タグ:遺伝子治療

[出典] "Engineering domain-inlaid SaCas9 adenine base editors with reduced RNA off-targets and increased on-target DNA editing" Nguyen Tran, MT, Mohd Khalid MKN, Wang Q. et al. Nat Commun. 2020-09-25. [crisp_bio注] 文中の [#n] は、原論文のreferencesの番 ... もっと読む

[出典] "The sustained expression of Cas9 targeting toxic RNAs reverses disease phenotypes in mouse models of myotonic dystrophy type 1" Batra R [..] Yeo GW. Nat Biomed Eng. 2020-09-14. https://doi.org/10.1038/s41551-020-00607-7 筋強直性ジストロフィー1型 ... もっと読む

[出典] "Gene Therapy for glaucoma by ciliary body aquaporin 1 disruption using CRISPR-Cas9" Wu J [..] Chu CJ. Molecular Therapy. 2020-01-10. ; ブログ "Could It Be A One-Stop Solution For The Most Common Cause Of Irreversible Blindness? " Smith V. BSGCT bl ... もっと読む

[出典] REVIEW "Targeting Alternative Splicing as a Potential Therapy for Episodic Ataxia Type 2" Jaudon F [..] Cingolani LA. Biomedicines. 2020-09-05.  反復発作性運動失調症 2 型 (episodic ataxia type 2; EA2)の原因は、P/Q型電位依存性カルシウムチャネルの ... もっと読む

[出典] "Optimization of S. aureus dCas9 and CRISPRi elements for a single adeno-associated virus that targets an endogenous gene" Backstrom JR [..] Rex TS. Molecular Therapy Methods & Clinical Development. 2020-09-06.  CRISPRiシステムは遺伝子治療剤とし ... もっと読む

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