CRISPR Targets Repeat Sequences in Huntington's

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Peter Attia's podcast with Feng Zhang on October 28, 2024

General HealthContent

Quotes From Source

Huntington's disease is caused by mutations in the Huntington gene...

Story of claim

CRISPR aims to treat Huntington's by editing expanded repeat sequences in the Huntington gene, potentially reducing harmful protein production.

  • Goal: To alleviate Huntington's disease symptoms by shortening harmful gene repeats.
  • Proof: Editing repeat sequences could reduce the deleterious effects of the Huntington gene mutation.
  • Nuances:
    • Precise editing needed due to involvement in coding regions.
    • Delivery to brain cells remains a challenge.
  • Impact on Life: Successful editing could delay onset or reduce severity of Huntington's symptoms, improving patient outcomes.

Investments

  • Price: High cost due to complexity of editing and delivery.
  • Time: Long research and development period; clinical trials needed.
  • Effort: Requires breakthroughs in delivery methods and precision editing.

Risks

Potential off-target effects or incomplete editing could worsen symptoms.

Alternatives

  • Gene silencing therapies targeting Huntington protein production.

Get Started 🚀

  • Explore CRISPR technologies focused on repeat sequence editing.
  • Assess current delivery methods to brain cells.
  • Partner with neurological research centers for collaborative studies.

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