Summary
Gene therapy is advancing toward reversing aging biomarkers and making cells completely virus-resistant. Covers the latest beyond-CRISPR editing tools, multiplexed editing that changes millions of sites at once, and why pig organ transplants may soon solve the donor shortage.
Key Points
- Base editing represents an advancement beyond traditional CRISPR/Cas9 with fewer off-target effects
- Multiplexed genome editing can make thousands to millions of edits per cell simultaneously
- Genetic recoding could make cells completely resistant to all viruses by altering the host genetic code
- Genome Project-Write aims to synthesize entire genomes from scratch for therapeutic applications
- Gene therapy approaches show promise for reversing aging biomarkers in animal models
- Xenotransplantation using engineered pig organs may solve the organ shortage crisis
- Gene drive technology could eliminate disease-carrying mosquitoes and other vectors
Key Moments
Gene Therapy Discussion
Today's episode is an incredible treat for those of you among us that are true, bona fide believers in the ability of technical innovations to potentially change the world and even foundations of biology and...
"We discussed the advance from CRISPR Cas9 gene editing to what is known as base editing and why base editing may be the key to unlock the full potential of gene editing, taking us from a technology..."
Gene Therapy: How To
You know, clean water, roads, you know, cell phones are getting accessible in remote parts of the world.
"So you can think of CRISPR as a subset of editing. Editing is a subset of genome engineering. And genome engineering is not a subset of, but it's kind of a Venn diagram overlapping set with therapies and GMOs and so forth."
Alzheimer Prevention Discussion
Pigs are very close to humans in their organs. That's why they're being used as transplants, but they're also imperfect.
"If I remember correctly, you enhanced the brain organoid to...I think you edited it from APOE4, which if you're homozygous, you have like a 20-fold increased risk for Alzheimer's to APOE3. Right."
Gene Therapy Discussion
And, you know, I'm just sort of interested in the public response to that sort of medical technology and use of it versus the CRISPR editor babies in 2018. If they were proportional.
"And, you know, I'm just sort of interested in the public response to that sort of medical technology and use of it versus the CRISPR editor babies in 2018. If they were proportional."
Vaccine Science Discussion
It's a set of technologies, tools, possessions. That multiplex editing will be something that won't be germalined, but it will be just as surely inherited.
"It's a set of technologies, tools, possessions. That multiplex editing will be something that won't be germalined, but it will be just as surely inherited. Right."
Gene Therapy Discussion
The latest round of vaccines are kind of in a format of gene therapy and are very inexpensive compared to most gene therapies that are typically $2 million.
"The latest round of vaccines are kind of in a format of gene therapy and are very inexpensive compared to most gene therapies that are typically $2 million."
Vaccine Science Discussion
So, I think one approach to...so the extinction of species is one part of it. So, for example, you could make mosquitoes resistant to malaria.
"So, I think one approach to...so the extinction of species is one part of it. So So you can do gene drives whose intention is to make a species resistant to something that's bad for a third species."
