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 …

Viral strategies for targeting the central and peripheral nervous systems

CN Bedbrook, BE Deverman… - Annual review of …, 2018 - annualreviews.org
Recombinant viruses allow for targeted transgene expression in specific cell populations
throughout the nervous system. The adeno-associated virus (AAV) is among the most …

HIV-1 nuclear import in macrophages is regulated by CPSF6-capsid interactions at the nuclear pore complex

DA Bejarano, K Peng, V Laketa, K Börner, KL Jost… - Elife, 2019 - elifesciences.org
Nuclear entry of HIV-1 replication complexes through intact nuclear pore complexes is
critical for successful infection. The host protein cleavage-and-polyadenylation-specificity …

Mechanistic model for production of recombinant adeno-associated virus via triple transfection of HEK293 cells

TNT Nguyen, S Sha, MS Hong, AJ Maloney… - … Therapy Methods & …, 2021 - cell.com
Manufacturing of recombinant adeno-associated virus (rAAV) viral vectors remains
challenging, with low yields and low full: empty capsid ratios in the harvest. To elucidate the …

[HTML][HTML] Targeting microglia with lentivirus and AAV: Recent advances and remaining challenges

ME Maes, G Colombo, R Schulz, S Siegert - Neuroscience letters, 2019 - Elsevier
Microglia have emerged as a critical component of neurodegenerative diseases. Genetic
manipulation of microglia can elucidate their functional impact in disease. In neuroscience …

Small but increasingly mighty: latest advances in AAV vector research, design, and evolution

D Grimm, H Büning - Human gene therapy, 2017 - liebertpub.com
Recombinant gene delivery vectors derived from naturally occurring or genetically
engineered adeno-associated viruses (AAV) have taken center stage in human gene …

[HTML][HTML] Using a barcoded AAV capsid library to select for clinically relevant gene therapy vectors

K Pekrun, G De Alencastro, QJ Luo, J Liu, Y Kim… - JCI insight, 2019 - ncbi.nlm.nih.gov
While gene transfer using recombinant adeno-associated viral (rAAV) vectors has shown
success in some clinical trials, there remain many tissues that are not well transduced …

Pre-arrayed pan-AAV peptide display libraries for rapid single-round screening

K Börner, E Kienle, LY Huang, J Weinmann, A Sacher… - Molecular therapy, 2020 - cell.com
Display of short peptides on the surface of adeno-associated viruses (AAVs) is a powerful
technology for the generation of gene therapy vectors with altered cell specificities and/or …

Adeno-associated virus genome interactions important for vector production and transduction

AC Maurer, MD Weitzman - Human Gene Therapy, 2020 - liebertpub.com
Recombinant adeno-associated virus has emerged as one of the most promising gene
therapy delivery vectors. Development of these vectors took advantage of key features of the …

The assembly-activating protein promotes stability and interactions between AAV's viral proteins to nucleate capsid assembly

AC Maurer, S Pacouret, AKC Diaz, J Blake… - Cell reports, 2018 - cell.com
The adeno-associated virus (AAV) vector is a preferred delivery platform for in vivo gene
therapy. Natural and engineered variations of the AAV capsid affect a plurality of phenotypes …