基本信息
浏览量:57
职业迁徙
个人简介
New techniques for replacing, silencing or editing the underlying genetic defects provide new hope. One striking example is gene therapy for spinal muscular atrophy, in which infants who would never have been able to sit up now have the potential to lead relatively normal lives. But the number of successes is small. The challenge is delivering the gene replacement, gene silencing, or gene editing to the right cells in the human body.
Kunwoo and colleagues developed a novel delivery technology. They have published multiple high-profile papers, including one written in collaboration with Nobel laureate Jennifer Doudna, and demonstrated that gene editing can show therapeutic effects in mice with Duchenne muscular dystrophy and autism. NIH Director, Francis Collins, wrote in an article that “this approach is especially exciting because it also holds potential for treating or even curing many other genetic diseases.”
GenEdit has continued to improve the delivery technology. The innovation comes from the NanoGalaxy™ platform, which deploys machine learning to evaluate the results from systematic screening of thousands of unique polymer nanoparticles and create optimized tissue-specific delivery systems. These unique polymer nanoparticles can then be used to deliver the components required for gene replacement, gene silencing, or gene editing to the right tissue, maximizing therapeutic benefits. Kunwoo envisions a world where we can edit out disease and rewrite patient health and to make these new genetic medicines available to patients throughout the world.
Kunwoo and colleagues developed a novel delivery technology. They have published multiple high-profile papers, including one written in collaboration with Nobel laureate Jennifer Doudna, and demonstrated that gene editing can show therapeutic effects in mice with Duchenne muscular dystrophy and autism. NIH Director, Francis Collins, wrote in an article that “this approach is especially exciting because it also holds potential for treating or even curing many other genetic diseases.”
GenEdit has continued to improve the delivery technology. The innovation comes from the NanoGalaxy™ platform, which deploys machine learning to evaluate the results from systematic screening of thousands of unique polymer nanoparticles and create optimized tissue-specific delivery systems. These unique polymer nanoparticles can then be used to deliver the components required for gene replacement, gene silencing, or gene editing to the right tissue, maximizing therapeutic benefits. Kunwoo envisions a world where we can edit out disease and rewrite patient health and to make these new genetic medicines available to patients throughout the world.
研究兴趣
论文共 246 篇作者统计合作学者相似作者
按年份排序按引用量排序主题筛选期刊级别筛选合作者筛选合作机构筛选
时间
引用量
主题
期刊级别
合作者
合作机构
Advanced drug delivery reviews (2023): 115026-115026
J. Comput. Des. Eng.no. 1 (2023): 221-232
SSRN Electronic Journal (2022)
加载更多
作者统计
合作学者
合作机构
D-Core
- 合作者
- 学生
- 导师
数据免责声明
页面数据均来自互联网公开来源、合作出版商和通过AI技术自动分析结果,我们不对页面数据的有效性、准确性、正确性、可靠性、完整性和及时性做出任何承诺和保证。若有疑问,可以通过电子邮件方式联系我们:report@aminer.cn