Sökning: onr:"swepub:oai:DiVA.org:uu-483848" >
Assessment of the p...
Assessment of the percentage of full recombinant adeno-associated virus particles in a gene therapy drug using CryoTEM
-
- Colomb-Delsuc, Mathieu (författare)
- Vironova AB, Stockholm, Sweden.
-
- Raim, Roman (författare)
- Baxalta Innovat GmbH, Orth, Austria.
-
- Fiedler, Christian (författare)
- Baxalta Innovat GmbH, Orth, Austria.
-
visa fler...
-
- Reuberger, Stefan (författare)
- Baxalta Innovat GmbH, Orth, Austria.
-
- Lengler, Johannes (författare)
- Baxalta Innovat GmbH, Orth, Austria.
-
- Nordstrom, Rickard (författare)
- Vironova AB, Stockholm, Sweden.
-
- Ryner, Martin (författare)
- Vironova AB, Stockholm, Sweden.
-
- Folea, Ioana Mihaela (författare)
- Vironova AB, Stockholm, Sweden.
-
- Kraus, Barbara (författare)
- Baxalta Innovat GmbH, Orth, Austria.
-
- Bort, Juan A. Hernandez (författare)
- Baxalta Innovat GmbH, Orth, Austria.
-
- Sintorn, Ida-Maria, 1976- (författare)
- Uppsala universitet,Avdelningen för visuell information och interaktion,Vironova AB, Stockholm, Sweden.
-
visa färre...
-
Vironova AB, Stockholm, Sweden Baxalta Innovat GmbH, Orth, Austria. (creator_code:org_t)
- 2022-06-03
- 2022
- Engelska.
-
Ingår i: PLOS ONE. - : Public Library of Science (PLoS). - 1932-6203. ; 17:6
- Relaterad länk:
-
https://doi.org/10.1...
-
visa fler...
-
https://uu.diva-port... (primary) (Raw object)
-
https://urn.kb.se/re...
-
https://doi.org/10.1...
-
visa färre...
Abstract
Ämnesord
Stäng
- In spite of continuous development of gene therapy vectors with thousands of drug candidates in clinical drug trials there are only a small number approved on the market today stressing the need to have characterization methods to assist in the validation of the drug development process. The level of packaging of the vector capsids appears to play a critical role in immunogenicity, hence an objective quantitative method assessing the content of particles containing a genome is an essential quality measurement. As transmission electron microscopy (TEM) allows direct visualization of the particles present in a specimen, it naturally seems as the most intuitive method of choice for characterizing recombinant adeno-associated virus (rAAV) particle packaging. Negative stain TEM (nsTEM) is an established characterization method for analysing the packaging of viral vectors. It has however shown limitations in terms of reliability. To overcome this drawback, we propose an analytical method based on CryoTEM that unambiguously and robustly determines the percentage of filled particles in an rAAV sample. In addition, we show that at a fixed number of vector particles the portion of filled particles correlates well with the potency of the drug. The method has been validated according to the ICH Q2 (R1) guidelines and the components investigated during the validation are presented in this study. The reliability of nsTEM as a method for the assessment of filled particles is also investigated along with a discussion about the origin of the observed variability of this method.
Ämnesord
- MEDICIN OCH HÄLSOVETENSKAP -- Medicinsk bioteknologi -- Medicinsk bioteknologi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Medical Biotechnology -- Medical Biotechnology (hsv//eng)
Publikations- och innehållstyp
- ref (ämneskategori)
- art (ämneskategori)
Hitta via bibliotek
-
PLOS ONE
(Sök värdpublikationen i LIBRIS)
Till lärosätets databas
- Av författaren/redakt...
-
Colomb-Delsuc, M ...
-
Raim, Roman
-
Fiedler, Christi ...
-
Reuberger, Stefa ...
-
Lengler, Johanne ...
-
Nordstrom, Ricka ...
-
visa fler...
-
Ryner, Martin
-
Folea, Ioana Mih ...
-
Kraus, Barbara
-
Bort, Juan A. He ...
-
Sintorn, Ida-Mar ...
-
visa färre...
- Om ämnet
-
- MEDICIN OCH HÄLSOVETENSKAP
-
MEDICIN OCH HÄLS ...
-
och Medicinsk biotek ...
-
och Medicinsk biotek ...
- Artiklar i publikationen
-
PLOS ONE
- Av lärosätet
-
Uppsala universitet