[出典] "Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair" Sweeney CL, Pavel-Dinu M [..] Porteus MH, Malech HL, de Ravin SS. Gene Ther. 2021-02-24. https://doi.o ...
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タグ：遺伝子治療
[レビュー] CRISPR-Cas9遺伝子編集による先天性心疾患変異修復の試み
[出典] REVIEW "Application of CRISPR-Cas9 gene editing for congenital heart disease" Seok H, Deng R, Cowan DB, Wang D-Z. Clin Exp Pediatr. 2021-03-02. https://doi.org/10.3345/cep.2020.02096 9種類の先天性心疾患の責任遺伝子の表 (Table 1)と、病因変異修復の ...
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マイクロニードル経皮パッチを介したCas9遺伝子編集とステロドの共送達による炎症性皮膚障害療法
[出典] "Microneedle-assisted genome editing: A transdermal strategy of targeting NLRP3 by CRISPR-Cas9 for synergistic therapy of inflammatory skin disorders" Wan T, Pan Q, Ping Y (浙江大学). Sci Adv. 2021-03-10. https://doi.org/10.1126/sciadv.abe2888 著 ...
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CRISPR/Cas9によるアレル特異的KOによる網膜色素変性症 (RP)療法
[出典] "Allele-Specific Knockout by CRISPR/Cas to Treat Autosomal Dominant Retinitis Pigmentosa Caused by the G56R Mutation in NR2E3" Diakatou M [..] Kalatzis V. Int J Mol Sci 2021-03-05. https://doi.org/10.3390/ijms22052607 University of Montpellierの研 ...
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ヒト造血幹細胞におけるαグロビン遺伝子座のCRISPR/Cas9編集によるβサラセミアの修復
2021-03-01 06:34 初稿; 19:56 投稿タイトル修正 ヒト造血肝細胞 →ヒト造血幹細胞---------------------------------------------------------------------------------------------------------------[出典] "Correction of β-thalassemia by CRISPR/Cas9 editing of the ...
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