296 related articles for article (PubMed ID: 27484861)
1. Induced Pluripotent Stem Cells for Disease Modeling and Evaluation of Therapeutics for Niemann-Pick Disease Type A.
Long Y; Xu M; Li R; Dai S; Beers J; Chen G; Soheilian F; Baxa U; Wang M; Marugan JJ; Muro S; Li Z; Brady R; Zheng W
Stem Cells Transl Med; 2016 Dec; 5(12):1644-1655. PubMed ID: 27484861
[TBL] [Abstract][Full Text] [Related]
2. Niemann-Pick Disease Type C: Induced Pluripotent Stem Cell-Derived Neuronal Cells for Modeling Neural Disease and Evaluating Drug Efficacy.
Yu D; Swaroop M; Wang M; Baxa U; Yang R; Yan Y; Coksaygan T; DeTolla L; Marugan JJ; Austin CP; McKew JC; Gong DW; Zheng W
J Biomol Screen; 2014 Sep; 19(8):1164-73. PubMed ID: 24907126
[TBL] [Abstract][Full Text] [Related]
3. High sphingomyelin levels induce lysosomal damage and autophagy dysfunction in Niemann Pick disease type A.
Gabandé-Rodríguez E; Boya P; Labrador V; Dotti CG; Ledesma MD
Cell Death Differ; 2014 Jun; 21(6):864-75. PubMed ID: 24488099
[TBL] [Abstract][Full Text] [Related]
4. Neural stem cells for disease modeling and evaluation of therapeutics for Tay-Sachs disease.
Vu M; Li R; Baskfield A; Lu B; Farkhondeh A; Gorshkov K; Motabar O; Beers J; Chen G; Zou J; Espejo-Mojica AJ; Rodríguez-López A; Alméciga-Díaz CJ; Barrera LA; Jiang X; Ory DS; Marugan JJ; Zheng W
Orphanet J Rare Dis; 2018 Sep; 13(1):152. PubMed ID: 30220252
[TBL] [Abstract][Full Text] [Related]
5. iPS-derived neural stem cells for disease modeling and evaluation of therapeutics for mucopolysaccharidosis type II.
Hong J; Cheng YS; Yang S; Swaroop M; Xu M; Beers J; Zou J; Huang W; Marugan JJ; Cai X; Zheng W
Exp Cell Res; 2022 Mar; 412(1):113007. PubMed ID: 34990619
[TBL] [Abstract][Full Text] [Related]
6. An induced pluripotent stem cell line (TRNDi009-C) from a Niemann-Pick disease type A patient carrying a heterozygous p.L302P (c.905 T>C) mutation in the SMPD1 gene.
Baskfield A; Li R; Beers J; Zou J; Liu C; Zheng W
Stem Cell Res; 2019 Jul; 38():101461. PubMed ID: 31132580
[TBL] [Abstract][Full Text] [Related]
7. Massive Accumulation of Sphingomyelin Affects the Lysosomal and Mitochondria Compartments and Promotes Apoptosis in Niemann-Pick Disease Type A.
Carsana EV; Lunghi G; Prioni S; Mauri L; Loberto N; Prinetti A; Zucca FA; Bassi R; Sonnino S; Chiricozzi E; Duga S; Straniero L; Asselta R; Soldà G; Samarani M; Aureli M
J Mol Neurosci; 2022 Jul; 72(7):1482-1499. PubMed ID: 35727525
[TBL] [Abstract][Full Text] [Related]
8. Sphingomyelin-induced inhibition of the plasma membrane calcium ATPase causes neurodegeneration in type A Niemann-Pick disease.
Pérez-Cañamás A; Benvegnù S; Rueda CB; Rábano A; Satrústegui J; Ledesma MD
Mol Psychiatry; 2017 May; 22(5):711-723. PubMed ID: 27620840
[TBL] [Abstract][Full Text] [Related]
9. Neural stem cells for disease modeling of Wolman disease and evaluation of therapeutics.
Aguisanda F; Yeh CD; Chen CZ; Li R; Beers J; Zou J; Thorne N; Zheng W
Orphanet J Rare Dis; 2017 Jun; 12(1):120. PubMed ID: 28659158
[TBL] [Abstract][Full Text] [Related]
10. Intracerebroventricular infusion of acid sphingomyelinase corrects CNS manifestations in a mouse model of Niemann-Pick A disease.
