CRISPR therapeutics (a LinkAGE webinar)

This LinkAGE webinar provides an overview of CRISPR therapies and the history of gene editing. It was presented by cellular therapeutics expert Dr James Patterson. We have provided timestamps, video chapters and transcriptions to help you find what you need. The webinar and Q&A was broadcast live on 21 February 2023.

By the end of this webinar, you should be able to:

- explain CRISPR and the history of gene editing;
- describe why Cas9 is the de facto gene editing system;
- summarise Cas enzymes and their potential use;
- describe the CRISPR therapeutics landscape; and
- reflect on the key challenges facing CRISPR’s use in the clinic and its future outlook.

What is the LinkAGE expert webinar series?
The Genomics Education Programme's LinkAGE (Linking Academia and Genomics Education) series will aim to provide expert seminars on exciting ‘bench-to-bedside’ projects that are likely to impact on clinical practice in the not-too-distant future.

Who is the LinkAGE expert webinar series aimed at?
This webinar series is aimed at researchers, clinicians and other healthcare professionals who already have a strong understanding of fundamental genomics concepts. If you are just starting your learning journey, or need a quick refresher, we encourage you to visit our website: www.genomicseducation.hee.nhs.uk

Meet the speaker
James Patterson, MB/PhD, currently works in Cambridge, UK on his start-up Xap Therapeutics. James studied medicine at Cambridge, where he spent most summers working on a diverse set of projects, from worms to cancer stem cells. He graduated with a degree in medicine/biochemistry before moving to the Francis Crick Institute to complete his MB/PhD on fission yeast cell size control with the Nobel prize winner Sir Paul Nurse. Separate to his academic work, James developed and built an IP focused biotech company – Auxolytic – which demonstrated proof of concept of a CRISPR-engineered cell therapy safety switch. In 2018, James founded Xap Therapeutics, a biotech company developing a CRISPR engineered, iPSC-derived delivery vehicle for complex therapeutics.

TIMESTAMPS
00:00 Introductions and talk overview
01:45 History and the present of gene editing
05:24 What is gene editing? How does gene editing work (in detail)?
10:59 The ZFN and TALEN gene editing systems
14:40 About the CRISPR/Cas system
21:50 CRISPR in practice
28:59 CRISPR use cases: from drug discovery to as a drug itself
35:10 CRISPR/Cas limitations
38:23 CRISPR's future uses: as a nickase or epigenome modifier
42:39 CRISPR in the clinic
45:45 CRISPR's economics and CRISPR ethics
49:40 Q&A and wrap-up