| 1 |
34844123 | Vesicle cholesterol controls exocytotic fusion pore. | 2022 Jan |
2 |
| 2 |
34886684 | Wnt5a Promotes Lysosomal Cholesterol Egress and Protects Against Atherosclerosis. | 2022 Jan 21 |
4 |
| 3 |
34936700 | CRISPR screens for lipid regulators reveal a role for ER-bound SNX13 in lysosomal cholesterol export. | 2022 Feb 7 |
1 |
| 4 |
34952292 | Consistently High Agreement Between Independent Raters of Niemann-Pick Type C1 Clinical Severity Scale in Phase 2/3 Trial. | 2022 Feb |
1 |
| 5 |
35007562 | Elevated granulocyte-colony stimulating factor and hematopoietic stem cell mobilization in Niemann-Pick type C1 disease. | 2022 Feb |
1 |
| 6 |
35081253 | Severely impaired CTL killing is a feature of the neurological disorder Niemann-Pick disease type C1. | 2022 Mar 24 |
2 |
| 7 |
35086560 | Identification of novel mutations among Iranian NPC1 patients: a bioinformatics approach to predict pathogenic mutations. | 2022 Jan 27 |
1 |
| 8 |
35159129 | Assessment of FDA-Approved Drugs as a Therapeutic Approach for Niemann-Pick Disease Type C1 Using Patient-Specific iPSC-Based Model Systems. | 2022 Jan 18 |
1 |
| 9 |
35159316 | Impact of Organelle Transport Deficits on Mitophagy and Autophagy in Niemann-Pick Disease Type C. | 2022 Feb 1 |
1 |
| 10 |
35263388 | Reovirus infection is regulated by NPC1 and endosomal cholesterol homeostasis. | 2022 Mar |
4 |
| 11 |
35370195 | [Inclusion Solves Insolubility -Translational Research Cycle from Bedside to Bench and Bench to Bedside for Drug Development Targeting Niemann-Pick Disease Type C]. | 2022 |
2 |
| 12 |
35428843 | Sterolight as imaging tool to study sterol uptake, trafficking and efflux in living cells. | 2022 Apr 15 |
1 |
| 13 |
35587334 | CRISPR-Cas9-Mediated NPC1 Gene Deletion Enhances HEK 293 T Cell Adhesion by Regulating E-Cadherin. | 2022 May 19 |
1 |
| 14 |
35626680 | Stable Integration of Inducible SPLICS Reporters Enables Spatio-Temporal Analysis of Multiple Organelle Contact Sites upon Modulation of Cholesterol Traffic. | 2022 May 14 |
2 |
| 15 |
35631123 | Redirecting Imipramine against Bluetongue Virus Infection: Insights from a Genome-wide Haploid Screening Study. | 2022 May 22 |
1 |
| 16 |
32880929 | Molecular dynamics study with mutation shows that N-terminal domain structural re-orientation in Niemann-Pick type C1 is required for proper alignment of cholesterol transport. | 2021 Mar |
13 |
| 17 |
33256498 | Gene Therapy in a Mouse Model of Niemann-Pick Disease Type C1. | 2021 Jun |
1 |
| 18 |
33308480 | NPC1-mTORC1 Signaling Couples Cholesterol Sensing to Organelle Homeostasis and Is a Targetable Pathway in Niemann-Pick Type C. | 2021 Feb 8 |
3 |
| 19 |
33376205 | Selective Aster inhibitors distinguish vesicular and nonvesicular sterol transport mechanisms. | 2021 Jan 12 |
1 |
| 20 |
33428961 | Identification of potential inhibitors of protein-protein interaction useful to fight against Ebola and other highly pathogenic viruses. | 2021 Feb |
1 |
| 21 |
33445799 | Pluripotent Stem Cells for Disease Modeling and Drug Discovery in Niemann-Pick Type C1. | 2021 Jan 12 |
1 |
| 22 |
33462517 | Ebola virus glycoprotein interacts with cholesterol to enhance membrane fusion and cell entry. | 2021 Feb |
1 |
| 23 |
33561418 | How NPC1 Loss Twists the TORCque of Lysosomes. | 2021 Feb 8 |
1 |
| 24 |
33598405 | Successful implementation of classical ketogenic dietary therapy in a patient with Niemann-Pick disease type C. | 2021 Jun |
2 |
| 25 |
33907281 | Phagocytic and pinocytic uptake of cholesterol in Tetrahymena thermophila impact differently on gene regulation for sterol homeostasis. | 2021 Apr 27 |
