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Can CRISPR Cure Autism?

In the latest study, published by researchers from the University of California at Berkeley, the University of Texas, and GenEdit, have adapted CRISPR-Gold technology in order to treat autistic symptoms in mice.

Beating the Autistic Odds with CRISPR

One of the major highlights of the new research is the fact that CRISPR-cas9 could be a way to edit certain genes that relate to fragile X syndrome, which is a single-gene disorder frequently connected with autism, and autistic symptoms were improved on. Research provides additional evidence that conveyance of CRISPR-based therapeutics may be effectively accomplished via gold nanoparticles.

Mutations in the FMR1 gene is the cause of Fragile X syndrome. This gene plays a key role in encoding the protein, FMRP, the regulator in the production of different proteins and contributes markedly to the development of synapses.

Most of the time Fragile X occurs because of a mutation, which results in the enlargement of a part of the genetic code called CGG triplet repeat. This segment is repeated 5 to 40 times in healthy people, but patients with FXS, the CGG triplet may repeat over 200 times. This expansion hinders the gene from creating FMRP, which interferes with synapses and impacts nervous system functions, which in turn leads to behavioral, intellectual, and developmental disorders.

FXS is a health condition that mutations caused in a single gene, becoming an ideal candidate for CRISPR-based therapies. Th signs of FXS happen to match the excessive repetitive behaviors often used to diagnose autism, suggesting alleviating autism symptoms via genome engineering.

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