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Therapeutic Human Gene Editing with Dr. David Liu

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Over the past five to 10 years, development in the field of human gene editing has advanced at an unprecedented speed. We are now living in an age where therapeutic human gene editing is already a reality for some of us. Today we are joined by Dr. David Liu to discuss these exciting developments. Dr. Liu is a Richard Merkin Professor, Director of the Merkin Institute of Transformative Technologies and Healthcare, Vice-Chair of the Faculty at the Broad Institute of Harvard and MIT, and the Co-founder or Founder of nine biotech or therapeutics companies. He's published over 195 scientific papers and is the inventor of over 75 issued patents. Today he introduces us to the three main forms of modern genome editing, how they work, their benefits, and their limitations. He explains how the development of CRISPR had an enormous impact on the reach and scope of this field and describes some of the massive developments achieved by his labs in base editing and prime editing. Tune in today to hear how these different types of modalities might be best suited for different types of therapeutic interventions and Dr. Liu’s thoughts on whether or not one method will replace another. Find out if either of these technologies is more amenable to addressing polygenic diseases, the roles and complications of viral and non-viral delivery vectors, and how Dr. Liu believes these technologies will extend in the future. For all this and more, tune in today!

Key Points From This Episode:

- Dr. David Liu’s background and some of his impressive credentials.
- An introduction to gene editing and how Dr. Liu became interested in it.
- The groundbreaking development made by Peter Dervan and how this influenced the field.
- Dr. Liu’s Unifactor 2000 project to develop a series of RNA molecules that could target any site in the genome and why it failed.
- An introduction to the three main forms of modern genome editing.
- What zinc fingers are and how they can be used to bind a DNA sequence of one's choosing.
- What tail nucleases are and how tail proteins can bind a DNA sequence of one's choosing.
- The breakthrough that came with the CRISPR system and how it initiated this modern gene-editing renaissance.
- How the ease and accessibility of CRISPR has had an enormous impact on the reach and the scope of these developments.
- What base editing and prime editing are and how they work together.
- Some of the outcomes that can occur when you cut the DNA in a chromosome.
- The two realizations that motivated Dr. Liu’s lab’s developments of base editing and prime editing.
- Dr. Liu explains in detail how base editors and prime editors change DNA.
- How Dr. Liu and his lab took the development of the CRISPR approach to develop these new techniques and how this was influenced by Homology Directed Repair.
- What different types of modalities may be best suited for different types of therapeutic interventions.
- Why one would pursue a CRISPR double-stranded break-based method versus doing base editing or prime editing.
- How the companies Prime Medicine and Beam that have licensed these methods work together in the best interests of the patient.
- Dr. Liu’s thoughts on whether or not prime editing is going to replace base editing.
- Insight into the need for PAM sequences in Cas9 and the advantage of prime editing as it does not need a specific PAM sequence.
- How Dr. Liu believes these technologies will extend in the future.
- Thoughts on whether or not one flavor of these technologies is more amenable to addressing polygenic diseases.
- What a delivery vehicle is, the differences between viral and non-viral vehicles, and the complications of using viral delivery vectors.
- Concerns with regard to immunogenicity and immune reaction from an LMP and how this relates to the COVID vaccine.

Tweetables:

“There are already humans walking this earth that have had their genomes modified, specifically in a way to counteract a genetic disease. That's really a pretty momentous era in the history of man.” — @davidrliu [0:10:37]

“The motivation behind base editing was the simple recognition that for most genetic diseases, to study the disease or to treat the disease, we can't really do that well if we just mess up the gene. We need to actually precisely fix it.” — @davidrliu [0:13:10]

“I think that's one of the potential downsides of viral delivery is that whenever you're introducing new DNA into a cell, you have to be very careful to minimize the possibility that the DNA gets in and initiates cancer.” — @davidrliu [0:56:33]

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