Dodge JC; Clarke J; Treleaven CM; Taksir TV; Griffiths DA; Yang W; Fidler JA; Passini MA; Karey KP; Schuchman EH; Cheng SH; Shihabuddin LS
Exp Neurol; 2009 Feb; 215(2):349-57. PubMed ID: 19059399
[TBL] [Abstract][Full Text] [Related]
11. Neural stem cells for disease modeling and evaluation of therapeutics for infantile (CLN1/PPT1) and late infantile (CLN2/TPP1) neuronal ceroid lipofuscinoses.
Sima N; Li R; Huang W; Xu M; Beers J; Zou J; Titus S; Ottinger EA; Marugan JJ; Xie X; Zheng W
Orphanet J Rare Dis; 2018 Apr; 13(1):54. PubMed ID: 29631617
[TBL] [Abstract][Full Text] [Related]
12. Apolipoprotein D-mediated preservation of lysosomal function promotes cell survival and delays motor impairment in Niemann-Pick type A disease.
Pascua-Maestro R; Corraliza-Gomez M; Fadrique-Rojo C; Ledesma MD; Schuchman EH; Sanchez D; Ganfornina MD
Neurobiol Dis; 2020 Oct; 144():105046. PubMed ID: 32798728
[TBL] [Abstract][Full Text] [Related]
13. Lipid-induced lysosomal damage after demyelination corrupts microglia protective function in lysosomal storage disorders.
Gabandé-Rodríguez E; Pérez-Cañamás A; Soto-Huelin B; Mitroi DN; Sánchez-Redondo S; Martínez-Sáez E; Venero C; Peinado H; Ledesma MD
EMBO J; 2019 Jan; 38(2):. PubMed ID: 30530526
[TBL] [Abstract][Full Text] [Related]
14. An Early-Onset Neuronopathic Form of Acid Sphingomyelinase Deficiency: A SMPD1 p.C133Y Mutation in the Saposin Domain of Acid Sphingomyelinase.
Ota S; Noguchi A; Kondo D; Nakajima Y; Ito T; Arai H; Takahashi T
Tohoku J Exp Med; 2020 Jan; 250(1):5-11. PubMed ID: 31941852
[TBL] [Abstract][Full Text] [Related]
15.
Manthe RL; Rappaport JA; Long Y; Solomon M; Veluvolu V; Hildreth M; Gugutkov D; Marugan J; Zheng W; Muro S
J Pharmacol Exp Ther; 2019 Sep; 370(3):823-833. PubMed ID: 31101681
[TBL] [Abstract][Full Text] [Related]
16. Modeling CNS Involvement in Pompe Disease Using Neural Stem Cells Generated from Patient-Derived Induced Pluripotent Stem Cells.
Cheng YS; Yang S; Hong J; Li R; Beers J; Zou J; Huang W; Zheng W
Cells; 2020 Dec; 10(1):. PubMed ID: 33375166
[TBL] [Abstract][Full Text] [Related]
17. Sphingomyelin 16:0 is a therapeutic target for neuronal death in acid sphingomyelinase deficiency.
Gaudioso Á; Jiang X; Casas J; Schuchman EH; Ledesma MD
Cell Death Dis; 2023 Apr; 14(4):248. PubMed ID: 37024473
[TBL] [Abstract][Full Text] [Related]
18. δ-Tocopherol reduces lipid accumulation in Niemann-Pick type C1 and Wolman cholesterol storage disorders.
Xu M; Liu K; Swaroop M; Porter FD; Sidhu R; Firnkes S; Ory DS; Marugan JJ; Xiao J; Southall N; Pavan WJ; Davidson C; Walkley SU; Remaley AT; Baxa U; Sun W; McKew JC; Austin CP; Zheng W
J Biol Chem; 2012 Nov; 287(47):39349-60. PubMed ID: 23035117
[TBL] [Abstract][Full Text] [Related]
19. β-Cyclodextrin-threaded biocleavable polyrotaxanes ameliorate impaired autophagic flux in Niemann-Pick type C disease.
Tamura A; Yui N
J Biol Chem; 2015 Apr; 290(15):9442-54. PubMed ID: 25713067
[TBL] [Abstract][Full Text] [Related]
20. SMPD1 expression profile and mutation landscape help decipher genotype-phenotype association and precision diagnosis for acid sphingomyelinase deficiency.
Wang R; Qin Z; Huang L; Luo H; Peng H; Zhou X; Zhao Z; Liu M; Yang P; Shi T
Hereditas; 2023 Mar; 160(1):11. PubMed ID: 36907956
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]