タグ:遺伝子治療

[出典] "Regulation of Cas9 by viral proteins Tat and Rev for HIV-1 inactivation" Vergara-Mendoza M [..] Viveros-Rogel M. Antiviral Res 2020-06-21.  抗レトロウイルス併用療法 (combined antiretroviral therapy: cART)によってHIV-1の複製を制御し、疾患の進行を ... もっと読む

[出典] "Systemic nanoparticle delivery of CRISPR-Cas9 ribonucleoproteins for effective tissue specific genome editing" Wei T, Cheng Q, Min YL, Olson EN, Siegwart DJ. Nat Commun 2020-06-26.  Cas9/sgRNAをRNPの形式でデリバリーすることで、オフターゲット編集 ... もっと読む

[出典] "Efficient gene editing of human long-term hematopoietic stem cells validated by clonal tracking" Ferrari S, Jacob A [..] Genovese  P, Naldini  L. Nat Biotechnol 2020-06-29. 患者由来の造血幹細胞前駆細胞 (HSPC)にex vivoで遺伝子編集を加えた上で、 ... もっと読む

[出典] NEWS "In its first tough test, CRISPR base editing slashes cholesterol levels in monkeys" Begley S STAT 2020-06-27; ICCSR 2020講演者・Verve Therapeutics共同設立者Kiran Musunuruのツイート[文末に引用] ICCRS (国際幹細胞学会) 2020 Virtual Meetingの"C ... もっと読む

[出典] "Comparative targeting analysis of KLF1, BCL11A, and HBG1/2 in CD34+ HSPCs by CRISPR/Cas9 for the induction of fetal hemoglobin" Lamsfus-Calle A, Daniel-Moreno A [..] Mezger M. Sci Rep 2020-06-23.  βグロビン異常症は、βグロビン遺伝子 (HBB)の変異 ... もっと読む

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