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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 …
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 …
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 …
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 …
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 …
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 …
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 …
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 …
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 …
relies on the safe and efficacious delivery of genetic cargo to the appropriate targets. The …