本期节目,我们请到了来自美国 Life Edits 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