Genome-Editing Tools Compose New Models of Autism
Genome sequencing studies of individuals with autism have left researchers with an embarrassment of riches: hundreds of single-letter mutations of uncertain relevance to the complex brain disorder.
Thanks to a suite of new tools in synthetic biology, it's nowpossible to test the relevance of these genetic glitches quickly andcheaply by inserting them into living cells in the lab and observing theeffects.
Synthetic biologists manipulate the body’s building blocks — DNA, RNAand proteins — to engineer structures that do not exist in the naturalworld.
On 15 February, two groups published back-to-back papers in Sciencedescribing a new synthetic technology known as CRISPR, for clusteredregularly interspaced short palindromic repeats. This method allowsresearchers to create molecular scissors that cut and paste essentiallyany mutation into the genome of any cell, including a human stem cell.
In the 9 May issue of Cell, researchers reported that they had used these molecular scissors to insert several mutations into mouse cells at the same time.
"What took us, in the past, three years to make — a triple-knockout mouse — we can do now basically in three weeks," says Rudolf Jaenisch, lead investigator of the Cell study and professor of biology at the Massachusetts Institute of Technology (MIT) and the Whitehead Institute.
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