The AAV9 gene therapy vector presented in this study is safe in mice and non-human primates and highly efficacious without causing overexpression toxicity, a major challenge for clinical translation of Rett syndrome gene therapy vectors to date. Our team designed a new truncated methyl-CpG-binding...
8. Sinnett SE, Hector RD, Gadalla KK, Heindel C, Chen D, Zaric V, et al. Improved MECP2 Gene Therapy Extends the Survival of MeCP2-Null Mice without Apparent Toxicity after Intracisternal Delivery. Mol Ther Methods Clin Dev 2017, 5: 106–115. ...
TherapiestargetedatMECP2针对MECP2基因的标靶治疗方法
wpre可以通过改善转录终止来减少病毒mrna的通读转录,这反过来又会增加病毒滴度和表达。(gene therapy volume 14,第1298-1304页(2007年))。 138、非病毒载体 139、在特定实施方式中,根据本发明使用的载体是非病毒载体。通常,非病毒载体可以是包括编码mecp2或其变体的核酸序列的质粒。
3.https://clinicaltrials.gov/ 4.https://rettsyndromenews.com/2021/06/11/alcyone-therapeutics-launches-focus-gene-therapy-rett-other-neurologic-ills/ FYI:总结生物医药更多知识点,敬请关注公众平台「佰傲谷BioValley」 部分图源网络,侵权联删
2024年12月12日,辉大基因/中国科学院上海药物所杨辉团队及临港实验室胥春龙在Nature Neuroscience发表题为An RNA editing strategy rescues gene duplication in a mouse model of MECP2 duplication syndrome and nonhuman primates的研究论文。该研究通过单次侧脑室注射方式,使用单个腺相关病毒 (AAV)载体递送辉大基因...
2024年12月12日,辉大基因/中国科学院上海药物所杨辉团队及临港实验室胥春龙在Nature Neuroscience发表题为An RNA editing strategy rescues gene duplication in a mouse model of MECP2 duplication syndrome and nonhuman primates的研究论...
In the present paper we review the neurobiology of MeCP2 and consider the various genetic (including gene therapy), pharmacological and environmental interventions that have been, and could be, developed to attempt phenotypic rescue in RTT. Such approaches are already providing valuable insights into...
(Gene Therapy Volume 14,第1298-1304页(2007年))。 非病毒载体 在特定实施方式中,根据本发明使用的载体是非病毒载体。通常,非病毒载体可以是包括编码MECP2或其变体的核酸序列的质粒。 包装的MECP2序列 MECP2基因序列也可以作为包装病毒载体的组成部分提供。一般而言,包装病毒载体包括包装在衣壳中的病毒载体。病毒...
50.gadalla,k.k.e.,vudhironarit,t.,hector,r.d.,sinnett,s.,bahey,n.g.,bailey,m.e.s.,gray,s.j.,cobb,s.r.(2017)developmentofanovelaavgenetherapycassettewithimprovedsafetyfeaturesandefficacyinamousemodelofrettsyndrome.molthermethodsclindev.5:180-190.doi:10.1016/j.omtm.2017.04.007. 51....