Сζȣ⣺ζǷԽԽ
زrecombinant Adeno-associated Virus, rAAVڰ٣۾ࡢϵͳĶѾ֤ʵ徲ԺͺԡٴǰٴоУrAAV鿽ȷζȷȷͿظҩҪͬڿѧоУrAAVĻ鿽ȷζڻõġȹ̵ġɿġظʵŲɻȱárAAVĻ鿽ȷζΪζȣͨͨ¿ѰrAAVȷġ
㲻֪ĵζ
ζһƽζȺѬȾζȡ
ζָIJŨȡζͨŨġζȷ綨עѬȾζȣrAAVѬȾζȿƾ֤תеת䣬ҲͨҺĶı䣨Lock et al., 2010qPCRձӦrAAV嶨Ҫ֮һ
ѬȾζָתϸIJŨȡѬȾζͨͨ꾡תⶨоʾrAAVζѬȾζȵıԼΪ50:1Zeltner et al., 2010
PFUTUVGGCеζȣɵɵֲ
鲡ĵζֵλʽñPFU/mLTU/mLVG/mLGC/mLȣЩҪڲԼ׳ɵģʵӦЩζȵλ?
PFU/mLǿհγɵλΪPlaque Forming Unit per mLдٲϸѽIJĵζȱ궨
TU/mLǻԵζȵλΪTransduction Units per mLдҪָѬȾϸеIJĿĵζȲⶨ
ˣҪڿойעòͺ͵ζۺжϡ
DzrAAVļԽߣЧԽ
һЩоУrAAV¿ǻתƷ߷ӦѱȷΪ±̭ȫʧԵԭɡͼrAAVпܱanti-AAV壬͵ζȵкͿкͣӶЧţȻrAAV¿Ǽʹö¿Tϸ߽鵼ӶɨתİϸЧʧص߷Ӧʵ鹤ߵʧMingozzi et al., 2013
rAAVĵζȼʹüӦֻܼһģڣԽԽãʹõЧ
ͼ1 ϵͳתȾЧ֮Ĺϵ
Mingozzi et al., 2013
ȷrAAVζ
ֻζʱǻ
ֻʱǻ徲
ֻʱǻʵ
ֻʱǻȹ
ڿĵط
ֻΪṩȹ̵ĸƷʲƷ
UGר
ûʹֿɼ
Ӫҵѯ
UGиAAVװӪҵɨ·άϵUGǽϵ
ο
[1] Lock, Martin, et al. Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material. Human gene therapy 21.10 (2010): 1273-1285. PubMed PMID: 20486768. PubMed Central PMCID: PMC2957240.
[2] Zeltner, Nadja, et al. Near-perfect infectivity of wild-type AAV as benchmark for infectivity of recombinant AAV vectors. Gene therapy 17.7 (2010): 872-879. PubMed PMID: 20336156. PubMed Central PMCID: PMC2900506.
[3] Mingozzi F, High KA. Immune responses to AAV vectors: overcoming barriers to successful gene therapy. Blood. 2013 Jul 4;122(1):23-36. doi: 10.1182/blood-2013-01-306647. Epub 2013 Apr 17. PMID: 23596044; PMCID: PMC3701904.
[4] Lang JF, Toulmin SA, et al. Standard screening methods underreport AAV-mediated transduction and gene editing. Nat Commun. 2019 Jul 30;10(1):3415. doi: 10.1038/s41467-019-11321-7. PMID: 31363095; PMCID: PMC6667494.
