[注] LNP (脂質ナノ粒子)；HSPC (ヒト造血幹・前駆細胞)[出典] "Lipid nanoparticles allow efficient and harmless ex vivo gene editing of human hematopoietic cells" Vavassori V, Ferrari S [..] Naldini L. Blood 2023-08-31. https://doi.org/10.1182/blood.20220193 …
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2023年09月
嚢胞性線維症 (CF) の責任変異W1282Xを、塩基エディターABEとHITIで修復
[出典] "Use of adenine base editing and homology-independent targeted integration strategies to correct the cystic fibrosis causing variant, W1282X" Mention K [..] Harrison PT. Hum Mol Genet. 2023-08-31. https://doi.org/10.1093/hmg/ddad143 [著者所属] U Co …
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![[論説] 心臓を癒す：循環器疾患の治療を革新しつつある遺伝子治療の潜在的可能性を解き放つ](https://livedoor.blogimg.jp/crisp_bio/imgs/4/e/4e52454f-s.png)
[論説] 心臓を癒す：循環器疾患の治療を革新しつつある遺伝子治療の潜在的可能性を解き放つ
[出典] "Healing hearts: unlocking the potential of gene therapy in revolutionizing cardiovascular disease treatment" Beqqali A, Baker A. Biochem (London) 2023-08-23. https://doi.org/10.1042/bio_2023_129 [著者所属] U Edinburgh；Fig. 1 (循環器疾患に対する遺 …
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ゲノム・ターゲティングが新生児I型シトルリン血症マウスモデルを救う
[注] シトルリン血症 (Citrullinemia type I; アルギノコハク酸シンテターゼ*欠損症)；[*] argininosuccinate synthetase (ASS1)[出典] "Lethality rescue and long-term amelioration of a citrullinemia type I mouse model by neonatal promoterless gene-targeting comb …
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CRISPR非依存で、核、ミトコンドリア、および葉緑体ゲノムDNAの鎖特異的塩基編集を実現するCyDENT
[注] CyDENT: cytidine deaminase–exonuclease–nickase–TALE[出典] "Strand-preferred base editing of organellar and nuclear genomes using CyDENT" Hu J [..] Zhao KT, Gao C. Nat Biotechnol. 2023-08-28. https://doi.org/10.1038/s41587-023-01910-9 [著者所属] …
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