A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna

Global gene delivery to the CNS has therapeutic importance for the treatment of neurological disorders that affect the entire CNS. Due to direct contact with the CNS, cerebrospinal fluid (CSF) is an attractive route for CNS gene delivery. A safe and effective route to achieve global gene distributio...

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Veröffentlicht in:Mol Ther
Hauptverfasser: Taghian, Toloo, Marosfoi, Miklos G., Puri, Ajit S., Cataltepe, Oguz.I., King, Robert M., Diffie, Elise B., Maguire, Anne S., Martin, Douglas R., Fernau, Deborah, Batista, Ana Rita, Kuchel, Tim, Christou, Chris, Perumal, Raj, Chandra, Sundeep, Gamlin, Paul D., Bertrand, Stephanie G., Flotte, Terence R., McKenna-Yasek, Diane, Tai, Phillip W.L., Aronin, Neil, Gounis, Matthew J., Sena-Esteves, Miguel, Gray-Edwards, Heather L.
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society of Gene & Cell Therapy 2020
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Original Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7002897/
https://ncbi.nlm.nih.gov/pubmed/31813800
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2019.11.012
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