Comprehensive characterization and quantification of adeno associated vectors by size exclusion chromatography and multi angle light scattering

Abstract Adeno associated virus (AAV) capsids are a leading modality for in vivo gene delivery. Complete and precise characterization of capsid particles, including capsid and vector genome concentration, is necessary to safely and efficaciously dose patients. Size exclusion chromatography (SEC) cou...

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Autores principales:	Nicole L. McIntosh, Geoffrey Y. Berguig, Omair A. Karim, Christa L. Cortesio, Rolando De Angelis, Ayesha A. Khan, Daniel Gold, John A. Maga, Vikas S. Bhat
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2021
Materias:	Medicine R Science Q
Acceso en línea:	https://doaj.org/article/c3c8db3276f844adbcb7cbaf0943c311
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spelling	oai:doaj.org-article:c3c8db3276f844adbcb7cbaf0943c3112021-12-02T14:06:56ZComprehensive characterization and quantification of adeno associated vectors by size exclusion chromatography and multi angle light scattering10.1038/s41598-021-82599-12045-2322https://doaj.org/article/c3c8db3276f844adbcb7cbaf0943c3112021-02-01T00:00:00Zhttps://doi.org/10.1038/s41598-021-82599-1https://doaj.org/toc/2045-2322Abstract Adeno associated virus (AAV) capsids are a leading modality for in vivo gene delivery. Complete and precise characterization of capsid particles, including capsid and vector genome concentration, is necessary to safely and efficaciously dose patients. Size exclusion chromatography (SEC) coupled to multiangle light scattering (MALS) offers a straightforward approach to comprehensively characterize AAV capsids. The current study demonstrates that this method provides detailed AAV characterization information, including but not limited to aggregation profile, size-distribution, capsid content, capsid molar mass, encapsidated DNA molar mass, and total capsid and vector genome titer. Currently, multiple techniques are required to generate this information, with varying accuracy and precision. In the current study, a new series of equations for SEC-MALS are used in tandem with intrinsic properties of the capsids and encapsidated DNA to quantify multiple physical AAV attributes in one 20-min run with minimal sample manipulation, high accuracy, and high precision. These novel applications designate this well-established method as a powerful tool for product development and process analytics in future gene therapy programs.Nicole L. McIntoshGeoffrey Y. BerguigOmair A. KarimChrista L. CortesioRolando De AngelisAyesha A. KhanDaniel GoldJohn A. MagaVikas S. BhatNature PortfolioarticleMedicineRScienceQENScientific Reports, Vol 11, Iss 1, Pp 1-12 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Medicine R Science Q
spellingShingle	Medicine R Science Q Nicole L. McIntosh Geoffrey Y. Berguig Omair A. Karim Christa L. Cortesio Rolando De Angelis Ayesha A. Khan Daniel Gold John A. Maga Vikas S. Bhat Comprehensive characterization and quantification of adeno associated vectors by size exclusion chromatography and multi angle light scattering
description	Abstract Adeno associated virus (AAV) capsids are a leading modality for in vivo gene delivery. Complete and precise characterization of capsid particles, including capsid and vector genome concentration, is necessary to safely and efficaciously dose patients. Size exclusion chromatography (SEC) coupled to multiangle light scattering (MALS) offers a straightforward approach to comprehensively characterize AAV capsids. The current study demonstrates that this method provides detailed AAV characterization information, including but not limited to aggregation profile, size-distribution, capsid content, capsid molar mass, encapsidated DNA molar mass, and total capsid and vector genome titer. Currently, multiple techniques are required to generate this information, with varying accuracy and precision. In the current study, a new series of equations for SEC-MALS are used in tandem with intrinsic properties of the capsids and encapsidated DNA to quantify multiple physical AAV attributes in one 20-min run with minimal sample manipulation, high accuracy, and high precision. These novel applications designate this well-established method as a powerful tool for product development and process analytics in future gene therapy programs.
format	article
author	Nicole L. McIntosh Geoffrey Y. Berguig Omair A. Karim Christa L. Cortesio Rolando De Angelis Ayesha A. Khan Daniel Gold John A. Maga Vikas S. Bhat
author_facet	Nicole L. McIntosh Geoffrey Y. Berguig Omair A. Karim Christa L. Cortesio Rolando De Angelis Ayesha A. Khan Daniel Gold John A. Maga Vikas S. Bhat
author_sort	Nicole L. McIntosh
title	Comprehensive characterization and quantification of adeno associated vectors by size exclusion chromatography and multi angle light scattering
title_short	Comprehensive characterization and quantification of adeno associated vectors by size exclusion chromatography and multi angle light scattering
title_full	Comprehensive characterization and quantification of adeno associated vectors by size exclusion chromatography and multi angle light scattering
title_fullStr	Comprehensive characterization and quantification of adeno associated vectors by size exclusion chromatography and multi angle light scattering
title_full_unstemmed	Comprehensive characterization and quantification of adeno associated vectors by size exclusion chromatography and multi angle light scattering
title_sort	comprehensive characterization and quantification of adeno associated vectors by size exclusion chromatography and multi angle light scattering
publisher	Nature Portfolio
publishDate	2021
url	https://doaj.org/article/c3c8db3276f844adbcb7cbaf0943c311
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Comprehensive characterization and quantification of adeno associated vectors by size exclusion chromatography and multi angle light scattering

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