208 related articles for article (PubMed ID: 34329663)
1. 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]
2. 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]
3. Neuropathology in mouse models of mucopolysaccharidosis type I, IIIA and IIIB.
Wilkinson FL; Holley RJ; Langford-Smith KJ; Badrinath S; Liao A; Langford-Smith A; Cooper JD; Jones SA; Wraith JE; Wynn RF; Merry CL; Bigger BW
PLoS One; 2012; 7(4):e35787. PubMed ID: 22558223
[TBL] [Abstract][Full Text] [Related]
4. Lysosomal storage of heparan sulfate causes mitochondrial defects, altered autophagy, and neuronal death in the mouse model of mucopolysaccharidosis III type C.
Pshezhetsky AV
Autophagy; 2016 Jun; 12(6):1059-60. PubMed ID: 25998837
[TBL] [Abstract][Full Text] [Related]
5. Differential distribution of heparan sulfate glycoforms and elevated expression of heparan sulfate biosynthetic enzyme genes in the brain of mucopolysaccharidosis IIIB mice.
McCarty DM; DiRosario J; Gulaid K; Killedar S; Oosterhof A; van Kuppevelt TH; Martin PT; Fu H
Metab Brain Dis; 2011 Mar; 26(1):9-19. PubMed ID: 21225451
[TBL] [Abstract][Full Text] [Related]
6. 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]
7. Central nervous system pathology in preclinical MPS IIIB dogs reveals progressive changes in clinically relevant brain regions.
Egeland MT; Tarczyluk-Wells MM; Asmar MM; Adintori EG; Lawrence R; Snella EM; Jens JK; Crawford BE; Wait JCM; McCullagh E; Pinkstaff J; Cooper JD; Ellinwood NM
Sci Rep; 2020 Nov; 10(1):20365. PubMed ID: 33230178
[TBL] [Abstract][Full Text] [Related]
8. 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]
9. 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]
10. Tralesinidase Alfa Enzyme Replacement Therapy Prevents Disease Manifestations in a Canine Model of Mucopolysaccharidosis Type IIIB.
Ellinwood NM; Valentine BN; Hess AS; Jens JK; Snella EM; Jamil M; Hostetter SJ; Jeffery ND; Smith JD; Millman ST; Parsons RL; Butt MT; Chandra S; Egeland MT; Assis AB; Nelvagal HR; Cooper JD; Nestrasil I; Mueller BA; Labounek R; Paulson A; Prill H; Liu XY; Zhou H; Lawrence R; Crawford BE; Grover A; Cherala G; Melton AC; Cherukuri A; Vuillemenot BR; Wait JCM; O'Neill CA; Pinkstaff J; Kovalchin J; Zanelli E; McCullagh E
J Pharmacol Exp Ther; 2022 Sep; 382(3):277-286. PubMed ID: 35717448
[TBL] [Abstract][Full Text] [Related]
11. Accelerated clinical disease and pathology in mucopolysaccharidosis type IIIB and GalNAc transferase double knockout mice.
Mohammed EE; Snella EM; Rutz-Mendicino MM; Echevarria FD; Awedikian R; Whitley EM; Ellinwood NM
Mol Genet Metab; 2012 Sep; 107(1-2):129-35. PubMed ID: 22867887
[TBL] [Abstract][Full Text] [Related]
12. 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]
13. 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]
14. 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]
15. Targeting Heparan Sulfate Proteoglycans as a Novel Therapeutic Strategy for Mucopolysaccharidoses.
De Pasquale V; Sarogni P; Pistorio V; Cerulo G; Paladino S; Pavone LM
Mol Ther Methods Clin Dev; 2018 Sep; 10():8-16. PubMed ID: 29942826
[TBL] [Abstract][Full Text] [Related]
16. Pathogenic Roles of Heparan Sulfate and Its Use as a Biomarker in Mucopolysaccharidoses.
Minami K; Morimoto H; Morioka H; Imakiire A; Kinoshita M; Yamamoto R; Hirato T; Sonoda H
Int J Mol Sci; 2022 Oct; 23(19):. PubMed ID: 36233030
[TBL] [Abstract][Full Text] [Related]
17. Caprine mucopolysaccharidosis-IIID: clinical, biochemical, morphological and immunohistochemical characteristics.
Jones MZ; Alroy J; Boyer PJ; Cavanagh KT; Johnson K; Gage D; Vorro J; Render JA; Common RS; Leedle RA; Lowrie C; Sharp P; Liour SS; Levene B; Hoard H; Lucas R; Hopwood JJ
J Neuropathol Exp Neurol; 1998 Feb; 57(2):148-57. PubMed ID: 9600207
[TBL] [Abstract][Full Text] [Related]
18. EGFR activation triggers cellular hypertrophy and lysosomal disease in NAGLU-depleted cardiomyoblasts, mimicking the hallmarks of mucopolysaccharidosis IIIB.
De Pasquale V; Pezone A; Sarogni P; Tramontano A; Schiattarella GG; Avvedimento VE; Paladino S; Pavone LM
Cell Death Dis; 2018 Jan; 9(2):40. PubMed ID: 29348482
[TBL] [Abstract][Full Text] [Related]
19. 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]
20. Enzyme Replacement Therapy for Mucopolysaccharidosis IIID using Recombinant Human α-
Wang F; Moen DR; Sauni C; Kan SH; Li S; Le SQ; Lomenick B; Zhang X; Ekins S; Singamsetty S; Wood J; Dickson PI; Chou TF
Mol Pharm; 2021 Jan; 18(1):214-227. PubMed ID: 33320673
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
