The authors of a paper, published in the journal Science, in an attempt to study the function of genes implicated in autism spectrum disorders (ASDs), applied a gene-editing and single-cell–sequencing system, Perturb-Seq, to knock out 35 ASD candidate genes in multiple mice embryos.
They described how the ‘Perturb-Seq’ method they developed can investigate the function of many different genes in many different cell types at once.
Directing the large-scale method to the study of dozens of genes that are associated with ASD, they identified how specific cell types in the developing mouse brain are impacted by mutations.
"The field has been limited by the sheer time and effort that it takes to make one model at a time to test the function of single genes. Now, we have shown the potential of studying gene function in a developing organism in a scalable way, which is an exciting first step to understanding the mechanisms that lead to autism spectrum disorder and other complex psychiatric conditions, and to eventually develop treatments for these devastating conditions," co-senior author Paola Arlotta, the Golub Family Professor of Stem Cell and Regenerative Biology at Harvard.
The method is also broadly applicable to other organs, enabling scientists to better understand a wide range of disease and normal processes, she said.
The study was also led by co-senior authors Aviv Regev, who was a core member of the Broad Institute during the study and is currently executive vice president of Genentech research and early development, and Feng Zhang, a core member of the Broad Institute and an investigator at MIT's McGovern Institute.
"exciting" - Google News
December 01, 2020 at 07:35PM
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Researchers make 'exciting first step' to better understanding development and treatment of autism - BioPharma-Reporter.com
"exciting" - Google News
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