1 |
| 26 |
33924575 | Pathophysiological In Vitro Profile of Neuronal Differentiated Cells Derived from Niemann-Pick Disease Type C2 Patient-Specific iPSCs Carrying the NPC2 Mutations c.58G>T/c.140G>T. | 2021 Apr 13 |
3 |
| 27 |
34004149 | Lysosomes to the rescue: Anterograde axonal lysosome transport and neuronal proteostasis. | 2021 May 17 |
1 |
| 28 |
34019311 | NPC1 regulates the distribution of phosphatidylinositol 4-kinases at Golgi and lysosomal membranes. | 2021 Jul 1 |
2 |
| 29 |
34035463 | Platycodin D, a natural component of Platycodon grandiflorum, prevents both lysosome- and TMPRSS2-driven SARS-CoV-2 infection by hindering membrane fusion. | 2021 May |
3 |
| 30 |
34113546 | A phase 1/2 open label nonrandomized clinical trial of intravenous 2-hydroxypropyl-β-cyclodextrin for acute liver disease in infants with Niemann-Pick C1. | 2021 Sep |
1 |
| 31 |
34138521 | Hepatocellular carcinoma as a complication of Niemann-Pick disease type C1. | 2021 Oct |
1 |
| 32 |
34151204 | Inhibition of Histone Deacetylases 1, 2, and 3 Enhances Clearance of Cholesterol Accumulation in Niemann-Pick C1 Fibroblasts. | 2021 Jun 11 |
2 |
| 33 |
34183444 | Increased exosome secretion in neurons aging in vitro by NPC1-mediated endosomal cholesterol buildup. | 2021 Aug |
3 |
| 34 |
34192549 | Structural basis for sterol sensing by Scap and Insig. | 2021 Jun 29 |
1 |
| 35 |
34296265 | Transcriptome of HPβCD-treated Niemann-Pick disease type C1 cells highlights GPNMB as a biomarker for therapeutics. | 2021 Nov 30 |
1 |
| 36 |
34377675 | A neuropathological cell model derived from Niemann-Pick disease type C patient-specific iPSCs shows disruption of the p62/SQSTM1-KEAP1-NRF2 Axis and impaired formation of neuronal networks. | 2021 Sep |
1 |
| 37 |
34407999 | Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann-Pick disease type C1 mice. | 2021 Oct |
3 |
| 38 |
34445564 | Myelin Defects in Niemann-Pick Type C Disease: Mechanisms and Possible Therapeutic Perspectives. | 2021 Aug 17 |
2 |
| 39 |
34450201 | Identification of Niemann-Pick C1 protein as a potential novel SARS-CoV-2 intracellular target. | 2021 Oct |
1 |
| 40 |
34481829 | HSP90 inhibitors reduce cholesterol storage in Niemann-Pick type C1 mutant fibroblasts. | 2021 |
1 |
| 41 |
34502342 | The Antihypertensive Drug Telmisartan Protects Oligodendrocytes from Cholesterol Accumulation and Promotes Differentiation by a PPAR-γ-Mediated Mechanism. | 2021 Aug 30 |
2 |
| 42 |
34580197 | IP3R-driven increases in mitochondrial Ca2+ promote neuronal death in NPC disease. | 2021 Oct 5 |
4 |
| 43 |
34592985 | A human iPSC-derived inducible neuronal model of Niemann-Pick disease, type C1. | 2021 Oct 1 |
3 |
| 44 |
34700109 | Synthesis and biological evaluation of cationic TopFluor cholesterol analogues. | 2021 Dec |
3 |
| 45 |
34810067 | Comprehensive-targeted lipidomic analysis in Niemann-Pick C disease. | 2021 Dec |
1 |
| 46 |
31506030 | Molecular dynamics simulations reveal structural differences among wild-type NPC1 protein and its mutant forms. | 2020 Aug |
1 |
| 47 |
31509197 | Individualized management of genetic diversity in Niemann-Pick C1 through modulation of the Hsp70 chaperone system. | 2020 Jan 1 |
3 |
| 48 |
31902793 | STARD1 and NPC1 expression as pathological markers associated with astrogliosis in post-mortem brains from patients with Alzheimer's disease and Down syndrome. | 2020 Jan 5 |
1 |
| 49 |
31919352 | Structural basis for itraconazole-mediated NPC1 inhibition. | 2020 Jan 9 |
4 |
| 50 |
32060698 | New variants in Spanish Niemann-Pick type c disease patients. | 2020 Mar |
1 |