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InsightsܲrAAVSf9ϸϵͳ293ϸϵͳĸ

ʱ䣺2022-03-25 ȶȣ
 
ز(Recombinant Adeno-associated Virus, rAAV)һֶ²ԵĵDNAԭԵͣתЧʸߣҲҸƵص㣬ʵںЧڣrAAVĻƲƷѾУȡЧrAAVҲΪҵΪֿȵDzƷ
 
 
 

ôܹЧ徲ͻrAAVϵͳ֣һʹþװϵͳ˲ʱתȾ293ϸһּʹЯrAAV顢RepѰ׺CapѰ׵ĸ״ѬȾSpodoptera frugiperdaSf9ϸӶrAAVϵͳкβʵٴӦʱЧ

 

293ϸrAAVװϵͳ

תȾ293ϸʵҼҩҵõrAAVҪ֮һϵͳҪͬʱʹôĿĻ򣩡װAAVƹЧRepѰ¿ѰCap븨ٲҪԪ벡ֳͬʱתȾ293ϸӶrAAVFDA׼ZolgensmaLuxturnarAAVƲƷʹתȾ293ϸϵͳ
 
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ͼ1 תȾ293ϸrAAV

ͼƬȪԴMueller C et al., Curr Protoc Microbiol. 2012

 

Sf9װϵͳ

Baculovirus/Sf9һ滻鶯ϸϵrAAVƻϵͳʹø״壨Baculovirus expression vectors, BEVsѬȾSf9ϸrAAVSf9ϸԴڱѰ׵ص㣬ϵͳʹúrAAVԼAAV-RepAAV-Cap״תSf9ϸʹrAAVSf9ϸװյִģrAAVĿ

 

Sf9ϸڴģӦиܶ˸ϵͳ׷Ŵģͬʱ״ڲ鶯ֲǷ²ԵģҽѪϵͳתȾ293ϸϵͳΪ徲2012꣬ŷҩƷEMA׼׸rAAVҩGlyberaʹSf9ϸϵͳrAAV

 

293ϸװϵͳSf9װϵͳIJ

ֻ10ϵ½׼ɻAAVĻƲƷٶսrAAVIJԭԵ20209£Neil G. RumachikMol Ther Methods Clin Dev.Ͷ293ϸSf9ϸrAAVϵͳ鷢Ҫбʡ¿Ѱ׷(post-translational modifications, PTMs)/ѬȾЧʣͼ2

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ͼ2 Sf9ϸװϵͳ293ϸװϵͳrAAV

ͼƬȪԴNeil G. Rumachik et al., Mol Ther Methods Clin Dev. 2020

 

ȷָ

1İװϵͳrAAV¿ǾвPTMsǻữͼ׻ͼ3

2װϵͳװУrAAV׻ϵͳƺԻ鱬෴ãSf9ϸϵͳrAAVƻ293ϸrAAV򼤻

 

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ͼ3 293ϸsf9ϸƽ̨rAAV¿β

ͼƬȪԴNeil G. Rumachik et al., Mol Ther Methods Clin Dev. 2020

 

3ϵͳrAAVĿտʼҲ𣬣293ϸrAAVտͣͼ3װ̱ĺѰأSf9ϸϵͳտԽϸߣøԺѰˮйأģ״ϵͳҪôǽṹԵģҪô벡й

4⣬293ϸrAAVչֳǿڼѬȾоվʵ飬ͨעվͨβϵͳҩע䣬293ϸrAAVиõѬȾͼ4

 

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ͼ4 ϵͳrAAV⼰еѬȾ

ͼƬȪԴNeil G. Rumachik et al., Mol Ther Methods Clin Dev. 2020

 

ˣЩչʾԿ293ϸrAAVܱSf9иõѬȾЧʡߵҲٴӦõDZȻɷ񶨵ǣSf9ϵͳrAAVװϵͳиߵrAAVװʣʱգSf9װϵͳDZΪҪʵ

 

UGӵȨHEK293ϸϵɶԻڸϸrAAVϵͳչŻˢ£õrAAVϵͳͬʱUGʹеGMPװоrAAVƱǮȫ̭μʼȾûʹֿɼ

 

 

 

 
 
 
 

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UGиrAAVװҪȡrAAVƷôɨ·άϵUGɣ

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[1] Rumachik NG, Malaker SA, Poweleit N, Maynard LH, Adams CM, Leib RD, Cirolia G, Thomas D, Stamnes S, Holt K, Sinn P, May AP, Paulk NK. Methods Matter: Standard Production Platforms for Recombinant AAV Produce Chemically and Functionally Distinct Vectors. Mol Ther Methods Clin Dev. 2020 May 22;18:98-118. doi: 10.1016/j.omtm.2020.05.018. PMID: 32995354; PMCID: PMC7488757.
[2] Wang D, Tai PWL, Gao G. Adeno-associated virus vector as a platform for gene therapy delivery. Nat Rev Drug Discov. 2019 May;18(5):358-378. doi: 10.1038/s41573-019-0012-9. PMID: 30710128; PMCID: PMC6927556.
[3] Bulcha JT, Wang Y, Ma H, Tai PWL, Gao G. Viral vector platforms within the gene therapy landscape. Signal Transduct Target Ther. 2021 Feb 8;6(1):53. doi: 10.1038/s41392-021-00487-6. PMID: 33558455; PMCID: PMC7868676.
[4] Mueller C, Ratner D, Zhong L, Esteves-Sena M, Gao G. Production and discovery of novel recombinant adeno-associated viral vectors. Curr Protoc Microbiol. 2012 Aug;Chapter 14:Unit14D.1. doi: 10.1002/9780471729259.mc14d01s26. PMID: 22875565; PMCID: PMC4358842.
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