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 …
treatment of neurological disorders. Among the available delivery vehicles, adeno …
[HTML][HTML] Neurodegeneration cell per cell
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 …
terminal damage of specific brain regions. This is misleading as it tells little about the initial …
A comparative analysis of microglial inducible Cre lines
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 …
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
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 …
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 …
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 …
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
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 …
strategies for Alzheimer's disease (AD). Here, we highlight the newly identified protective …
Movers and shakers: microglial dynamics and modulation of neural networks
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 …
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 …
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
Tissue macrophages, including microglia, are notoriously resistant to genetic manipulation.
Here, we report the creation of Adeno-associated viruses (AAV) variants that efficiently and …
Here, we report the creation of Adeno-associated viruses (AAV) variants that efficiently and …