Journey to the Center of the Cell: Tracing the Path of AAV Transduction

BP Dhungel, CG Bailey, JEJ Rasko - Trends in Molecular Medicine, 2021 - cell.com
As adeno-associated virus (AAV)-based gene therapies are being increasingly approved for
use in humans, it is important that we understand vector–host interactions in detail. With the …

Impact of AAV capsid-specific T-cell responses on design and outcome of clinical gene transfer trials with recombinant adeno-associated viral vectors: an evolving …

HCJ Ertl, KA High - Human gene therapy, 2017 - liebertpub.com
Recombinant adenovirus-associated (rAAV) vectors due to their ease of construction, wide
tissue tropism, and lack of pathogenicity remain at the forefront for long-term gene …

Gene therapy for alpha-1 antitrypsin deficiency: an update

D Pires Ferreira, AM Gruntman… - Expert Opinion on …, 2023 - Taylor & Francis
Introduction Altering the human genetic code has been explored since the early 1990s as a
definitive answer for the treatment of monogenic and acquired diseases which do not …

Targeting the lung epithelium after intravenous delivery by directed evolution of underexplored sites on the AAV capsid

D Goertsen, N Goeden, NC Flytzanis… - … Therapy-Methods & …, 2022 - cell.com
Advances in adeno-associated virus (AAV) engineering have provided exciting new tools for
research and potential solutions for gene therapy. However, the lung has not received the …

Radioiodinated Capsids Facilitate In Vivo Non-Invasive Tracking of Adeno-Associated Gene Transfer Vectors

P Kothari, BP De, B He, A Chen, MJ Chiuchiolo… - Scientific reports, 2017 - nature.com
Viral vector mediated gene therapy has become commonplace in clinical trials for a wide
range of inherited disorders. Successful gene transfer depends on a number of factors, of …

Tissue and cell-type-specific transduction using rAAV vectors in lung diseases

K Kochergin-Nikitsky, L Belova, A Lavrov… - Journal of molecular …, 2021 - Springer
Gene therapy of genetically determined diseases, including some pathologies of the
respiratory system, requires an efficient method for transgene delivery. Recombinant adeno …

sRAGE inhibits the mucus hypersecretion in a mouse model with neutrophilic asthma

X Zhang, J Xie, H Sun, Q Wei… - Immunological …, 2022 - Taylor & Francis
Background Neutrophilic asthma (NA) may result in irreversible airflow limitations. Soluble
advanced glycosylation receptor (sRAGE) has been shown to be associated with …

Impact of natural or synthetic singletons in the capsid of human bocavirus 1 on particle infectivity and immunoreactivity

J Fakhiri, KP Linse, M Mietzsch, M Xu… - Journal of …, 2020 - Am Soc Microbiol
ABSTRACT Human bocavirus 1 (HBoV1) is a parvovirus that gathers increasing attention
due to its pleiotropic role as a pathogen and emerging vector for human gene therapy …

Self-complementary and tyrosine-mutant rAAV vectors enhance transduction in cystic fibrosis bronchial epithelial cells

M Lopes-Pacheco, JZ Kitoko, MM Morales… - Experimental Cell …, 2018 - Elsevier
Recombinant adeno-associated virus (rAAV) vector platforms have shown considerable
therapeutic success in gene therapy for inherited disorders. In cystic fibrosis (CF) …

A Genome-Wide Knock-Out Screen Identifies Novel Host Cell Entry Factor Requirements for Divergent Adeno-Associated Virus Serotypes

AM Dudek - 2019 - search.proquest.com
Abstract Adeno-Associated Virus (AAV) is a non-pathogenic virus that has been harnessed
as a vector system for therapeutic gene transfer. Despite decades of research on AAV as a …