The FDA is set to decide Friday on a gene editing technique for patients with sickle cell disease. It would be the first use of CRISPR in the United States to treat a disease. CBS News' chief medical correspondent Dr. Jon LaPook has more. ...
随着生物技术的不断发展,我们期待能有更多创新疗法问世,让人类能够早日攻克癌症! 《自然》完整英文报道链接:http://www.nature.com/news/crispr-gene-editing-tested-in-a-person-for-the-first-time-1.20988 本文经微信公众号“药明康德”授权转载。 延伸阅读 ①中国开启全球首个CRISPR基因编辑人体试验,能治癌症么?
Rational engineering of minimally immunogenic nucleases for gene therapy CRISPR-mediated genome editing tools are entering the clinic, but concerns remain about the potential immunogenicity of these bacterial-derived proteins. Here, Raghavan et al. engineer two Cas effectors, Cas9 and Cas12, for reduc...
2. https://info.evaluate.com/rs/607-YGS-364/images/Gene%20Editing%20-%20Overhyped%20or%20Unstoppable%20Tide%20Whitepaper.pdf 3. https://investors.beamtx.com/news-releases/news-release-details/beam-therapeutics-reports-pipeline-and-business-updates-and 4. https://www.fda.gov/news-events/pr...
Some researchers hoping that gene-editing technology can conquer forms of Alzheimer’s caused by genetic mutations. By Tosin Thompson Twitter Facebook Email Last month saw the first-ever approval of a gene therapy that uses the CRISPR–Cas9 gene-editing tool, a treatment for the blood conditions...
[3] Frangoul et al., (2020). CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia. NEJM, DOI: 10.1056/NEJMoa2031054. 免责声明:药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的,文中观点不代表药明康德...
[3] Frangoul et al., (2020). CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia. NEJM, DOI: 10.1056/NEJMoa2031054. @药明康德 内容团队编辑 版权说明:本文来自药明康德内容团队,欢迎个人转发,谢绝媒体或机构未经授权以任何形式转载至其他平台。
[1] FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease. Retrieved Dec 8, 2023 from https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease [2] ...
[2] First sickle cell patient treated with CRISPR gene-editing still thriving. Retrieved March 9, 2023, from https://www.npr.org/sections/health-shots/2021/12/31/1067400512/first-sickle-cell-patient-treated-with-crispr-gene-editing-still-thriving ...
2021年6月26日,NEJM杂志发表了一篇题为:CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis 的论文,公布了诺奖得主Jennifer Doudna创立的Intellia Therapeutics和再生元合作开发治疗转甲状腺素蛋白淀粉样变性(ATTR)的CRISPR基因编辑疗法NTLA-2001I期临床结果(NCT04601051)。据悉,这也是首个公布的体内CRISPR基因...