Autism traits may be edited out in the future using new genetic techniques: Scientists complete successful trial to change the way the brain works
- Scientists used gene editing technology to change the way the brain works
- They targeted habit-forming genes to reduce jumping and digging in mice
- The same therapy could be used to fight drug addiction or epilepsy, experts say
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Repetitive actions which are common in children with autism could be stopped by changing the way the brain works, research shows.
By using cutting-edge technology to edit DNA, scientists think they can reduce physical tics caused by autism, such as rocking back and forth or flapping the arms.
The gene-editing process involves changing DNA in the brain to change physical characteristics – in this case the habits which cause people’s impulsive movements.
A successful trial on laboratory mice showed the technique could reduce symptoms caused by one of the most common forms of autism by up to 70 per cent.
The team from the University of Texas hope the discovery will lead to more ways to tackle autism, which is thought to affect more than one in 100 children.
Scientists used a process called gene editing to change the DNA and genetics of mice with autism, and reduced their compulsive jumping and digging behaviour
Autism is a disability which affects the brains of children and changes the way they perceive the world and have relationships with other people.
There are various ways it affects people and some people are more affected than others, but it is commonly associated with a difficulty understanding emotions or taking things people say too literally.
Some 700,000 people in the UK and more than 3.5million Americans are thought to be autistic.
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The condition may also cause people to develop repetitive behaviours, which the scientists in this study aim to combat.
The autistic mice used by researchers had a habit of digging obsessively or leaping in the air.
Mice reduced compulsive digging and jumping
In the study a special kind of enzyme was sent into the mice’s brains where it changed the mouse’s DNA – a process called CRISPR gene editing.
DNA is a sequence of chemicals and the process effectively works by reordering the sequence, which then changes the genetics of the mouse – this can in turn change physical things about the animal.
By editing the genes of a specific part of the brain so they did not work, scientists were able to reduce the mice’s digging behaviour by around 30 per cent, and their jumping by about 70 per cent.
The researchers say this is the first time gene editing has been used in this way.
‘This is the first time we changed symptoms by editing a gene in the brain’
THE SIGNS AND SYMPTOMS OF AUTISM
According to the Centers for Disease Control and Prevention, people with autism have trouble with social, emotional and communication skills that usually develop before the age of three and last throughout a person’s life.
Specific signs of autism include:
- Reactions to smell, taste, look, feel or sound are unusual
- Difficulty adapting to changes in routine
- Unable to repeat or echo what is said to them
- Difficulty expressing desires using words or motions
- Unable to discuss their own feelings or other people’s
- Difficulty with acts of affection like hugging
- Prefer to be alone and avoid eye contact
- Difficulty relating to other people
- Unable to point at objects or look at objects when others point to them
Study leader Hye Young Lee, from the University of Texas said: ‘There are no treatments or cures for autism yet, and many of the clinical trials of small-molecule treatments targeting proteins that cause autism have failed.
‘This is the first case where we were able to edit a causal gene for autism in the brain and show rescue of the behavioral symptoms.’
Another expert who was not involved with the study, but invented the process, said it had caused ‘significant behavioural changes’.
The researchers think CRISPR could be used to tackle other brain problems like drug addiction, schizophrenia or epilepsy.
Autism spectrum disorder is thought to have various causes, including multiple genetic problems, which could make other types of the condition more difficult to tackle this way.
But it has potential for treating those whose autism is caused by a single faulty gene – such as people with fragile X syndrome, which the mice in the study had.
Process could also help fight drug addiction or epilepsy
Fragile X syndrome occurs in about one of every 4,000 boys and 6,000 girls.
The gene editing enzymes used by the scientists went to the area of the brain which is known to control habits, and changed DNA there.
Past studies have tried to use injected drugs to target the same area of the brain but have not been successful, the researchers say.
But the CRISPR enzymes can be used to target different types of cells and may also be useful in finding a way to treat muscular dystrophy by targeting muscle cells.
The team’s findings are published in the journal Nature Biomedical Engineering.
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