gene therapy
細胞特異的アデノ随伴ウィルスによる遺伝子治療 【紹介文献】 "Cell-Selective Adeno-Associated Virus-Mediated SCN1A Gene Regulation Therapy Rescues Mortality and Seizure Phenotypes in a Dravet Syndrome Mouse Model and Is Well Tolerated in Nonh…
【紹介文献】 CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis Gillmore et al., N Engl J Med, 2021, 385(6):493-502 https://www.nejm.org/doi/full/10.1056/NEJMoa2107454 【発表概要】 CRISPR-Cas9を用いたゲノム編集は遺伝子治療への…
A part of direct conversion technology of non-neuronal cells into neurons in vivo 【紹介文献】 "Therapeutically viable generation of neurons with antisense oligonucleotide suppression of PTB" Maimon R et al., Nat Neurosci. 2021 24. 1089-10…
【紹介文献】 Reprogramming to recover youthful epigenetic information and restore vision Lu et al., 2020, Nature, 588, 124-129 www.nature.com 【発表概要】 老化は退行性変化であり，組織の機能不全や最終的には死につながる現象です．その要因とし…
【紹介文献】 "Gene therapy conversion of striatal astrocytes into GABAergic neurons in mouse models of Huntington’s disease" Wu et al., Nature Communications, 11, 1105, 2020 www.nature.com 【発表概要】 ハンチントン病（ＨＤ）の原因は、脳の…
