207 related articles for article (PubMed ID: 34411609)
1. Disease modeling for Mucopolysaccharidosis type IIIB using patient derived induced pluripotent stem cells.
Huang W; Cheng YS; Yang S; Swaroop M; Xu M; Huang W; Zheng W
Exp Cell Res; 2021 Oct; 407(1):112785. PubMed ID: 34411609
[TBL] [Abstract][Full Text] [Related]
2. 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]
3. Competitive binding of extracellular accumulated heparan sulfate reduces lysosomal storage defects and triggers neuronal differentiation in a model of Mucopolysaccharidosis IIIB.
De Pasquale V; Scerra G; Scarcella M; D'Agostino M; Pavone LM
Biochim Biophys Acta Mol Cell Res; 2021 Oct; 1868(11):119113. PubMed ID: 34329663
[TBL] [Abstract][Full Text] [Related]
4. Disease correction in mucopolysaccharidosis type IIIB mice by intraparenchymal or cisternal delivery of a capsid modified AAV8 codon-optimized NAGLU vector.
Rouse CJ; Hawkins K; Kabbej N; Dalugdug J; Kunta A; Kim MJ; Someya S; Herbst Z; Gelb M; Dinelli I; Butterworth E; Falk DJ; Rosenkrantz E; Elmohd H; Khaledi H; Mowafy S; Ashby F; Heldermon CD
Hum Mol Genet; 2023 Jan; 32(3):417-430. PubMed ID: 35997776
[TBL] [Abstract][Full Text] [Related]
5. Generation of two induced pluripotent stem cells lines from a Mucopolysaccharydosis IIIB (MPSIIIB) patient.
Vallejo-Diez S; Fleischer A; Martín-Fernández JM; Sánchez-Gilabert A; Bachiller D
Stem Cell Res; 2018 Dec; 33():180-184. PubMed ID: 30408744
[TBL] [Abstract][Full Text] [Related]
6. Molecular genetics of mucopolysaccharidosis type IIIA and IIIB: Diagnostic, clinical, and biological implications.
Yogalingam G; Hopwood JJ
Hum Mutat; 2001 Oct; 18(4):264-81. PubMed ID: 11668611
[TBL] [Abstract][Full Text] [Related]
7. Residual N-acetyl-α-glucosaminidase activity in fibroblasts correlates with disease severity in patients with mucopolysaccharidosis type IIIB.
Meijer OLM; Welling L; Valstar MJ; Hoefsloot LH; Brüggenwirth HT; van der Ploeg AT; Ruijter GJG; Wagemans T; Wijburg FA; van Vlies N
J Inherit Metab Dis; 2016 May; 39(3):437-445. PubMed ID: 26907177
[TBL] [Abstract][Full Text] [Related]
8. An induced pluripotent stem cell line (TRNDi006-A) from a MPS IIIB patient carrying homozygous mutation of p.Glu153Lys in the NAGLU gene.
Huang W; Xu M; Li R; Baskfield A; Kouznetsova J; Beers J; Zou J; Liu C; Zheng W
Stem Cell Res; 2019 May; 37():101427. PubMed ID: 30933722
[TBL] [Abstract][Full Text] [Related]
9. Modeling neuronal defects associated with a lysosomal disorder using patient-derived induced pluripotent stem cells.
Lemonnier T; Blanchard S; Toli D; Roy E; Bigou S; Froissart R; Rouvet I; Vitry S; Heard JM; Bohl D
Hum Mol Genet; 2011 Sep; 20(18):3653-66. PubMed ID: 21685203
[TBL] [Abstract][Full Text] [Related]
10. Glycosaminoglycans and mucopolysaccharidosis type III.
Jakobkiewicz-Banecka J; Gabig-Ciminska M; Kloska A; Malinowska M; Piotrowska E; Banecka-Majkutewicz Z; Banecki B; Wegrzyn A; Wegrzyn G
Front Biosci (Landmark Ed); 2016 Jun; 21(7):1393-409. PubMed ID: 27100513
[TBL] [Abstract][Full Text] [Related]
11. Mucopolysaccharidosis IIIB and mild skeletal anomalies: coexistence of NAGLU and CYP26B1 missense variations in the same patient in a Chinese family.
Li J; Xie H; Jiang Y
BMC Med Genet; 2018 Apr; 19(1):51. PubMed ID: 29606097
[TBL] [Abstract][Full Text] [Related]
12. Biochemical evaluation of intracerebroventricular rhNAGLU-IGF2 enzyme replacement therapy in neonatal mice with Sanfilippo B syndrome.
