Impact of VP1-specific protein sequence motifs on adeno-associated virus type 2 intracellular trafficking and nuclear entry

R Popa-Wagner, M Porwal, M Kann, M Reuss… - Journal of …, 2012 - Am Soc Microbiol
Adeno-associated virus type 2 (AAV2) has gained much interest as a gene delivery vector. A
hallmark of AAV2-mediated gene transfer is an intracellular conformational change of the …

Understanding and tackling immune responses to adeno-associated viral vectors

H Costa-Verdera, C Unzu, E Valeri, S Adriouch… - Human Gene …, 2023 - liebertpub.com
As the clinical experience in adeno-associated viral (AAV) vector-based gene therapies is
expanding, the necessity to better understand and control the host immune responses is …

[HTML][HTML] Development of capsid-and genome-modified optimized AAVrh74 vectors for muscle gene therapy

J Shoti, K Qing, GD Keeler, D Duan, BJ Byrne… - … Therapy Methods & …, 2023 - cell.com
The first generation of adeno-associated virus (AAV) vectors composed of the naturally
occurring capsids and genomes, although effective in some instances, are unlikely to be …

Delivering AAV to the central nervous and sensory systems

CW Peters, CA Maguire, KS Hanlon - Trends in pharmacological sciences, 2021 - cell.com
As gene therapy enters mainstream medicine, it is more important than ever to have a grasp
of exactly how to leverage it for maximum benefit. The development of new targeting …

[HTML][HTML] Local magnetic delivery of adeno-associated virus AAV2 (quad YF)-mediated BDNF gene therapy restores hearing after noise injury

S Mukherjee, M Kuroiwa, W Oakden, BT Paul… - Molecular Therapy, 2022 - cell.com
Moderate noise exposure may cause acute loss of cochlear synapses without affecting the
cochlear hair cells and hearing threshold; thus, it remains" hidden" to standard clinical tests …

[HTML][HTML] Corneal regeneration using gene therapy approaches

S Sarkar, P Panikker, S D'Souza, R Shetty, RR Mohan… - Cells, 2023 - mdpi.com
One of the most remarkable advancements in medical treatments of corneal diseases in
recent decades has been corneal transplantation. However, corneal transplants, including …

Adeno-associated virus serotype 6 capsid tyrosine-to-phenylalanine mutations improve gene transfer to skeletal muscle

C Qiao, W Zhang, Z Yuan, JH Shin, J Li… - Human gene …, 2010 - liebertpub.com
Adeno-associated viral (AAV) vectors are the most efficient in vivo gene transfer tools for
gene therapy applications. Efforts have been made to translate encouraging results in small …

AAV's anatomy: roadmap for optimizing vectors for translational success

AM Mitchell, SC Nicolson, JK Warischalk… - Current gene …, 2010 - ingentaconnect.com
Adeno-Associated Virus based vectors (rAAV) are advantageous for human gene therapy
due to low inflammatory responses, lack of toxicity, natural persistence, and ability to …

Viruses exploit the function of epidermal growth factor receptor

K Zheng, K Kitazato, Y Wang - Reviews in medical virology, 2014 - Wiley Online Library
Epidermal growth factor receptor (EGFR) is a receptor tyrosine kinase that regulates cellular
homeostatic processes. Following ligand binding, EGFR activates different downstream …

Mechanistic insights into the enhancement of adeno-associated virus transduction by proteasome inhibitors

AM Mitchell, RJ Samulski - Journal of virology, 2013 - Am Soc Microbiol
Proteasome inhibitors (eg, bortezomib, MG132) are known to enhance adeno-associated
virus (AAV) transduction; however, whether this results from pleotropic proteasome inhibition …