How could CRISPR-Cas9 accelerate our discovery of treatments for genetic brain disorders?

In this episode, we take a look at how AAV-Perturb-Seq can change the way scientists study genetic disorders affecting the brain and other organs. Using adeno-associated viruses (AAVs) to deliver guide-RNA libraries for CRISPR-Cas9, this technique can help us understand complex conditions like 22q11.2 deletion syndrome and potentially uncover underlying disease mechanisms at single-cell resolution.

Antonio Santinha is a postdoctoral researcher at the D-BSSE of ETH Zurich in Basel, Switzerland, where he continues leading work combining CRISPR and single-cell omics techniques to study brain disorders.

Read the paper on AAV-Perturb-Seq:

Santinha, A.J., Klingler, E., Kuhn, M. et al. Transcriptional linkage analysis with in vivo AAV-Perturb-seq. Nature 622, 367–375 (2023). https://doi.org/10.1038/s41586-023-06570-y

Introduction: to min. 16:00