Scientists have used a herd of genetically engineered sheep to identify a promising treatment for a deadly genetic brain disease affecting young children.
Scientists from the UK and the US say their work could lead to the development of drugs to alleviate childhood Batten’s disease.
Batten’s disease is known as a rare genetic disease that causes disturbances at the level of the nervous system and the constant deterioration of the patient’s body systems, which ultimately leads to blindness, loss of speech, paralysis, and then early death.
It is inherited from two asymptomatic parents, each of whom is a carrier of a rare recessive genetic mutation. Children who carry two copies of this defective gene begin to suffer vision loss, cognitive impairment and movement problems, followed by seizures and early death.
While there is no cure for Batten disease, medical professionals are focusing on treating the symptoms to make life easier for carriers of the defective gene.
Scientists led by Professor Jonathan Cooper from Washington University School of Medicine in St. LouisIn lab tests with colleagues at Collaborations Pharmaceuticals, they did their initial experiments on mice that had a form of Batten’s disease known as CLN1 that could be treated with the missing enzyme.
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Study leader Professor Tom Wishart of the University of Edinburgh’s Roslyn Institute, where cloning techniques were used to create Dolly the sheep in 1996, said: “It was encouraging, but we needed to test the treatment on larger brains with a structure very similar to that of a child. It cannot be extrapolated directly from mice to humans. It is important to have a larger intermediate model.”
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So the project scientists used Crispr-Cas9 gene-editing technology to create a copy of the defective gene responsible for CLN1 in sheep.
The scientists were able to create a small herd of sheep, each designed to carry one functional copy of the CLN1 gene.
“Sheep ovaries were collected from slaughterhouses, the eggs were removed and fertilized. Crispr reagents were then added to make the necessary changes to CLN1, and then the eggs were implanted in surrogate sheep,” Wishart said.
“They are asymptomatic carriers, just like the parents of children with Batten’s disease. With this herd, we can raise sheep with two defective copies. These sheep continue to develop disease like children and are the subjects of our clinical trials.”
Children succumb to this form of Batten’s disease because they lack an enzyme produced by healthy CLN1 genes, so the children develop Batten’s disease. This enzyme is needed so that lysosomes (organelles in animal cells that contain digestive enzymes that break down excess or lost organelles, food, viruses, and bacteria) can process the waste that accumulates in cells. This process is difficult in Batten’s disease.
Studies done on mice have shown that injecting the missing enzyme into the brain resulted in significant improvements. The group found that going directly to human trials was neither possible nor safe.
“Two important questions can be overlooked: how to get the drug to the right place in the big brain and how to increase the doses,” Cooper added.
The team got the answers by experimenting with half a dozen sheep raised in a herd of sheep with two defective CLN1 genes.
The results of the experiments revealed many signs of human disease. According to a research article published in the journal, by calculating the appropriate dose and how it is delivered to the sheep’s brain, scientists can see improvements in the disease. of Clinical study.
The scientists say the results are promising, but stress that several years of research is still needed to improve treatments.
“We have received amazing information that will one day help develop treatments for children,” said Wishart.
Cooper echoed Wishart’s point, saying, “We still have a long way to go, but we have taken a very important step.”
Source: Guardian
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