Ожидаемая новость. Технологию редактирования генома CRISPR-Cas9 попробовали применить к расстройствам аутического спектра. На мышах, естественно.
CRISPR Editing Reduces Repetitive Behavior in Mice with a Form of Autism. О самом эксперименте в заметке буквально пара абзацев:
In the experiment on mice with FXS, the researchers injected CRISPR-Gold carrying the Cas9 complex into the striatum of the brain, a region known to mediate habit formation, including that related to repetitive behaviors common to ASD. The Cas9 targeted an excitatory receptor, the metabotropic glutamate receptor 5 (mGluR5), that is involved in communication between neurons and is known to be dysregulated in FXS. By disabling the gene for mGluR5, the researchers were able to dampen exaggerated signaling between cells and lessen repetitive behavior.
“Before this experiment, we didn’t know if the mGluR5 receptor in the striatum was specifically involved in exaggerated repetitive behavior; that is an important biological finding of our study,” Lee said.
In mice with FXS, the repetitive behavior included obsessive digging and periodic leaps into the air. The digging was reduced by about 30 percent, while the leaping went down 70 percent. Approximately 50 percent of the mGluR5 genes in the striatum were edited, which cut the number of receptor proteins by nearly half.
В основном написано о модификации метода с применением золотых частиц в противоположность традиционному вирусному. Последнее время технологию CRISPR-Cas9 критикуют. В одних материалах находят многочисленные нецелевые мутации, правда без подтверждения. В другой
статье была информация, что метод действует в клетках, в которых не работает внутренняя защита от изменений и такие клетки могут превращаться в раковые.