Adeno-associated virus vector as a platform for gene therapy delivery

D Wang, PWL Tai, G Gao - Nature reviews Drug discovery, 2019 - nature.com
Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the
treatment of a variety of human diseases. Recent advances in develo** clinically …

[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 …

An Overview of Methods and Tools for Transfection of Eukaryotic Cells in vitro

A Fus-Kujawa, P Prus, K Bajdak-Rusinek… - … in bioengineering and …, 2021 - frontiersin.org
Transfection is a powerful analytical tool enabling studies of gene products and functions in
eukaryotic cells. Successful delivery of genetic material into cells depends on DNA quantity …

Adeno-associated virus (AAV) as a vector for gene therapy

MF Naso, B Tomkowicz, WL Perry III, WR Strohl - BioDrugs, 2017 - Springer
There has been a resurgence in gene therapy efforts that is partly fueled by the identification
and understanding of new gene delivery vectors. Adeno-associated virus (AAV) is a non …

Capsid modifications for targeting and improving the efficacy of AAV vectors

H Büning, A Srivastava - Molecular therapy Methods & clinical development, 2019 - cell.com
In the past decade, recombinant vectors based on a non-pathogenic parvovirus, the adeno-
associated virus (AAV), have taken center stage as a gene delivery vehicle for the potential …

CRISPR/Cas9 genome editing for neurodegenerative diseases

JN Nojadeh, NSB Eryilmaz, Bİ Ergüder - EXCLI journal, 2023 - pmc.ncbi.nlm.nih.gov
Gene therapy has emerged as a promising therapeutic strategy for various conditions,
including blood disorders, ocular disease, cancer, and nervous system disorders. The …

Pharmacology of recombinant adeno-associated virus production

M Penaud-Budloo, A François, N Clément… - … therapy Methods & …, 2018 - cell.com
Recombinant adeno-associated viral (rAAV) vectors have been used in more than 150
clinical trials with a good safety profile and significant clinical benefit in many genetic …

Thermal stability as a determinant of AAV serotype identity

A Bennett, S Patel, M Mietzsch, A Jose… - … Therapy Methods & …, 2017 - cell.com
Currently, there are over 150 ongoing clinical trials utilizing adeno-associated viruses
(AAVs) to target various genetic diseases, including hemophilia (AAV2 and AAV8) …

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 …

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 …