IIIB 型粘多糖病: AAV 疗法的现状回顾和探索
CJ Rouse, VN Jensen, CD Heldermon - 中国神经再生研究(英文版), 2024 - sjzsyj.com.cn
… of MPS IIIB, current and potential treatment options, the … -associated virus (AAV) gene therapy
approaches, and how novel … were used: MPS IIIA, MPS IIIB, MPS IIIC, MPS IIID, and MPS. …
approaches, and how novel … were used: MPS IIIA, MPS IIIB, MPS IIIC, MPS IIID, and MPS. …
粘多糖疾病的先天免疫
O Mandolfo, BW Bigger - 中国神经再生研究(英文版), 2023 - sjzsyj.com.cn
… medical care for severe mucopolysaccharidosis (MPS) type IH … : In all, we propose that an
iPSC-based gene therapy … late diagnosis or for which no treatment is currently available. Firstly…
iPSC-based gene therapy … late diagnosis or for which no treatment is currently available. Firstly…
利用基因修饰间充质干细胞提高β 氨基己糖苷酶A 的活性
AA Shaimardanova, DS Chulpanova… - 中国神经再生研究 …, 2024 - sjzsyj.com.cn
… -mediated gene therapy is considered a promising treatment … transplantation or umbilical
cord blood cell transplantation (… gene mRNA, respectively, per 1 µg of total RNA (Figure 3A). …
cord blood cell transplantation (… gene mRNA, respectively, per 1 µg of total RNA (Figure 3A). …
GM2 神经节苷脂病的治疗: 静脉内给予HexA 过表达的hUCBMC 的可行性
AA Shaimardanova, DS Chulpanova… - 中国神经再生研究 …, 2022 - sjzsyj.com.cn
… safety of cell-mediated gene therapy for Sandhoff disease and … shown that even with
intracerebral administration of AAV9, … in mucopolysaccharidosis IIIA. EMBO Mol Med 12:e11185. …
intracerebral administration of AAV9, … in mucopolysaccharidosis IIIA. EMBO Mol Med 12:e11185. …