OTOF基因的致病性突变导致了OTOF蛋白的缺乏或功能失调,从而引发常染色体隐性耳聋9型(DFNB9)。DFNB9的特征是先天性或语言前期的严重到完全的双侧听力损失,占遗传性耳聋的2-8%。 通过双AAV载体携带人类OTOF基因的AAV1-hOTOF在Otof−...
再生医学网获悉,近日,复旦大学附属眼耳鼻喉科医院舒易来教授、李华伟教授、王武庆教授,联合哈佛大学医学院陈正一教授在Nature Medicine上报告了双耳AAV1-hOTOF基因治疗DFNB9患儿的安全性和有效性。 听觉与视觉是人类探索世界的主要途径,通过聆听万籁之声,能够帮助我们更加清晰与全面地认知世界。不过,对于先天性耳聋患者来...
OTOF基因的致病性突变导致了OTOF蛋白的缺乏或功能失调,从而引发常染色体隐性耳聋9型(DFNB9)。DFNB9的特征是先天性或语言前期的严重到完全的双侧听力损失,占遗传性耳聋的2-8%。 通过双AAV载体携带人类OTOF基因的AAV1-hOTOF在Otof−/−小鼠和非人灵长类动物中已被证明是有效且安全的。目前,试验已显示,基因治疗...
OTOF基因的致病性突变导致了OTOF蛋白的缺乏或功能失调,从而引发常染色体隐性耳聋9型(DFNB9)。DFNB9的特征是先天性或语言前期的严重到完全的双侧听力损失,占遗传性耳聋的2-8%。 通过双AAV载体携带人类OTOF基因的AAV1-hOTOF在Otof−/−小鼠和非人灵长类动物中已被证明是有效且安全的。目前,试验已显示,基因治疗...
Methods:This single-arm, single-centre trial enrolled children (aged 1-18 years) with severe-to-complete hearing loss and confirmed mutations in both alleles of OTOF, and without bilateral cochlear implants. A single injection of AAV1-hOTOF was administered into the cochlea through the round ...
·0 kHz. In the participant who received the 9 × 1011vg dose, the average ABR threshold was improved from greater than 95 dB at baseline to 68 dB at 4 weeks, 53 dB at 13 weeks, and 45 dB at 26 weeks. In those who received 1·5 × 1012AAV1-hOTOF, the average ABR...
Methods This single-arm, single-centre trial enrolled children (aged 1–18 years) with severe-to-complete hearing loss and confirmed mutations in both alleles of OTOF , and without bilateral cochlear implants . A single injection of AAV1-hOTOF was administered into the cochlea through the ...
Here, we designed an OTOF gene therapy agent by dual-adeno-associated virus 1 (AAV1) carrying human OTOF coding sequences with the expression driven by the hair cell–specific promoter Myo15, AAV1-hOTOF. To develop a clinical application of AAV1-hOTOF gene therapy, we evaluated its ...
③The Lancet:AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial基于腺相关病毒(AAV)的AAV1-hOTOF用于常染色体隐性遗传性耳聋9型(DFNB9)的一项单臂试验DOI: 10.1016/S0140-6736(23)02874-X④JAMA:Protein Score May Reclassify Death Risk in Patients With Heart Failure蛋白质...
Awesome porn videos from various niches will not leave you unsatisfied. Busty MILFs, slutty amateurs, hardcore anal, sweet lesbians, outdoor sex and many other exciting XXX clips.