Precision Medicine, GMOs, Designer Babies, and the Future of Gene Editing
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This document discusses the future possibilities of CRISPR gene editing technology. It provides an overview of gene editing tools like zinc finger nucleases, TALENs, and CRISPR/Cas9. CRISPR is highlighted as being extremely efficient, able to multiplex, insert, delete or alter DNA sequences, relatively inexpensive, and simple to use. Potential applications discussed include curing diseases like cancer and malaria, genetically modifying animals, and editing human embryos. However, challenges like off-target effects, delivery mechanisms, patent battles, and ethical issues still need to be addressed before clinical use in humans. The document encourages further exploration of CRISPR's potential for treating inherited diseases.
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Precision Medicine, GMOs, Designer Babies, and the Future of Gene Editing
- 1. Precision Medicine, GMOs, Designer Babies, and the Future ofGene Editing Exploring the Possibilities of CRISPR Adrianne Stone
- 2. You want to do what to my genes? Gene Editing process of adding, removing, modifying, or replacing the DNA of an organism
- 3. Gene Editing Toolbox Zn Finger Nucleases TALENs CRISPR/Cas9
- 9. So what’s the big deal about CRISPR? 1. Extremely efficient 2. Multiplexing ability 3. Insert, delete, or alter any sequence 4. Relatively inexpensive 5. Very simple to use
- 12. How CRISPR/ Cas9Works (VIDEO) Please enjoy the following video from the journal Nature about CRISPR Cas9. To view at your own leisure visit medicineofme.com/crispr-brca
- 16. So what’s the hold up? Off-Target Effects Delivery Mechanism
- 18. Ethical Issues
- 20. Questions? For more information on CRISPR and the latest news on gene editing for hereditary diseases please see the page I created especially for attendees of this conference medicineofme.com/crispr-brca @adrianneelayne
Editor's Notes
- Define gene editing – accomplished through use of nucleases (enzymes that cut DNA)
- 1991 – First engineered nuclease (Zn Finger) published. Dominant technology for about 10 years but was imperfect difficulty with certain or large targets Very expensive to produce ($4-7k/ZFN) TALEN’s introduced in early aughts Popular because could be made “in-house” by labs so less expensive and faster Larger molecules however mean harder to use in some application CRISPR/Cas9 splashed onto the scene in 2012 and the field exploded
- Unlike other Car-T cell therapies, this approach removed certain receptors from generic donor T cells to create “invisible” CAR-T cells that didn’t attack the host’s body.
- Used a gene drive to insert two anti-malarial parasite genes into mosquitos. Gene drive insures that nearly 100% of progeny carry the
- A Chinese research team landed in hot water recently for announcing they had used CRISPR to edit defective human embryos with the blood disease mutation for Beta Thalassemia that they had obtained from local IVF facilities. They were not implanted and only a few of the 86 embryos tested actually had the gene correction. Beta Thalassemia is caused by a single nucleotide change and a good candidate for this type of treatment, but many in the scientific community feel it is far too early to begin work on human embryos
- This one is also in China They have really adopted CRISPR to make customized animals Goats – larger muscles and longer hair Dogs – muscles Sheep – muscles Pigs – consumer-sold micro pigs Monkeys – producing disease models for neurological conditions
- Stands for “Clustered Regularly Interspaced Short Palindromic Repeats” (you can see why we use CRISPR) Region of DNA in the Bacterial genome that keeps a copy of
- Sickle cell anemia Muscular Dystrophy Congenital Blindness Cancer (Lung Cancer/ CarT Cell therapy) HIV
- Using alternative versions of Cas proteins can generate novel uses. Using these new systems we can detect viral, bacterial, or tumor DNA from samples and either cleave an additional synthetic DNA piece to release a fluorescent signal (DETECTR) or a pregnancy-test like paper strip (SHERLOCK) Also using CRISPR to label cells in a living organism (ex. Tumor cells)
- OTE Delivery – viral? Nanoparticles? Some tissues easier than others (ex. blood) Also incomplete understanding of exact genetic causes of certain traits (so no designer babies any time soon)
- Designer Babies Rampant gene drives De-extinction
- CRISPR applications to HBOCs