Microglia in neurodegenerative diseases: mechanism and potential therapeutic targets

C Gao, J Jiang, Y Tan, S Chen - Signal transduction and targeted …, 2023 - nature.com
Microglia activation is observed in various neurodegenerative diseases. Recent advances in
single-cell technologies have revealed that these reactive microglia were with high spatial …

[HTML][HTML] Lysosomal functions of progranulin and implications for treatment of frontotemporal dementia

MJ Simon, T Logan, SL DeVos, G Di Paolo - Trends in Cell Biology, 2023 - cell.com
Loss-of-function heterozygous mutations in GRN, the gene encoding progranulin (PGRN),
were identified in patients with frontotemporal lobar degeneration (FTLD) almost two …

[HTML][HTML] Rescue of a lysosomal storage disorder caused by Grn loss of function with a brain penetrant progranulin biologic

T Logan, MJ Simon, A Rana, GM Cherf, A Srivastava… - Cell, 2021 - cell.com
GRN mutations cause frontotemporal dementia (GRN-FTD) due to deficiency in progranulin
(PGRN), a lysosomal and secreted protein with unclear function. Here, we found that Grn …

FUS and TDP-43 phases in health and disease

B Portz, BL Lee, J Shorter - Trends in biochemical sciences, 2021 - cell.com
The distinct prion-like domains (PrLDs) of FUS and TDP-43, modulate phase transitions that
result in condensates with a range of material states. These assemblies are implicated in …

Progranulin as a therapeutic target in neurodegenerative diseases

H Rhinn, N Tatton, S McCaughey, M Kurnellas… - Trends in …, 2022 - cell.com
Progranulin (PGRN, encoded by the GRN gene) plays a key role in the development,
survival, function, and maintenance of neurons and microglia in the mammalian brain. It …

Protein condensation diseases: therapeutic opportunities

M Vendruscolo, M Fuxreiter - Nature communications, 2022 - nature.com
Condensed states of proteins, including liquid-like membraneless organelles and solid-like
aggregates, contribute in fundamental ways to the organisation and function of the cell …

Targeting neuroinflammation in Alzheimer's disease: From mechanisms to clinical applications

ZZ Si, CJ Zou, X Mei, XF Li, H Luo, Y Shen… - Neural regeneration …, 2023 - journals.lww.com
Alzheimer's disease is characterized by sustained neuroinflammation leading to memory
loss and cognitive decline. The past decade has witnessed tremendous efforts in …

Deficiency of the frontotemporal dementia gene GRN results in gangliosidosis

S Boland, S Swarup, YA Ambaw, PC Malia… - Nature …, 2022 - nature.com
Haploinsufficiency of GRN causes frontotemporal dementia (FTD). The GRN locus produces
progranulin (PGRN), which is cleaved to lysosomal granulin polypeptides. The function of …

Lysosomal dysfunction in neurodegeneration: emerging concepts and methods

V Udayar, Y Chen, E Sidransky, R Jagasia - Trends in neurosciences, 2022 - cell.com
The understanding of lysosomes has come a long way since the initial discovery of their role
in degrading cellular waste. The lysosome is now recognized as a highly dynamic organelle …

[HTML][HTML] Lysosome dysfunction as a cause of neurodegenerative diseases: Lessons from frontotemporal dementia and amyotrophic lateral sclerosis

J Root, P Merino, A Nuckols, M Johnson… - Neurobiology of disease, 2021 - Elsevier
Frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) are fatal
neurodegenerative disorders that are thought to exist on a clinical and pathological …