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

…, J Duque, M Lenser, TK Earr, H Nguyen, R Chau… - 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 –/– …

Molecular architecture determines brain delivery of a transferrin receptor–targeted lysosomal enzyme

…, JC Dugas, ME Pizzo, ER Thomsen, R Chau… - Journal of Experimental …, 2022 - rupress.org
Delivery of biotherapeutics across the blood–brain barrier (BBB) is a challenge. Many
approaches fuse biotherapeutics to platforms that bind the transferrin receptor (TfR), a brain …

[HTML][HTML] Novel App knock-in mouse model shows key features of amyloid pathology and reveals profound metabolic dysregulation of microglia

…, C Ha, AWS Leung, H Nguyen, R Chau… - Molecular …, 2022 - Springer
Background Genetic mutations underlying familial Alzheimer’s disease (AD) were identified
decades ago, but the field is still in search of transformative therapies for patients. While …

Discovery of Potent and Selective Dual Leucine Zipper Kinase/Leucine Zipper-Bearing Kinase Inhibitors with Neuroprotective Properties in In Vitro and In Vivo Models …

…, H Solanoy, H Nguyen, R Chau… - Journal of Medicinal …, 2022 - ACS Publications
Dual leucine zipper kinase (DLK) and leucine zipper-bearing kinase (LZK) are regulators of
neuronal degeneration and axon growth. Therefore, there is a considerable interest in …

Fibrillar Aβ causes profound microglial metabolic perturbations in a novel APP knock-in mouse model

…, TK Earr, CC Lin, S Davis, C Ha, H Nguyen, R Chau… - bioRxiv, 2021 - biorxiv.org
Microglial dysfunction is believed to play a pathogenic role in Alzheimer’s disease (AD).
Here, we characterize the amyloid-β related pathology and microglial responses in an …

DNL343 is an investigational CNS penetrant eIF2B activator that prevents and reverses the effects of neurodegeneration caused by the Integrated Stress Response

E Yulyaningsih, JH Suh, MH Fanok, R Chau… - bioRxiv, 2023 - biorxiv.org
The integrated stress response (ISR) is a conserved pathway in eukaryotic cells that is
activated in response to multiple sources of cellular stress. Although acute activation of this …

A brain penetrant progranulin biotherapeutic rescues lysosomal and inflammatory phenotypes in the brain of GRN knockout mice: Lipids in AD and FTD: From genes …

…, H Tran, M Lenser, H Nguyen, R Chau… - Alzheimer's & …, 2020 - Wiley Online Library
Background Heterozygous loss of function (LOF) mutations in GRN cause frontotemporal
dementia (FTD), a neurodegenerative disorder associated with lysosomal dysfunction, TDP‐43 …

Erratum: Rescue of a lysosomal storage disorder caused by Grn loss of function with a brain penetrant progranulin biologic (Cell (2021) 184 (18)(4651–4668. e25) …

…, J Duque, M Lenser, TK Earr, H Nguyen, R Chau… - …, 2024 - mayoclinic.elsevierpure.com
(Cell 184, 4651–4668. e1–e25; September 2, 2021) In the originally published version of this
article, we reported tissue and fluid concentrations of PTV: PGRN and Fc: PGRN following …

The Integrated Stress Response Is Modulated by eIF2B Agonist DNL343: Results From Phase 1 Healthy Subject and Phase 1b ALS Patient Studies.(P8-8.010)

…, B Willman-Yoswa, R Maciuca, S Dobbins, R Chau… - 2023 - AAN Enterprises
… Miss Chau has received personal compensation for serving as an employee of Denali
Therapeutics. Miss Chau has stock in Denali Therapeutics. Timothy Earr has received personal …

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

…, J Duque, M Lenser, TK Earr, H Nguyen, R Chau… - Cell, 2024 - pubmed.ncbi.nlm.nih.gov
… Todd Logan, Matthew J Simon, Anil Rana, Gerald M Cherf, Ankita Srivastava, Sonnet S Davis,
Ray Lieh Yoon Low, Chi-Lu Chiu, Meng Fang, Fen Huang, Akhil Bhalla, Ceyda Llapashtica …