Structure-guided AAV capsid evolution strategies for enhanced CNS gene delivery

TJ Gonzalez, A Mitchell-Dick, LO Blondel… - Nature …, 2023 - nature.com
Over the past 5 years, our laboratory has systematically developed a structure-guided library
approach to evolve new adeno-associated virus (AAV) capsids with altered tissue tropism …

Parvovirus glycan interactions

LY Huang, S Halder, M Agbandje-McKenna - Current opinion in virology, 2014 - Elsevier
Highlights•Glycan interactions are an essential first step in successful host infection by the
Parvovirinae subfamily of the Parvoviridae.•The binding sites for glycans overlap with …

Compact RNA editors with small Cas13 proteins

S Kannan, H Altae-Tran, X Jin, VJ Madigan… - Nature …, 2022 - nature.com
CRISPR–Cas13 systems have been developed for precise RNA editing, and can potentially
be used therapeutically when temporary changes are desirable or when DNA editing is …

Post‐translational modifications in capsid proteins of recombinant adeno‐associated virus (AAV) 1‐rh10 serotypes

B Mary, S Maurya, S Arumugam, V Kumar… - The FEBS …, 2019 - Wiley Online Library
Post‐translational modifications in viral capsids are known to fine‐tune and regulate several
aspects of the infective life cycle of several viruses in the host. Recombinant viruses that are …

Completion of the AAV structural atlas: serotype capsid structures reveals clade-specific features

M Mietzsch, A Jose, P Chipman, N Bhattacharya… - Viruses, 2021 - mdpi.com
The capsid structures of most Adeno-associated virus (AAV) serotypes, already assigned to
an antigenic clade, have been previously determined. This study reports the remaining …

Systemic delivery of adeno-associated viral vectors

D Duan - Current opinion in virology, 2016 - Elsevier
Highlights•Only intravascular delivery can truly change the course of systemic
diseases.•AAV has the capacity to escape from the blood and lead to bodywide gene …

Differential adeno-associated virus serotype-specific interaction patterns with synthetic heparins and other glycans

M Mietzsch, F Broecker, A Reinhardt… - Journal of …, 2014 - Am Soc Microbiol
All currently identified primary receptors of adeno-associated virus (AAV) are glycans.
Depending on the AAV serotype, these carbohydrates range from heparan sulfate …

Structure of the gene therapy vector, adeno-associated virus with its cell receptor, AAVR

NL Meyer, G Hu, O Davulcu, Q Xie, AJ Noble… - Elife, 2019 - elifesciences.org
Adeno-associated virus (AAV) vectors are preeminent in emerging clinical gene therapies.
Generalizing beyond the most tractable genetic diseases will require modulation of cell …

Characterization of the adeno-associated virus 1 and 6 sialic acid binding site

LY Huang, A Patel, R Ng, EB Miller, S Halder… - Journal of …, 2016 - Am Soc Microbiol
The adeno-associated viruses (AAVs), which are being developed as gene delivery vectors,
display differential cell surface glycan binding and subsequent tissue tropisms. For AAV …

Adeno-associated virus engineering and load strategy for tropism modification, immune evasion and enhanced transgene expression

X Zhou, J Liu, S Xiao, X Liang, Y Li, F Mo… - International Journal …, 2024 - Taylor & Francis
Gene therapy aims to add, replace or turn off genes to help treat disease. To date, the US
Food and Drug Administration (FDA) has approved 14 gene therapy products. With the …