Transcriptional linkage analysis with in vivo AAV-Perturb-seq | Wednesday November 1, 2023 | 3:00 pm–4:00 pm CET / 9:00 am–10:00 am EDT

Transcriptional linkage analysis with in vivo AAV-Perturb-seq

To facilitate high-throughput genotype-phenotype correlations in complex tissues, a team led by Randall Platt PhD, Professor at the Department of Biosystems Science and Engineering at ETH Zurich, developed adeno-associated virus (AAV)-mediated direct in vivo single-cell CRISPR screening or AAV-Perturb-seq.

In an article published in Nature, AAV-Perturb-seq is presented as a tuneable and widely applicable approach to transcriptional linkage analysis as well as high-throughput and high-resolution phenotyping of genetic alterations in vivo.

The team applied the method using CRISPR-Cas9 gene editing and transcriptional inhibition to systematically probe the phenotypic landscape underlying 22q11.2 deletion syndrome in the prefrontal cortex of adult mouse brains. These experiments led to the identification of three 22q11.2-linked genes involved in known as well as previously undescribed pathways regulating neuronal functions in vivo that explain about 40% of the transcriptional changes seen in a 22q11.2-deletion mouse model.

The outcome of this work so far suggests that the 22q11.2-deletion syndrome transcriptional phenotype observed in mature neurons might partially be explained by the broad dysregulation of a class of genes associated with disease susceptibility that are important for dysfunctional RNA processing and synaptic function.

In this webinar, Dr. Antonio Santinha, PhD and first author on the recent Nature article, will present AAV-Perturb-seq, and illustrate how it may be deployed as a flexible and scalable direct in vivo tool to understand genotype-phenotype correlations in the context of disease, with potential applications in identifying novel therapeutic targets.

 

What you will learn about in this webinar:

  • Screening
  • scRNA-seq
  • Neuroscience

 

Webinar Programme (CEST time zone):

  • 15.00 Welcome and introduction by Karen O'Hanlon Cohrt PhD., Editor-in-chief, CRISPR Medicine News
  • 15.05 Dr. Antonio Santinha, Ph.D., / Scientist, ETH Zürich, Switzerland | Transcriptional linkage analysis with in vivo AAV-Perturb-seq
  • 15.45 Q & A with Dr. Antonio Santinha
  • 16.00 Close by CRISPR Medicine News

 

Speaker | Title:

Dr. Antonio Santinha, Ph.D., / Scientist, ETH Zürich, Switzerland | Transcriptional linkage analysis with in vivo AAV-Perturb-seq

Speakers

Dr. Antonio Santinha, Ph.D., / Scientist, ETH Zürich, Switzerland

Dr. Antonio Santinha, Ph.D., / Scientist, ETH Zürich, Switzerland

Dr. Antonio Santinha is a postdoctoral researcher at ETH Zurich, who is working towards finding the best therapeutic interventions for the most prevalent human diseases, using high-parallel approaches. Antonio recently completed his PhD at ETH Zurich under the supervision of Dr. Randall Platt. His doctoral work focused on developing new high-throughput and information-rich techniques to explore genetic functions in vivo. His most recent work led to the development of AAV-Perturb-seq, a highly scalable technology for in vivo CRISPR screening with single-cell readouts in mouse models. Before joining the Platt Lab, Antonio obtained his BSc in Biochemistry and an MSc in Cellular and Molecular Biology from the University of Coimbra, Portugal.

CMN Webinar - Transcriptional linkage analysis with in vivo AAV-Perturb-seq
CMN Articles - CRISPR-Cas9

 

 

Chronic Granulomatous Disease, CGD, (NCT06325709)
Sponsors:
National Institute of Allergy and Infectious Diseases (NIAID)
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT06287099)
Sponsors:
Bioray Laboratories
IND Enabling
Phase I
Phase II
Phase III
IND Enabling
Phase I
Phase II
Phase III
IND Enabling
Phase I
Phase II
Phase III
Indicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT06300723)
Sponsors:
Bioray Laboratories
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT06287086)
Sponsors:
Bioray Laboratories
IND Enabling
Phase I
Phase II
Phase III
CMN Webinars - Your Gateway to CRISPR Medicine

If you have any questions or request for webinars, please contact us at: sales@crisprmedicinenews.com

Future CMN Webinars will be listed here.

Always stay updated - Sign up to weekly newsletter.

close
Search CRISPR Medicine