CRISPR Cas9 – Redesigning the Human Genome

Redesigning the Human Genome

With the advent of CRISPR Cas9, a precise genome cutting tool, it is now a reality that we can physically change the human genome. According to experts, we have the ability to cut out a section of our DNA enabling our cells to duplicate without the sequence that we removed. A removed sequence could be the code for a specific disease or mutation that we choose to eliminate. Scientists are also able to remove a section of DNA and replace it with a new sequence that has the specifications that we desire. This same approach could be used to replace a diseased sequence with a healthy one or to replace blue eyes with green.

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Scientists have been using genome editing technology since the 90’s to experiment with plants and even animals. The science and technology to do this work has been evolving and continues to be perfected. Up until recently though, almost all of the genome editing research focused on plants and small animals. The standards are much higher when working with humans; missing the target in a human is unacceptable. Now with CRISPR/Cas 9, researchers are able to precisely target and cut thus opening up the possibility to alter the human genome.

Scientists all over the world are focused on human genome editing. This work is almost entirely devoted to eliminating horrible human diseases such as HIV and sickle cell anemia in adult stem cells. Recently though, scientists in China have worked with human embryonic stem cells to try to eliminate the disease at its origination point. The research has sparked immense criticism and debate because, unlike adult stem cells, changes in embryonic cells will permanently change the genome for future generations.

This episode we bring Dr. Gang Bao, Foyt Family Chair of the Department of Engineering at Rice University and Director of the Nanomedicine Center for Nucleoprotein Machines. His lab, Bao lab, devotes its energies to gene targeting using engineered nucleases. His lab was the first in the world to recognize that CASPR Cas9 not only targeted the intended sequence, but also will unintentionally cut additional sequences across the genome that are identical or differ slightly from the intended target. A problem that needs to be resolved to use genome cutting as a reliable method for genetic modifications.

The discussion dives deep into the science; what we can currently achieve, how it works, and what he believes we will ultimately be able to do in the future. We also discuss the ethical consequences of genome editing and what world governments need to do as first steps to keep long term unintended and serious consequences from occurring.

You can see more of Dr. Gang Bao’s work at his Rice University lab website: http://bao.rice.edu/
You can see his bio here

Hear full interview on YouTube: