[HTML][HTML] AAV vectors: The Rubik's cube of human gene therapy
A Pupo, A Fernández, SH Low, A François… - Molecular Therapy, 2022 - cell.com
Defective genes account for∼ 80% of the total of more than 7,000 diseases known to date.
Gene therapy brings the promise of a one-time treatment option that will fix the errors in …
Gene therapy brings the promise of a one-time treatment option that will fix the errors in …
High-resolution native mass spectrometry
Native mass spectrometry (MS) involves the analysis and characterization of
macromolecules, predominantly intact proteins and protein complexes, whereby as much as …
macromolecules, predominantly intact proteins and protein complexes, whereby as much as …
Native mass spectrometry: recent progress and remaining challenges
Native mass spectrometry (nMS) has emerged as an important tool in studying the structure
and function of macromolecules and their complexes in the gas phase. In this review, we …
and function of macromolecules and their complexes in the gas phase. In this review, we …
Mass spectrometry using electrospray ionization
GRD Prabhu, ER Williams, M Wilm… - Nature Reviews Methods …, 2023 - nature.com
Applying a high voltage to a liquid passing through a capillary disintegrates the liquid
meniscus to generate a fine spray of charged droplets. As the droplets evaporate, polar …
meniscus to generate a fine spray of charged droplets. As the droplets evaporate, polar …
Orbitrap-based mass and charge analysis of single molecules
E Deslignière, A Rolland, EHTM Ebberink… - Accounts of chemical …, 2023 - ACS Publications
Conspectus Native mass spectrometry is nowadays widely used for determining the mass of
intact proteins and their noncovalent biomolecular assemblies. While this technology …
intact proteins and their noncovalent biomolecular assemblies. While this technology …
[HTML][HTML] Precision medicine: In vivo CAR therapy as a showcase for receptor-targeted vector platforms
A Michels, N Ho, CJ Buchholz - Molecular Therapy, 2022 - cell.com
Chimeric antigen receptor (CAR) T cells are a cancer immunotherapy of extremes.
Unprecedentedly effective, but complex and costly to manufacture, they are not yet a …
Unprecedentedly effective, but complex and costly to manufacture, they are not yet a …
[HTML][HTML] Assessing production variability in empty and filled adeno-associated viruses by single molecule mass analyses
EHTM Ebberink, A Ruisinger, M Nuebel… - … Therapy-Methods & …, 2022 - cell.com
Adeno-associated viruses (AAVs) are useful vehicles for gene therapy because of their
stability, low immunogenicity. and non-pathogenicity. However, disparity in AAV sample …
stability, low immunogenicity. and non-pathogenicity. However, disparity in AAV sample …
AAV capsid design: A Goldilocks challenge
S Zolotukhin, LH Vandenberghe - Trends in Molecular Medicine, 2022 - cell.com
In vivo therapeutic gene transfer has emerged as a novel class of medicines. Its feasibility
relies on the safe and efficacious delivery of genetic cargo to the appropriate targets. The …
relies on the safe and efficacious delivery of genetic cargo to the appropriate targets. The …
[HTML][HTML] The ice age-a review on formulation of adeno-associated virus therapeutics
P Grossen, IS Koukelli, J van Haasteren… - European Journal of …, 2023 - Elsevier
Gene therapies offer promising therapeutic alternatives for many disorders that currently lack
efficient treatment options. Due to their chemical nature and physico-chemical properties …
efficient treatment options. Due to their chemical nature and physico-chemical properties …
[HTML][HTML] Purity and DNA content of AAV capsids assessed by analytical ultracentrifugation and orthogonal biophysical techniques
K Richter, C Wurm, K Strasser, J Bauer, M Bakou… - European Journal of …, 2023 - Elsevier
Abstract Development and manufacturing adeno-associated virus (AAV)-based vectors for
gene therapy requires suitable analytical methods to assess the quality of the formulations …
gene therapy requires suitable analytical methods to assess the quality of the formulations …