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CRISPRbabies - talk and panel discussion

CMCB LifeScience Seminar for Scientists

Date:11/04/2019, 11:00 - 12:00
Speaker: Prof. Frank Buchholz & Prof. Min-Ae Lee Kirsch, University Hospital Dresden
Location: CRTD, auditorium

 

Speaker: Prof. Dr. Frank Buchholz, TU Dresden, Faculty of Medicine, University Cancer Center

Title: "Programmable nucleases and designer recombinases for genome surgery"

Abstract: Recent breakthroughs in the field of genome editing provide a genuine opportunity to establish innovative gene and cell therapy approaches to repair DNA lesions to replace, engineer or regenerate malfunctioning cells in vitro, or directly in the human body. Most of the recently developed genome editing technologies introduce double stranded DNA breaks at a target locus. These breaks are subsequently repaired by one of the cell intrinsic DNA repair mechanisms, typically inducing an abundance of random insertions and deletions (indels) at the target locus. We have employed the CRISPR/Cas9 system to utilize this property to investigate cancer mutations. How this approach can be used to dissect driver mutations from passenger mutations and how this knowledge could help to improve personalized therapies will be discussed.

For other applications, therapeutic genome editing should, however, be efficient and specific, without the introduction of indels. Site-specific recombinases (SSRs) allow genome editing without triggering cell intrinsic DNA repair pathways as these enzymes fulfill both cleavage and immediate resealing of the processed DNA, allowing precise, predictable and efficient genome editing in vivo. We use substrate-linked directed evolution coupled with rational design to program SSRs to target therapeutically relevant human genomic sites. Examples of our work will be presented, including the development and application of the broad-range anti-HIV-1 recombinase, Brec1. Because Brec1 efficiently and specifically eradicates the integrated HIV-1 provirus in humanized PDX models, it represents a promising new agent with curative potential.

Speaker: Prof. Dr. Min Ae Lee Kirsch, UKD, Clinic and Policlinic for Children and Youth Medicine

Title: "Therapeutic genome editing - promise and peril"

Abstract: Gene editing technologies pose significant implications for human health and disease. At the same time this technological innovation fundamentally impacts our role and responsibility as scientists in the societal and ethical debate as well as in policy-making processes. What are the real and perceived potential benefits and problems created by gene editing? How can we manage risks and benefits?