Delivery of therapeutic AAV9 vectors via cisterna magna to treat neurological disorders.
Delivery of therapeutic AAV9 vectors via cisterna magna to treat neurological disorders.
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通过小脑延髓池递送治疗性 AAV9 载体以治疗神经系统疾病。
DOI：
10.1016/j.molmed.2021.09.007
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发表时间：
2022
影响因子：
13.6
通讯作者：
Marchi PM
中科院分区：
文献类型：
--
作者：
Marchi PM
脑池内施用用于基因治疗的临床级腺相关病毒（AAV）载体。AAV制造业生产具有明确性质的高质量产品。对于小脑延髓池（ICM）内递送，将确定剂量的治疗性病毒注射到小脑和延髓背侧之间充满脑脊液（CSF）的蛛网膜下腔中。突出显示了解剖学递送位点及其各自的病毒生物分布概况沿着CNS。每种输送途径的利弊总结显示，ICM输送是其延伸中靶向CNS的最有前途的途径。然而，Ohno等人报告腰椎穿刺（LP）可获得相似的结果。
Intra-cisternal administration of clinical grade adeno-associated virus (AAV) vectors for gene therapy. The AAV manufacturing industry produces a high quality product of well-defined properties. For intra-cisterna magna (ICM) delivery, an established dose of therapeutic virus is injected in the cerebrospinal fluid (CSF)-filled subarachnoid space between the cerebellum and the dorsal side of the medulla oblongata.Overview of available routes for adeno-associated virus 9 (AAV9) delivery to the central nervous system (CNS). Anatomical delivery sites and their respective viral biodistribution profiles along the CNS are highlighted. A summary of pros and cons of each delivery route reveals ICM delivery as the most promising route to target the CNS in its extension. However, Ohno et al. reported that lumbar puncture (LP) can achieve similar results.
影响因子：
7.1
作者：
Hordeaux J;Dubreil L;Robveille C;Deniaud J;Pascal Q;Dequéant B;Pailloux J;Lagalice L;Ledevin M;Babarit C;Costiou P;Jamme F;Fusellier M;Mallem Y;Ciron C;Huchet C;Caillaud C;Colle MA
通讯作者：
Colle MA
DOI：
10.1016/j.omtm.2018.12.001
发表时间：
2019-06-14
影响因子：
4.7
作者：
Ohno, Kousaku;Samaranch, Lluis;Bankiewicz, Krystof S.
通讯作者：
Bankiewicz, Krystof S.
影响因子：
12.4
作者：
Taghian, Toloo;Marosfoi, Miklos G.;Gray-Edwards, Heather L.
通讯作者：
Gray-Edwards, Heather L.