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TitleProduction and characterization of an AAV1-VP3-only capsid: An analytical benchmark standard.
Journal, issue, pagesMol Ther Methods Clin Dev, Vol. 29, Page 460-472, Year 2023
Publish dateJun 8, 2023
AuthorsMario Mietzsch / Weijing Liu / Ke Ma / Antonette Bennett / Austin R Nelson / Keely Gliwa / Paul Chipman / Xiaofeng Fu / Shane Bechler / Robert McKenna / Rosa Viner /
PubMed AbstractAdeno-associated viruses (AAVs) are non-enveloped ssDNA icosahedral T = 1 viruses used as vectors for clinical gene delivery. Currently, there are over 200 AAV-related clinical trials and six ...Adeno-associated viruses (AAVs) are non-enveloped ssDNA icosahedral T = 1 viruses used as vectors for clinical gene delivery. Currently, there are over 200 AAV-related clinical trials and six approved biologics on the market. As such new analytical methods are continually being developed to characterize and monitor the quality and purity of manufactured AAV vectors, these include ion-exchange chromatography and Direct Mass Technology. However, these methods require homogeneous analytical standards with a high molecular weight standard comparable to the mass of an AAV capsid. Described here is the design, production, purification, characterization, and the cryo-electron microscopy structure of an AAV1-VP3-only capsid that fulfills this need as a calibrant to determine capsid mass, charge, homogeneity, and transgene packaging characteristics.
External linksMol Ther Methods Clin Dev / PubMed:37273903 / PubMed Central
MethodsEM (single particle)
Resolution2.27 Å
Structure data

29377

8fq4

EMDB-29377, PDB-8fq4:
AAV1 VP3 Only Capsid
Method: EM (single particle) / Resolution: 2.27 Å

Source
  • adeno-associated virus - 1
KeywordsVIRUS LIKE PARTICLE / AAV / gene therapy / capsid / VP3

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