crisp_bio

blogging about crisp bio-S&T, especially CRISPR.

タグ:遺伝子治療

[出典] "Precise correction of Duchenne muscular dystrophy exon deletion mutations by base and prime editing" F. Chemello, A. C. Chai, H. Li, [..] Olson. Sci Adv. 2021-04-30. https://doi.org/10.1126/sciadv.abg4910 University of Texas Southwestern Medical ... もっと読む

[出典] "Targeting the Apoa1 locus for liver-directed gene therapy" De Giorgi M [..] Lagor WR. Mol Ther-Meth Clin D. 2021-04-24 [Journal Pre-proof]. https://doi.org/10.1016/j.omtm.2021.04.011; Graphical Abstract https://ars.els-cdn.com/content/image/1-s2.0 ... もっと読む

[出典] "Optimizing Integration and Expression of Transgenic Bruton’s Tyrosine Kinase for CRISPR-Cas9-Mediated Gene Editing of X-Linked Agammaglobulinemia" Gray DH [..] Kohn DB. CRISPR J. 2021-04-20. https://doi.org/10.1089/crispr.2020.0080 UCLAの研究グ ... もっと読む

[出典] "Universal Gene Correction Approaches for β-hemoglobinopathies Using CRISPR-Cas9 and Adeno-Associated Virus Serotype 6 Donor Templates" Lamsfus-Calle A, Daniel-Moreno A [..] Mezger M. CRISPR J. 2021-04-20. https://doi.org/10.1089/crispr.2020.0141 ... もっと読む

2021-04-27 欧州医薬品庁 (EMA), CTX001™ を輸血依存性βサラセミア治療薬 (Transfusion-Dependent Beta Thalassemia: TDT)としてPRIME (PRIority MEdicines)プログラムに指定[出典] "Vertex and CRISPR Therapeutics Announce Priority Medicines (PRIME) Designation Gra ... もっと読む

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