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 …

Various AAV serotypes and their applications in gene therapy: an overview

SS Issa, AA Shaimardanova, VV Solovyeva… - Cells, 2023 - mdpi.com
Despite scientific discoveries in the field of gene and cell therapy, some diseases still have
no effective treatment. Advances in genetic engineering methods have enabled the …

Epidemiological and cohort study finds no association between COVID-19 and Guillain-Barré syndrome

S Keddie, J Pakpoor, C Mousele, M Pipis, PM Machado… - Brain, 2021 - academic.oup.com
Abstract Reports of Guillain-Barré syndrome (GBS) have emerged during the Coronavirus
disease 2019 (COVID-19) pandemic. This epidemiological and cohort study sought to …

[HTML][HTML] Manufacturing challenges and rational formulation development for AAV viral vectors

A Srivastava, KMG Mallela, N Deorkar… - Journal of pharmaceutical …, 2021 - Elsevier
Adeno-associated virus (AAV) has emerged as a leading platform for gene delivery for
treating various diseases due to its excellent safety profile and efficient transduction to …

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 …

Adeno-associated viruses (AAV) and host immunity–a race between the hare and the hedgehog

K Rapti, D Grimm - Frontiers in immunology, 2021 - frontiersin.org
Adeno-associated viruses (AAV) have emerged as the lead vector in clinical trials and form
the basis for several approved gene therapies for human diseases, mainly owing to their …

Production, processing, and characterization of synthetic AAV gene therapy vectors

J El Andari, D Grimm - Biotechnology journal, 2021 - Wiley Online Library
Over the last two decades, gene therapy vectors based on wild‐type Adeno‐associated
viruses (AAV) are safe and efficacious in numerous clinical trials and are translated into …

[HTML][HTML] Chromatographic strategies for the analytical characterization of adeno-associated virus vector-based gene therapy products

S Fekete, MK Aebischer, M Imiołek, T Graf… - TrAC Trends in …, 2023 - Elsevier
In recent years, the biopharmaceutical industry's interest in gene therapy modalities has
increased dramatically. To warrant their quality during manufacturing and …

Functional roles of the membrane-associated AAV protein MAAP

L Galibert, A Hyvönen, RAE Eriksson, S Mattola… - Scientific reports, 2021 - nature.com
With a limited coding capacity of 4.7 kb, adeno-associated virus (AAV) genome has evolved
over-lapping genes to maximise the usage of its genome. An example is the recently found …

Glycosylation shapes the efficacy and safety of diverse protein, gene and cell therapies

F Rocamora, AG Peralta, S Shin, J Sorrentino… - Biotechnology …, 2023 - Elsevier
Over recent decades, therapeutic proteins have had widespread success in treating a
myriad of diseases. Glycosylation, a near universal feature of this class of drugs, is a critical …