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https://www.researchgate.net/publication/51633210_Adeno-Associated_Viral_Gene_Delivery_in_Neurodegenerative_Disease
Adeno-associated viral vectors (AAV) have taken center stage for gene delivery to the central nervous system, given their ability to express genes in post mitotic cells for long periods with ...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6404499/
Oct 15, 2018 · Gene therapy via gene replacement or gene correction provides the potential for transformative therapies to delay or possibly stop further progression of the neurodegenerative disease in affected patients. Adeno-associated virus has been the vector of choice in recent clinical trials of therapies for neurodegenerative diseases due to its safety ...Author: Yuan Qu, Yi Liu, Ahmed Fayyaz Noor, Johnathan Tran, Rui Li
https://link.springer.com/protocol/10.1007/978-1-61779-328-8_29
Aug 14, 2011 · Adeno-associated viral (AAV) vectors provide one of the more promising approaches to gene delivery, and have been used extensively over the last 20 years. Derived from nonpathogenic parvoviruses, these vectors allow for stable and robust expression …Author: Peter F. Morgenstern, Roberta Marongiu, Sergei A. Musatov, Michael G. Kaplitt
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2880921/
Adeno-associated virus was first used as a viral vector for CNS gene delivery by replacing the rep and cap genes with E. coli LacZ gene and using adenovirus as a helper virus . The first generation of AAV vectors was helper-virus-based, but several groups developed a “helper-free” system where the helper genes could be provided by ...Author: Seung T. Lim, Mikko Airavaara, Brandon K. Harvey
https://www.liebertpub.com/doi/full/10.1089/hum.2016.087
Jun 07, 2016 · 185 Fu H, DiRosario J, Kang L, et al. Restoration of central nervous system α-N-acetylglucosaminidase activity and therapeutic benefits in mucopolysaccharidosis IIIB mice by a single intracisternal recombinant adeno-associated viral type 2 vector delivery. J Gene Med 2010;12:624–633. Crossref, Medline, Google ScholarAuthor: Michaël Hocquemiller, Laura Giersch, Mickael Audrain, Samantha Parker, Nathalie Cartier
https://www.sciencedirect.com/topics/neuroscience/adeno-associated-virus
Adeno-Associated Viral Vectors. Adeno-associated virus (AAV), one of the most attractive gene therapy vectors, usually interacts with heparan sulfate proteoglycans on cell membrane as the first step for cellular internalization. Like an adenovirus, it also induces strong immune responses, infects both dividing and nondividing cells, and remains ...
https://www.liebertpub.com/doi/full/10.1089/hum.2017.160
Gene therapy has also addressed complex neurodegenerative diseases, particularly Parkinson's disease, with encouraging results in human patients, demonstrating that specific targeting of central nervous system (CNS) cells is a relevant strategy for severe pathologies and that efficient access to the CNS with viral vectors is an achievable goal.Author: PiguetFrançoise, AlvesSandro, CartierNathalie
https://www.researchgate.net/publication/305783956_Adeno_Associated_Viral_Vector_Delivered_RNAi_for_Gene_Therapy_of_SOD1_Amyotrophic_Lateral_Sclerosis
Adeno Associated Viral Vector Delivered RNAi for Gene Therapy of SOD1 Amyotrophic Lateral Sclerosis ... is a fatal neurodegenerative disease caused by progressive loss of upper and lower motor ...
https://link.springer.com/protocol/10.1007/978-1-61779-328-8_34
Aug 14, 2011 · Cite this protocol as: Morgenstern P.F., Marongiu R., Musatov S.A., Kaplitt M.G. (2011) Erratum to: Adeno-Associated Viral Gene Delivery in Neurodegenerative Disease.Author: Peter F. Morgenstern, Roberta Marongiu, Sergei A. Musatov, Michael G. Kaplitt
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