Expanding RNAi therapeutics to extrahepatic tissues with lipophilic ...
2022年6月2日 · Here we show that conjugation of 2′-O-hexadecyl (C16) to siRNAs enables safe, potent and durable silencing in the central nervous system (CNS), eye and lung in rodents and non-human primates ...
「Alnylam」发刊 C16-siRNAs缀合疗法效果显著丨C16单体速览
小核酸巨头之一的 Alnylam 公司在《Nature Biotechnology》 期刊发表文章： Expanding RNAi therapeutics to extrahepatic tissues with lipophilic conjugates，研究显示 2'-O-十六烷基 (C16)与siRNA的缀合能够在啮齿类动物和 非人灵长类 动物的中枢神经系统 (CNS)、眼和肺中实现安全、有效和持久的沉默，具有广泛的细胞类型特异性 [1]。 目录. 关于2'-O-C16单体. C16-siRNAs的亲脂性. C16-siRNAs的生物活性. 申基C16单体产品. 2'-O-十六烷基 (C16)单体是指在核糖主链的2'位引 …
Alnylam C16平台CNS递送首获临床验证！siRNA治疗AD实现 …
2023年4月27日 · Alnylam已建立肝外递送配体发现平台，布局小分子/脂类（C16进行CNS递送）、多肽（收购PeptiDream，针对特定受体筛选/优化/合成候选多肽序列）、抗体 ...
Palmityl C16 Oligo (NHS) LMO - Gene Link
It is a C16 long chain saturated fatty acid. Palmitic acid is found naturally in palm oil and palm kernel oil, as well as in butter, cheese, milk and meat. Oligonucleotides are predominantly hydrophilic species and require help in permeating cell membranes.
Antisense oligonucleotides (ASOs) and C16-conjugated small interfering RNAs (siRNAs) are two distinct oligonucleotide-based approaches to huntingtin (HTT) lowering in development for Huntington’s disease. While ASOs are single-stranded and utilize RNAseH1 to cleave target RNAs, siRNAs are double-stranded and
Lipophilic siRNA conjugates yield durable silencing in ... - Nature
2022年6月8日 · Conjugating short interfering RNAs (siRNAs) to a lipophilic alkyl chain enabled safe delivery and long-term mRNA silencing in the brain, eye and lung in animal models, thereby opening new...
Palmityl C16 (5') LMO Oligo Modifications from Gene Link
It is a C16 long chain saturated fatty acid. Palmitic acid is found naturally in palm oil and palm kernel oil, as well as in butter, cheese, milk and meat. Oligonucleotides are predominantly hydrophilic species and require help in permeating cell membranes.
It is a C16 long chain saturated fatty acid. Palmitic acid is found naturally in palm oil and palm kernel oil, as well as in butter, cheese, milk and meat. Oligonucleotides are predominantly hydrophilic species and require help in permeating cell membranes.
使用亲脂性缀合物将 RNAi 疗法扩展到肝外组织,Nature …
在这里，我们展示了 2'- O的缀合 siRNA 的十六烷基 (C16) 可在具有广泛细胞类型特异性的啮齿动物和非人类灵长类动物的中枢神经系统 (CNS)、眼睛和肺中实现安全、有效和持久的沉默。 我们表明，鞘内或脑室内递送的 C16-siRNA 在 CNS 区域和细胞类型中都很活跃，具有持续的 RNA 干扰 (RNAi) 活性至少 3 个月。 类似地，对眼睛进行玻璃体内给药或对肺进行鼻内给药会导致有效且持久的抑制作用。 靶向淀粉样蛋白前体蛋白的 siRNA 的临床前疗效通过在阿尔茨海默病小鼠模 …
利用亲脂性结合物可将RNAi治疗剂扩展到肝外组织—小柯机器人— …
Here we show that conjugation of 2′-O-hexadecyl (C16) to siRNAs enables safe, potent and durable silencing in the central nervous system (CNS), eye and lung in rodents and non-human primates ...