AAV-based in vivo gene therapy for neurological disorders

Q Ling, JA Herstine, A Bradbury, SJ Gray - Nature Reviews Drug …, 2023 - nature.com
Recent advancements in gene supplementation therapy are expanding the options for the
treatment of neurological disorders. Among the available delivery vehicles, adeno …

[HTML][HTML] Neurodegeneration cell per cell

S Balusu, R Praschberger, E Lauwers, B De Strooper… - Neuron, 2023 - cell.com
The clinical definition of neurodegenerative diseases is based on symptoms that reflect
terminal damage of specific brain regions. This is misleading as it tells little about the initial …

A comparative analysis of microglial inducible Cre lines

TE Faust, PA Feinberg, C O'Connor, R Kawaguchi… - Cell reports, 2023 - cell.com
Cre/loxP technology has revolutionized genetic studies and allowed for spatial and temporal
control of gene expression in specific cell types. Microglial biology has particularly benefited …

Human OPRM1 and murine Oprm1 promoter driven viral constructs for genetic access to μ-opioidergic cell types

GJ Salimando, S Tremblay, BA Kimmey, J Li… - Nature …, 2023 - nature.com
With concurrent global epidemics of chronic pain and opioid use disorders, there is a critical
need to identify, target and manipulate specific cell populations expressing the mu-opioid …

Adeno-associated virus–based gene therapy treats inflammatory kidney disease in mice

G Wu, S Liu, J Hagenstein, M Alawi, FE Hengel… - The Journal of clinical …, 2024 - jci.org
Adeno-associated virus (AAV) is a promising in vivo gene delivery platform showing
advantages in delivering therapeutic molecules to difficult or undruggable cells. However …

High-titer AAV disrupts cerebrovascular integrity and induces lymphocyte infiltration in adult mouse brain

Y Guo, J Chen, W Ji, L Xu, Y **e, S He, C Lai… - … Therapy-Methods & …, 2023 - cell.com
The brain is often described as an" immune-privileged" organ due to the presence of the
blood-brain-barrier (BBB), which limits the entry of immune cells. In general, intracranial …

[HTML][HTML] New insights into innate immunity in Alzheimer's disease: From APOE protective variants to therapies

Y Chen, DM Holtzman - Trends in Immunology, 2024 - cell.com
Recent discoveries of rare variants of human APOE may shed light on novel therapeutic
strategies for Alzheimer's disease (AD). Here, we highlight the newly identified protective …

Movers and shakers: microglial dynamics and modulation of neural networks

BS Whitelaw, MB Stoessel, AK Majewska - Glia, 2023 - Wiley Online Library
Microglia are multifaceted cells that act as immune sentinels, with important roles in
pathological events, but also as integral contributors to the normal development and function …

Lactate promotes microglial scar formation and facilitates locomotor function recovery by enhancing histone H4 lysine 12 lactylation after spinal cord injury

X Hu, J Huang, Z Li, J Li, F Ouyang, Z Chen… - Journal of …, 2024 - Springer
Lactate-derived histone lactylation is involved in multiple pathological processes through
transcriptional regulation. The role of lactate-derived histone lactylation in the repair of …

Targeted evolution of adeno-associated virus capsids for systemic transgene delivery to microglia and tissue-resident macrophages

A Young, B Neumann, M Segel… - Proceedings of the …, 2023 - National Acad Sciences
Tissue macrophages, including microglia, are notoriously resistant to genetic manipulation.
Here, we report the creation of Adeno-associated viruses (AAV) variants that efficiently and …