease and tries to correct it”, stated Dr. Gloria González Aseguinozala.

as declared in

New gene therapy for vision loss proven safe in humans

The researchers believe that in these patients, the immune system destroyed the virus before it could insert the therapeutic gene.
Participants were divided into five different groups that received increasing doses from 2X10^8 to 2X10^10 viral particles containing the therapeutic gene in 0.05 mL of fluid.
The virus penetrates retinal cells and deposits a gene, which turns the cells into factories for productions of a therapeutic protein, called sFLT01.
AMD is a leading cause of vision loss in the U.S., affecting an estimated 1.6 million Americans.
Eye specialists say the burden and discomfort of the regimen is responsible for many patients not getting injections as frequently as they need, causing vision loss.

New gene therapy
Spanish scientists develop New treatment method for Wilson disease by gene Therapy

as declared in

Experimental gene therapy proven safe for preserving vision of people with AMD

The researchers believe that in these patients, the immune system destroyed the virus before it could insert the therapeutic gene.
Participants were divided into five different groups that received increasing doses from 2X10^8 to 2X10^10 viral particles containing the therapeutic gene in 0.05 mL of fluid.
The virus penetrates retinal cells and deposits a gene, which turns the cells into factories for productions of a therapeutic protein, called sFLT01.
In a small and preliminary clinical trial, Johns Hopkins researchers and their collaborators have shown that an experimental gene therapy that uses viruses to introduce a therapeutic gene into the eye is safe and that it may be effective in preserving the vision of people with wet age-related macular degeneration (AMD).
AMD is a leading cause of vision loss in the U.S., affecting an estimated 1.6 million Americans.
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