How much do you know about Huntington’s disease? What are the symptoms of this complication in different people? This complication is an inherited disease for which no cure has been found so far. In various experiments, researchers have succeeded in genetically manipulating this complication. We will provide this context.
Huntington’s is a genetic disease for which no cure has been found, according to IRNA on Monday. The gene for the disease is inherited predominantly, and any parent who carries the gene can pass it on to their children with a 50 percent chance.
However, if a child does not receive the disease from any of their carrier parents, the disease will not be transmitted to their children.
The cause of the disease progresses slowly and steadily until it manifests itself in the third or fourth decade of life. Usually between the ages of 15 and 20, the symptoms of the disease develop to such an extent that the patient becomes completely incapacitated and eventually leads to his death.
Researchers at Imperial College London in a study on mice have developed a new treatment for the disease that can stop the activity of a genetic mutation that is responsible for severe muscle problems and involuntary movements for several months.
They hope that this protein-based treatment – applied through a single injection – will be tested in humans over the next five years if promising results in future studies.
The researchers developed the new treatment based on an engineered protein called zinc finger.
The “zinc fingers” are designed to attach to DNAs of Huntington’s mutated gene transcripts, effectively blocking their ability to express and produce harmful proteins that accumulate in the brain.
In this experiment, researchers treated 12 mice with Huntington’s disease by providing a single injection of zinc finger.
The researchers used brain scans to assess the progression of the disease after treatment. They found that after just three weeks, an average of 77% of the expression of the mutated gene was suppressed. Unwanted genes were suppressed by 48% in the twelfth week.
The researchers found that by 24 weeks, 23 percent of the unwanted genes were still suppressed, with some effects seen in some mice for up to six months after the initial injection.
The results of this study are published in the journal Molecular Neurodegeneration.