Kan SH; Elsharkawi I; Le SQ; Prill H; Mangini L; Cooper JD; Lawrence R; Sands MS; Crawford BE; Dickson PI
Mol Genet Metab; 2021 Jun; 133(2):185-192. PubMed ID: 33839004
[TBL] [Abstract][Full Text] [Related]
13. Macrophage enzyme and reduced inflammation drive brain correction of mucopolysaccharidosis IIIB by stem cell gene therapy.
Holley RJ; Ellison SM; Fil D; O'Leary C; McDermott J; Senthivel N; Langford-Smith AWW; Wilkinson FL; D'Souza Z; Parker H; Liao A; Rowlston S; Gleitz HFE; Kan SH; Dickson PI; Bigger BW
Brain; 2018 Jan; 141(1):99-116. PubMed ID: 29186350
[TBL] [Abstract][Full Text] [Related]
14. Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB.
Kan SH; Aoyagi-Scharber M; Le SQ; Vincelette J; Ohmi K; Bullens S; Wendt DJ; Christianson TM; Tiger PM; Brown JR; Lawrence R; Yip BK; Holtzinger J; Bagri A; Crippen-Harmon D; Vondrak KN; Chen Z; Hague CM; Woloszynek JC; Cheung DS; Webster KA; Adintori EG; Lo MJ; Wong W; Fitzpatrick PA; LeBowitz JH; Crawford BE; Bunting S; Dickson PI; Neufeld EF
Proc Natl Acad Sci U S A; 2014 Oct; 111(41):14870-5. PubMed ID: 25267636
[TBL] [Abstract][Full Text] [Related]
15. BMN 250, a fusion of lysosomal alpha-N-acetylglucosaminidase with IGF2, exhibits different patterns of cellular uptake into critical cell types of Sanfilippo syndrome B disease pathogenesis.
Yogalingam G; Luu AR; Prill H; Lo MJ; Yip B; Holtzinger J; Christianson T; Aoyagi-Scharber M; Lawrence R; Crawford BE; LeBowitz JH
PLoS One; 2019; 14(1):e0207836. PubMed ID: 30657762
[TBL] [Abstract][Full Text] [Related]
16. Untypically mild phenotype of a patient suffering from Sanfilippo syndrome B with the c.638C>T/c.889C>T (p.Pro213Leu/p.Arg297Ter) mutations in the NAGLU gene.
Pierzynowska K; Mański A; Limanówka M; Wierzba J; Gaffke L; Anikiej P; Węgrzyn G
Mol Genet Genomic Med; 2020 Sep; 8(9):e1356. PubMed ID: 32578945
[TBL] [Abstract][Full Text] [Related]
17. A model of mucopolysaccharidosis IIIB (Sanfilippo syndrome type IIIB): N-acetyl-alpha-D-glucosaminidase deficiency in Schipperke dogs.
Ellinwood NM; Wang P; Skeen T; Sharp NJ; Cesta M; Decker S; Edwards NJ; Bublot I; Thompson JN; Bush W; Hardam E; Haskins ME; Giger U
J Inherit Metab Dis; 2003; 26(5):489-504. PubMed ID: 14518829
[TBL] [Abstract][Full Text] [Related]
18. Molecular defects in Sanfilippo syndrome type B (mucopolysaccharidosis IIIB).
Beesley CE; Jackson M; Young EP; Vellodi A; Winchester BG
J Inherit Metab Dis; 2005; 28(5):759-67. PubMed ID: 16151907
[TBL] [Abstract][Full Text] [Related]
19. Trehalose reduces retinal degeneration, neuroinflammation and storage burden caused by a lysosomal hydrolase deficiency.
Lotfi P; Tse DY; Di Ronza A; Seymour ML; Martano G; Cooper JD; Pereira FA; Passafaro M; Wu SM; Sardiello M
Autophagy; 2018; 14(8):1419-1434. PubMed ID: 29916295
[TBL] [Abstract][Full Text] [Related]
20. [Postnatal and prenatal diagnosis of mucopolysaccharidosis type III (Sanfilippo syndrome)].
Zhang WM; Shi HP; Meng Y; Li BT; Qiu ZQ; Liu JT
Zhonghua Er Ke Za Zhi; 2008 Jun; 46(6):407-10. PubMed ID: 19099774
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
