DNA helix

Autism is a complex disorder that likely does not have a single cause.  There have been several genes that are currently linked to autism.  Perhaps a bit of genetic tweaking could lessen or completely relieve the associated symptoms.

A new study shows promise to manage or reverse autism behaviors.  They achieved this by manipulating a single gene in young and old mice.  

“This suggests that even in the adult brain we have profound plasticity to some degree,” lead researcher Guoping Feng from the Massachusetts Institute of Technology (MIT) said in a statement. “There is more and more evidence showing that some of the defects are indeed reversible, giving hope that we can develop treatment for autistic patients in the future.”

Known as Shank3, this gene appears to be the link to autism behaviors.  Shank3 has the instructions for a protein found at the synapses between nerve cells.   

DNA helix

Shank3 has been noticed as mutated or even missing for a percentage of those on the autistic spectrum.  It is unclear how this affects the condition but the connection is clear.  By deleting Shank3, mice started developing autism-like behavior with deficits in social interactions and repetitive actions.

The testing on this was quite interesting.

Symptoms of autism usually appear at an early age, so scientists wanted a way to turn the gene on and off to see how the mice responded.  They derived a system to turn the gene “on” when they administered a drug called tamoxifen.

As it was described in Nature, when they turned on the gene, the social and repetitive behaviors of the mice reversed. They also noticed improved synapse function and the number of dendrites.  But in adult mice they did not observe complete reversal of coordination and anxiety.  However, if they turned the gene on within 20 days of birth, these functions reached the normal levels.  Early addressing of these genes appears to develop these skills while the neural circuits are still malleable.

While Snank3 is not associated with all cases of autism, it does show the possibility of gene manipulation and gene therapy to reversing or preventing the symptoms of autism.

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