医学遗传前沿

EP32 CRISPR定向进化与遗传病的基因治疗@杨添

本期节目,我们请到了来自美国 Life Edit Theraputics Inc.的杨添博士,一起聊一聊在美国 Biotech 的职业发展,CRISPR 基因编辑技术,定向进化技术, 以及遗传病的基因治疗~

主要内容

02:19 在学术界和工业界中如何选择

04:28 美国 Biotech 的生态与职业发展

11:16 CRISPR 技术的背景介绍

14:03 CRISPR 技术的迭代

18:13 Base editing 的定向进化

25:35 遗传病的基因治疗案例

关键词与相关链接

CRISPR背景介绍 :https://www.youtube.com/watch?v=7ESZTE6rjLI

Classcal CRISPR 与迭代新技术

Classical CRISPR (CRISPR 1.0): Double-strand break

  • https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-announces-strategic-transition-vivo-gene-editing
  • https://www.nejm.org/doi/full/10.1056/NEJMoa2309915
  • https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease
  • https://www.biospace.com/business/crisprs-casgevy-on-the-rise-with-more-gene-therapy-proof-of-concept-to-come-in-2025
  • https://www.nejm.org/doi/full/10.1056/NEJMoa2309149

Base Editing (CRISPR 2.0):https://www.youtube.com/watch?v=B1Bv-UmxugM

Prime Editing (CRISPR 3.0):https://www.youtube.com/watch?v=huDgW0wCi9c

Large Insertion, RNA Editing (CRISPR 4.0):

  • Tome:https://www.fiercebiotech.com/biotech/young-gene-editing-biotech-explores-strategic-options-investors-pullback
  • Tune:https://tunetx.com/tune-therapeutics-presents-first-data-supporting-tune-401-a-first-in-class-epigenetic-silencer-for-hepatitis-b/  

遗传病的Base editing治疗案例:

https://www.nejm.org/doi/full/10.1056/NEJMoa2504747

10.1038/s41588-025-02172-8

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