The Science and Ethics of Genome Editing - Professor Jennifer Doudna

Presentation to Convergence Science Network by Professor Jennifer Doudna, 13 February 2018.

The much publicised CRISPR/Cas9 gene editing is the holy grail in Genetics. Gene editing is providing the capacity to make any conceivable change to any gene in a wide variety of organisms.
It is being used around the world to understand the genetic basis of human diseases, to address agricultural and environmental challenges and in basic biological research. Most of these applications are not considered contentious, but what of the potential to modify human genomes or to eliminate pest species?  How far should we go in the use of gene editing?  In the first Convergence Science Network event of 2018, two leading international authorities, Jennifer Doudna and Kevin Esvelt, will talk about applications and the drawing of ethical boundaries in the use of gene editing.
CRISPR (Clustered Regularly Interspersed Palindromic Repeats) is a tool that enables scientists to edit, remove or replace the genes of many organisms, including humans. This technology is being widely used in model organisms and cultured human cells, accelerating research aimed at finding treatments for human disease.   But what of using gene editing itself to be the cure? Just two weeks ago, scientists in the US attempted to edit genes inside the body of a man who suffers from Hunter Syndrome, an incurable disease.  In February, CRISPR/Cas9 was used by the Francis Crick Institute in London to modify human embryos. As CRISPR could potentially be used to edit cells that give rise to sperm and eggs, ethical questions about humankind’s ability to alter human heredity are being asked.   
The tools of gene editing can also be rendered heritable so that a genetic change could be driven to spread throughout a population.  The use of ‘Gene Drive’ technology has been discussed as a possible strategy for the control of mosquitos that vector deadly diseases such as malaria and dengue fever.  But, are there risks in using Gene Drive technology?
What is CRISPR and why is it causing such a storm in the scientific community and receiving greater attention from government, ethicists, businesses and the public?  Are the promises for genome editing to cure genetic diseases in humans realistic?  What advances in gene editing are needed before it can be confidently applied to humans?  What limits and safeguards need to be considered in the use of this technology?

Professor Jennifer Doudna
Jennifer Doudna grew up in rural Hawaii, where she first became interested in the chemistry of living systems. Dr Doudna is currently the Li Ka Shing Chancellor’s Chair in Biomedical and Health Sciences and she is Professor of Molecular and Cell Biology and Professor of Chemistry at UC Berkeley and an Investigator of the Howard Hughes Medical Institute.  Professor Doudna’s research seeks to understand how RNA molecules control the expression of genetic information. Early in her career, Dr Doudna’s lab determined some of the first crystal structures of RNA and RNA-protein complexes, providing unprecedented insights into molecular function of non-protein-coding RNAs. More recently she and collaborator Emmanuelle Charpentier determined the mechanism of RNA-guided bacterial adaptive immunity by the CRISPR-Cas9 system, enabling them to harness this system for efficient genome engineering in animals and plants. CRISPR-Cas9 is a transformative technology that is revolutionizing the fields of genetics, molecular biology and medicine. Dr Doudna is a recipient of numerous awards and is a member of the National Academy of Sciences, the American Academy of Arts and Sciences, the National Academy of Medicine and the National Academy of Inventors.
Doudna has been widely acclaimed by the scientific community for her fundamental contributions to the fields of biochemistry and genetics, receiving many prestigious awards and fellowships. She was awarded the 2000 Alan T. Waterman Award for her research on ribozyme, and 2015 Breakthrough Prize in Life Sciences for her contributions to CRISPR/Cas9 genome editing technology (with Charpentier). She has also been a co-recipient of the Gruber Prize in Genetics (2015), the Canada Gairdner International Award (2016) and the Japan Prize (2017). She has also been recognized outside the scientific community, being named one of the Time 100 most influential people in 2015 (with Charpentier) and listed as a runner-up for Time Person of the Year in 2016 alongside other CRISPR researchers.