BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

139 related articles for article (PubMed ID: 29648648)

  • 1. FGF signaling deregulation is associated with early developmental skeletal defects in animal models for mucopolysaccharidosis type II (MPSII).
    Bellesso S; Salvalaio M; Lualdi S; Tognon E; Costa R; Braghetta P; Giraudo C; Stramare R; Rigon L; Filocamo M; Tomanin R; Moro E
    Hum Mol Genet; 2018 Jul; 27(13):2262-2275. PubMed ID: 29648648
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Neural cells generated from human induced pluripotent stem cells as a model of CNS involvement in mucopolysaccharidosis type II.
    Rybová J; Ledvinová J; Sikora J; Kuchař L; Dobrovolný R
    J Inherit Metab Dis; 2018 Mar; 41(2):221-229. PubMed ID: 29168031
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Correction of CNS defects in the MPSII mouse model via systemic enzyme replacement therapy.
    Polito VA; Abbondante S; Polishchuk RS; Nusco E; Salvia R; Cosma MP
    Hum Mol Genet; 2010 Dec; 19(24):4871-85. PubMed ID: 20876612
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Targeting Brain Disease in MPSII: Preclinical Evaluation of IDS-Loaded PLGA Nanoparticles.
    Rigon L; Salvalaio M; Pederzoli F; Legnini E; Duskey JT; D'Avanzo F; De Filippis C; Ruozi B; Marin O; Vandelli MA; Ottonelli I; Scarpa M; Tosi G; Tomanin R
    Int J Mol Sci; 2019 Apr; 20(8):. PubMed ID: 31022913
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Correction of Hunter syndrome in the MPSII mouse model by AAV2/8-mediated gene delivery.
    Cardone M; Polito VA; Pepe S; Mann L; D'Azzo A; Auricchio A; Ballabio A; Cosma MP
    Hum Mol Genet; 2006 Apr; 15(7):1225-36. PubMed ID: 16505002
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Enzyme replacement therapy (ERT) procedure for mucopolysaccharidosis type II (MPS II) by intraventricular administration (IVA) in murine MPS II.
    Higuchi T; Shimizu H; Fukuda T; Kawagoe S; Matsumoto J; Shimada Y; Kobayashi H; Ida H; Ohashi T; Morimoto H; Hirato T; Nishino K; Eto Y
    Mol Genet Metab; 2012 Sep; 107(1-2):122-8. PubMed ID: 22704483
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Perturbations in cell signaling elicit early cardiac defects in mucopolysaccharidosis type II.
    Costa R; Urbani A; Salvalaio M; Bellesso S; Cieri D; Zancan I; Filocamo M; Bonaldo P; Szabò I; Tomanin R; Moro E
    Hum Mol Genet; 2017 May; 26(9):1643-1655. PubMed ID: 28334757
    [TBL] [Abstract][Full Text] [Related]  

  • 8. CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome).
    Motas S; Haurigot V; Garcia M; Marcó S; Ribera A; Roca C; Sánchez X; Sánchez V; Molas M; Bertolin J; Maggioni L; León X; Ruberte J; Bosch F
    JCI Insight; 2016 Jun; 1(9):e86696. PubMed ID: 27699273
    [TBL] [Abstract][Full Text] [Related]  

