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 …

Native mass spectrometry (MS) involves the analysis and characterization of
macromolecules, predominantly intact proteins and protein complexes, whereby as much as …

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 …

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 …

Conspectus Native mass spectrometry is nowadays widely used for determining the mass of
intact proteins and their noncovalent biomolecular assemblies. While this technology …

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 …

Adeno-associated viruses (AAVs) are useful vehicles for gene therapy because of their
stability, low immunogenicity. and non-pathogenicity. However, disparity in AAV sample …

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 …

Gene therapies offer promising therapeutic alternatives for many disorders that currently lack
efficient treatment options. Due to their chemical nature and physico-chemical properties …

Abstract Development and manufacturing adeno-associated virus (AAV)-based vectors for
gene therapy requires suitable analytical methods to assess the quality of the formulations …