Adeno-associated virus as a delivery vector for gene therapy of human diseases

JH Wang, DJ Gessler, W Zhan, TL Gallagher… - Signal transduction and …, 2024 - nature.com
Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene
therapy owing to its minimal pathogenicity and ability to establish long-term gene expression …

Evaluating the state of the science for adeno-associated virus integration: An integrated perspective

DE Sabatino, FD Bushman, RJ Chandler, RG Crystal… - Molecular Therapy, 2022 - cell.com
On August 18, 2021, the American Society of Gene and Cell Therapy (ASGCT) hosted a
virtual roundtable on adeno-associated virus (AAV) integration, featuring leading experts in …

[HTML][HTML] Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species

M Tabebordbar, KA Lagerborg, A Stanton, EM King… - Cell, 2021 - cell.com
Replacing or editing disease-causing mutations holds great promise for treating many
human diseases. Yet, delivering therapeutic genetic modifiers to specific cells in vivo has …

Endocytosis of abiotic nanomaterials and nanobiovectors: Inhibition of membrane trafficking

P Makvandi, M Chen, R Sartorius, A Zarrabi… - Nano Today, 2021 - Elsevier
Humans are exposed to nanoscopical nanobiovectors (eg coronavirus SARS-CoV-2) as
well as abiotic metal/carbon-based nanomaterials that enter cells serendipitously or …

Extracellular vesicle–encapsulated Adeno-Associated viruses for therapeutic gene delivery to the heart

X Li, S La Salvia, Y Liang, M Adamiak, E Kohlbrenner… - Circulation, 2023 - ahajournals.org
BACKGROUND: Adeno-associated virus (AAV) has emerged as one of the best tools for
cardiac gene delivery due to its cardiotropism, long-term expression, and safety. However, a …

Adeno-associated virus (AAV) serotypes have distinctive interactions with domains of the cellular AAV receptor

S Pillay, W Zou, F Cheng, AS Puschnik… - Journal of …, 2017 - journals.asm.org
Adeno-associated virus (AAV) entry is determined by its interactions with specific surface
glycans and a proteinaceous receptor (s). Adeno-associated virus receptor (AAVR)(also …

[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 …

Selection of an efficient AAV vector for robust CNS transgene expression

KS Hanlon, JC Meltzer, T Buzhdygan, MJ Cheng… - … Therapy Methods & …, 2019 - cell.com
Adeno-associated virus (AAV) capsid libraries have generated improved transgene delivery
vectors. We designed an AAV library construct, iTransduce, that combines a peptide library …

AAV2. 7m8 is a powerful viral vector for inner ear gene therapy

K Isgrig, DS McDougald, J Zhu, HJ Wang… - Nature …, 2019 - nature.com
Adeno-associated virus (AAV) has been successfully used to deliver gene therapy to
improve auditory function in mouse models of hereditary hearing loss. Many forms of …

Combining different CRISPR nucleases for simultaneous knock-in and base editing prevents translocations in multiplex-edited CAR T cells

V Glaser, C Flugel, J Kath, W Du, V Drosdek, C Franke… - Genome Biology, 2023 - Springer
Background Multiple genetic modifications may be required to develop potent off-the-shelf
chimeric antigen receptor (CAR) T cell therapies. Conventional CRISPR-Cas nucleases …