Ep 35: How CRISPR Gene Therapy is Bringing Hope to the World

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Every year, approximately 8 million children are born with a serious genetic disorder, and 3 million of them die before the age of 5. This disease burden is about to change. In this episode, we launch the CRISPR Chronicles series that will run throughout season 3. Since the pivotal paper by Doudna and Charpentier in 2012, CRISPR has taken the world by storm. Scientists have used this genome engineering tool in the lab to quickly and easily create mutants to study gene function in laboratory animals. But more importantly, the power of CRISPR gene editing as a biomedical intervention to cure diseases has been realized. Currently, dozens of clinical trials are ongoing or on the verge of being launched to cure everything from genetic blindness and sickle cell anemia, to cancers and HIV. In fact, the Sickle Cell Disease treatment, exa-cel is poised to become the first CRISPR gene editing therapy to be approved by the FDA. Due to its far-reaching impacts, Doudna and Charpentier won the Nobel Prize in chemistry in 2020 for discovery of CRISPR-Cas9 gene editing; thereby, breaking the boundary as dual female winners of this prize.
In this series we will explore:

• How CRISPR gene editing works
• The CRISPR origin story and major milestones
• The many clinical trials giving hope to the millions of people worldwide suffering from cancers, and genetic and infectious diseases
• The ethical debate of using CRISPR gene editing technology

For more information on this topic, visit our website: welovesciencepodcast.com
Hear directly from Sickle Cell Disease patients who were cured during the clinical trial: Victoria Gray and Jimi Olaghere
Jump directly to the next episodes in the CRISPR series:

• Ep 38: A foundational discussion explaining the secret behind CRISPR gene therapy
• Ep 41: A look into the ups and downs of how the CRISPR saga developed
• Ep 47: The CRISPR Cure for Sickle Cell Disease

Other Great episodes:

• Ep 15: Dr Joseph Iacona on the drug development process in pharma
• Ep 23:

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