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相关概念视频

CRISPR01:59

CRISPR

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Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
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The basic reaction of homologous recombination (HR) involves two chromatids that contain DNA sequences sharing a significant stretch of identity. One of these sequences uses a strand from another as a template to synthesize DNA in an enzyme-catalyzed reaction. The final product is a novel amalgamation of the two substrates. To ensure an accurate recombination of sequences, HR is restricted to the S and G2 phases of the cell cycle. At these stages, the DNA has been replicated already and the...
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Bacteria and archaea are susceptible to viral infections just like eukaryotes; therefore, they have developed a unique adaptive immune system to protect themselves. Clustered regularly interspaced short palindromic repeats and CRISPR-associated proteins (CRISPR-Cas) are present in more than 45% of known bacteria and 90% of known archaea.
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Gene Therapy00:59

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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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CRISPR/Cas9-mediated Targeted Integration In Vivo Using a Homology-mediated End Joining-based Strategy
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基于CRISPR的疗法用于遗传性血管.

Danny M Cohn1, Padmalal Gurugama1, Markus Magerl1

  • 1From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.).

The New England journal of medicine
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PubMed
概括

一次剂量的NTLA-2002基因疗法显著减少了遗传性血管发作. 这种针对KLKB1的基于CRISPR的治疗方法显示出对终身控制胀发作的前景.

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科学领域:

  • 遗传学 遗传学 是一个
  • 药理学 药理学是指药理学的学科.
  • 免疫学 免疫学 免疫学

背景情况:

  • 遗传性血管 (HAE) 是一种罕见的遗传性疾病,导致严重的,不可预测的发作.
  • NTLA-2002是一种基于CRISPR的新型基因编辑疗法,旨在向KLKB1基因.
  • 单一的NTLA-2002管理旨在为HAE管理提供终身解决方案.

研究的目的:

  • 评估NTLA-2002在患有遗传性血管的成年人中的疗效和安全性.
  • 评估单剂量NTLA-2002对每月发作率和血卡利克林水平的影响.
  • 作为一个次要目标,探索患者报告的结果.

主要方法:

  • 一个2期,随机的,安慰剂对照试验,涉及HAE的成年人.
  • 参与者接受了NTLA-2002 (25毫克或50毫克) 或安慰剂的单剂量,比例为2:2:1.
  • 主要终点是从第1周到第16周的每月攻击率;安全性和kallikrein水平是次要终点.

主要成果:

  • 与安慰剂相比,NTLA-2002显著降低了75-77%的平均每月攻击率.
  • 观察到血卡利克林蛋白水平大幅降低 (25毫克-55%,50毫克-86%).
  • 在接受NTLA-2002的患者中,40% (25 mg) 和73% (50 mg) 在研究期间没有发作.

结论:

  • 在HAE患者中,单剂量NTLA-2002有效降低了血管发作和血卡利克林水平.
  • 基因编辑疗法显示出强大而持续的疗效.
  • 结果支持在HAE治疗中对NTLA-2002进行3期试验的进一步调查.