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 …

High-resolution native mass spectrometry

S Tamara, MA den Boer, AJR Heck - Chemical reviews, 2021 - ACS Publications
Native mass spectrometry (MS) involves the analysis and characterization of
macromolecules, predominantly intact proteins and protein complexes, whereby as much as …

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 …

Native mass spectrometry: recent progress and remaining challenges

KR Karch, DT Snyder, SR Harvey… - Annual review of …, 2022 - annualreviews.org
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 …

Single-ion mass spectrometry for heterogeneous and high molecular weight samples

MF Jarrold - Journal of the American Chemical Society, 2024 - ACS Publications
In charge detection mass spectrometry (CD-MS) the mass of each individual ion is
determined from the measurement of its mass to charge ratio (m/z) and charge. Performing …

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 …

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 …

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 …

Quantification of empty, partially filled and full adeno-associated virus vectors using mass photometry

C Wagner, FF Fuchsberger, B Innthaler… - International Journal of …, 2023 - mdpi.com
Adeno-associated viruses (AAV) are one of the most commonly used vehicles in gene
therapies for the treatment of rare diseases. During the AAV manufacturing process …

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 …