  • 9. IDS crossing of the blood-brain barrier corrects CNS defects in MPSII mice.
    Polito VA; Cosma MP
    Am J Hum Genet; 2009 Aug; 85(2):296-301. PubMed ID: 19679226
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Current and Future Treatment of Mucopolysaccharidosis (MPS) Type II: Is Brain-Targeted Stem Cell Gene Therapy the Solution for This Devastating Disorder?
    Horgan C; Jones SA; Bigger BW; Wynn R
    Int J Mol Sci; 2022 Apr; 23(9):. PubMed ID: 35563245
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Evaluation of cerebrospinal fluid heparan sulfate as a biomarker of neuropathology in a murine model of mucopolysaccharidosis type II using high-sensitivity LC/MS/MS.
    Tanaka N; Kida S; Kinoshita M; Morimoto H; Shibasaki T; Tachibana K; Yamamoto R
    Mol Genet Metab; 2018 Sep; 125(1-2):53-58. PubMed ID: 30064964
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Idursulfase enzyme replacement therapy in an adult patient with severe Hunter syndrome having a novel mutation of iduronate-2-sulfatase gene.
    Christianto A; Watanabe H; Nakajima T; Inazu T
    Clin Chim Acta; 2013 Aug; 423():66-8. PubMed ID: 23726270
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Iduronate-2-sulfatase transport vehicle rescues behavioral and skeletal phenotypes in a mouse model of Hunter syndrome.
    Arguello A; Meisner R; Thomsen ER; Nguyen HN; Ravi R; Simms J; Lo I; Speckart J; Holtzman J; Gill TM; Chan D; Cheng Y; Chiu CL; Dugas JC; Fang M; Lopez IA; Solanoy H; Tsogtbaatar B; Zhu Y; Bhalla A; Henne KR; Henry AG; Delucchi A; Costanzo S; Harris JM; Diaz D; Scearce-Levie K; Sanchez PE
    JCI Insight; 2021 Oct; 6(19):. PubMed ID: 34622797
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Glial degeneration with oxidative damage drives neuronal demise in MPSII disease.
    Zalfa C; Verpelli C; D'Avanzo F; Tomanin R; Vicidomini C; Cajola L; Manara R; Sala C; Scarpa M; Vescovi AL; De Filippis L
    Cell Death Dis; 2016 Aug; 7(8):e2331. PubMed ID: 27512952
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Prevention of Neurocognitive Deficiency in Mucopolysaccharidosis Type II Mice by Central Nervous System-Directed, AAV9-Mediated Iduronate Sulfatase Gene Transfer.
    Laoharawee K; Podetz-Pedersen KM; Nguyen TT; Evenstar LB; Kitto KF; Nan Z; Fairbanks CA; Low WC; Kozarsky KF; McIvor RS
    Hum Gene Ther; 2017 Aug; 28(8):626-638. PubMed ID: 28478695
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Establishment of the Effectiveness of Early Versus Late Stem Cell Gene Therapy in Mucopolysaccharidosis II for Treating Central Versus Peripheral Disease.
    Mandolfo O; Liao A; Singh E; O'leary C; Holley RJ; Bigger BW
    Hum Gene Ther; 2024 Apr; 35(7-8):243-255. PubMed ID: 37427450
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Hematopoietic Stem Cell Gene Therapy Corrects Neuropathic Phenotype in Murine Model of Mucopolysaccharidosis Type II.
    Wakabayashi T; Shimada Y; Akiyama K; Higuchi T; Fukuda T; Kobayashi H; Eto Y; Ida H; Ohashi T
    Hum Gene Ther; 2015 Jun; 26(6):357-66. PubMed ID: 25761450
    [TBL] [Abstract][Full Text] [Related]  

  • 18. High-Throughput Liquid Chromatography-Tandem Mass Spectrometry Quantification of Glycosaminoglycans as Biomarkers of Mucopolysaccharidosis II.
    Wang J; Bhalla A; Ullman JC; Fang M; Ravi R; Arguello A; Thomsen E; Tsogtbaatar B; Guo JL; Skuja LL; Dugas JC; Davis SS; Poda SB; Gunasekaran K; Costanzo S; Sweeney ZK; Henry AG; Harris JM; Henne KR; Astarita G
    Int J Mol Sci; 2020 Jul; 21(15):. PubMed ID: 32751752
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Effect of systemic high dose enzyme replacement therapy on the improvement of CNS defects in a mouse model of mucopolysaccharidosis type II.
    Cho SY; Lee J; Ko AR; Kwak MJ; Kim S; Sohn YB; Park SW; Jin DK
    Orphanet J Rare Dis; 2015 Oct; 10():141. PubMed ID: 26520066
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Convergent molecular mechanisms underlying cognitive impairment in mucopolysaccharidosis type II.
    Corrêa T; Poswar F; Santos-Rebouças CB
    Metab Brain Dis; 2022 Aug; 37(6):2089-2102. PubMed ID: 34797484
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 7.