CRISPR Off-Targets | Wed. June 23, 2021 | 3:00 pm–4:30 pm CEST / 9:00 am–10:30 am EDT | Free CMN Live Webinar

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CRISPR Off-Targets

CRISPR-Cas and related genome-editing tools have been shown to induce so called off-target editing events. Recently, researchers working with CRISPR have found that besides the small off-target insertions and deletions (indels) that are typically addressed using short read next-generation sequencing (NGS) methods, a range of on-target events may also occur after genome editing, including translocations, large deletions and chromosomal rearrangements.

Our understanding about what happens at the target site and at unintended sites throughout the genome after genome editing increases continuously, thanks to the emergence of new analytical technologies and methods. These novel approaches will enable better evaluation of the safety profile for a given therapeutic genome-editing modality.

Our speakers will present CAST-Seq and CRISPECTOR, which are two of the newest tools for the detection and characterisation of unintended on- and off-target events after genome editing.

 

In this webinar, you will learn about:

  • Gene editing and the assays used to assess gene-editing outcomes such as off-target edits and chromosomal rearrangements
  • How CAST-Seq can be used to assess the safety of designer nucleases
  • The advantages of the CRISPECTOR tool for off-target analysis
  • How CRISPECTOR increases accuracy of off-target measurements and detects adverse structural variations and translocation events that occur during genome editing.

 

Webinar Programme:

15.00 Welcome and introduction by CRISPR Medicine News

15.05 Julia Klermund PhD, Postdoc at Institute for Transfusion Medicine and Gene Therapy, Freiburg, Germany (CMN interview)

15.30 Ayal Hendel PhD, Principal investigator at Bar-Ilan University, Israel (CMN interview)

15.55 Introduction of guest panelist Roberto Nitsch PhD, Ass. Director of Gene Therapy at AstraZeneca, Gothenburg, Sweden (CMN interview)

16.00 Panel discussion with questions from audience

16.25 Close by CRISPR Medicine News

 

Topics:

  • Julia Klermund, Postdoctoral Researcher at Institute for Transfusion Medicine and Gene Therapy, Freiburg, Germany - "CAST-Seq, a novel preclinical assay to detect chromosomal rearrangements after therapeutic genome editing"
  • Ayal Hendel, Principal investigator at Bar-Ilan University, Israel - "CRISPECTOR provides accurate estimation of genome editing translocation and off-target activity"
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Speakers

Julia Klermund, Postdoctoral Researcher at Institute for Transfusion Medicine and Gene Therapy, Freiburg, Germany

Julia Klermund, Postdoctoral Researcher at Institute for Transfusion Medicine and Gene Therapy, Freiburg, Germany

CAST-Seq, a novel preclinical assay to detect chromosomal rearrangements after therapeutic genome editing

Dr. Julia Klermund is a postdoctoral researcher in Toni Cathomen’s lab at the Institute for Transfusion Medicine and Gene Therapy, at University Hospital Freiburg in Germany. Julia obtained a BSc in Biomedical Science at Griffith University in Brisbane Australia, and an MSc and PhD in molecular and cellular biology at Heidelberg University.

Her current research focus is gene editing in human haematopoeitic stem cells and off-target assays, and she was a co-author on the recently published CAST-Seq assay, which was developed in the Cathomen lab. Julia has also held a postdoc position focused on CRISPR imaging at the Salk Institute for Biological Studies in San Diego. During her MSc she completed an internship with Professor Yihai Cao, studying tumour microenvironment factors at the Karolinska Institute in Sweden. Julia’s research has been published in a number of high-impact peer-reviewed journals.

Ayal Hendel, Principal investigator at Bar-Ilan University, Israel

Ayal Hendel, Principal investigator at Bar-Ilan University, Israel

CRISPECTOR provides accurate estimation of genome editing translocation and off-target activity

Dr. Ayal Hendel is a Principal investigator and a Senior Lecturer in the Mina and Everard Goodman Faculty of Life Sciences at Bar-Ilan University. His research focuses on developing genome editing as a cure for childhood disorders and for cancer immunotherapies. Dr. Hendel received his BA from the Hebrew University of Jerusalem in Biology and his MSc and PhD from the Weizmann Institute of Science.

His postdoctoral work with Dr. Matthew Porteus at Stanford University led to therapeutically relevant genome-editing frequencies in human stem cells and resulted in two pending patents. Dr. Hendel is a member of the 8400 Health Network, which aims to gather leaders at the intersection of health and technology to help Israel become a life science powerhouse. He has won a number of awards and grants for his research, most recently the Israel Precision Medicine Partnership grant of the Israel Science Foundation.

Roberto Nitsch, PhD, Associate Director – Gene Therapy, AstraZeneca, Sweden

Roberto Nitsch, PhD, Associate Director – Gene Therapy, AstraZeneca, Sweden

Roberto Nitsch graduated in Medical Biotechnology from the University of Naples (Italy) where he also obtained his PhD in Molecular Genetics. He later moved to Vienna where he focused on mouse genetics and cancer biology, and lately recessive genetics. He then shifted his research topic to genome engineering with CRISPR/Cas9 and joined AstraZeneca in 2014 where he was responsible of the CRISPR mouse models for drug discovery and oncology.

Since 2017, he is Associate Director in the Clinical Pharmacology and Safety Sciences pioneering CRISPR safety assessments. Today Roberto is leading the Gene Therapy safety group towards the generation of safer CRISPR medicines and the de-risk of AAV for therapeutic applications.

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