Baby KJ: Personalized CRISPR Base Editing

Broadcast from KSQD, Santa Cruz on 1-01-2025:

• An emailer asks about omega-3 supplementation for memory at age 72. Dr. Dawn advises checking that fish oil capsules contain adequate DHA—at least 1,000 mg—since many omega-3 products have low DHA levels. She notes Medicare covers the same testing at standard labs as proprietary labs like OmegaQuant that charge patients directly. Beyond omega-3s, she emphasizes glucose control (hemoglobin A1c below 5.6) since the enzyme that breaks down insulin also clears beta-amyloid, and weight training to raise brain-derived neurotrophic factor (BDNF), which promotes new synapse formation essential for memory.
• Dr. Dawn reviews Popular Science's top 2025 health innovation: eye drops from Lens Therapeutics containing aceclidine that correct age-related farsightedness for 10 hours. The drops shrink the pupil to increase depth of field, improving near vision by three or more lines on eye charts within 30 minutes without affecting distance vision. Side effects include eye irritation, dimmed night vision, and headache.
• She describes Duke University's breakthrough allowing heart transplants from circulatory death donors using an on-table reanimation technique. This could expand the pediatric donor pool by 20%—critical since up to 20% of children die waiting for transplants.
• Dr. Dawn celebrates CAR-T immunotherapy for multiple myeloma, which saved her husband's life. Of 97 heavily pretreated patients, 38% achieved complete remission still present at five years, with over 50% total survival. The therapy removes T-cells, uses CRISPR to add receptors targeting cancer cell antigens, then reinfuses the modified cells.
• She highlights a UC Davis study showing remote blood pressure monitoring with home technology, education, and coaching dropped patients' average blood pressure from 150/80 to 125/74 in months—low-tech with high impact.
• Dr. Dawn explains the Nano Knife for prostate cancer, which uses localized electrical pulses delivered through thin wires to destroy tumors while sparing surrounding nerves. This minimally invasive approach could reduce erectile dysfunction and incontinence common with traditional surgery.
• She describes Gilead's Sunlenca, a twice-yearly injection for HIV prevention that's 99% effective. At $14,000 per injection in the US, proceeds help fund access in resource-limited countries where it can be distributed like a vaccination.
• Dr. Dawn discusses Journavx (suzetrigine), a new non-opioid pain medication working on sodium channels to block pain signals before reaching the brain. At $30 for 50 pills on GoodRx, it offers an alternative for surgical pain in patients with addiction history or genetic vulnerability to opioid dependence.
• She details the landmark case of Baby KJ, the first person to receive personalized CRISPR gene therapy. Born with a CPS1 enzyme deficiency causing toxic ammonia buildup, KJ was too small for liver transplant. Scientists identified his specific mutation and used CRISPR base editing delivered via lipid nanoparticles to correct a single DNA letter—changing an A to G—in his liver cells which restored enough function to be discharged home.
• Dr. Dawn reports surprising findings that COVID mRNA vaccines amplify cancer immunotherapy. Lung cancer patients who received COVID vaccination within 100 days of checkpoint inhibitor treatment had 56% three-year survival versus 31% for unvaccinated patients. The mechanism is unknown but may involve mRNA generally alerting the immune system.
• She revisits research showing Zostavax shingles vaccination reduced dementia risk by 20% over seven years. A natural experiment in Wales—where an age cutoff created comparable vaccinated and unvaccinated groups—provided strong evidence that preventing herpes zoster inflammation protects brain health.
• Dr. Dawn concludes with Huntington's disease breakthrough: microRNA therapy delivered by virus directly into the brain slowed disease progression by 75% over three years. The microRNA binds to Huntington protein mRNA, preventing ribosome translation and toxic protein production. Some patients returned to work; others expected to need wheelchairs are still walking.