BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

386 related articles for article (PubMed ID: 29580682)

  • 1. Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing.
    Laoharawee K; DeKelver RC; Podetz-Pedersen KM; Rohde M; Sproul S; Nguyen HO; Nguyen T; St Martin SJ; Ou L; Tom S; Radeke R; Meyer KE; Holmes MC; Whitley CB; Wechsler T; McIvor RS
    Mol Ther; 2018 Apr; 26(4):1127-1136. PubMed ID: 29580682
    [TBL] [Abstract][Full Text] [Related]  

  • 2. 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]  

  • 3. Characterization of a novel mucopolysaccharidosis type II mouse model and recombinant AAV2/8 vector-mediated gene therapy.
    Jung SC; Park ES; Choi EN; Kim CH; Kim SJ; Jin DK
    Mol Cells; 2010 Jul; 30(1):13-8. PubMed ID: 20652491
    [TBL] [Abstract][Full Text] [Related]  

  • 4. ZFN-mediated in vivo gene editing in hepatocytes leads to supraphysiologic α-Gal A activity and effective substrate reduction in Fabry mice.
    Pagant S; Huston MW; Moreira L; Gan L; St Martin S; Sproul S; Holmes MC; Meyer K; Wechsler T; Desnick RJ; Yasuda M
    Mol Ther; 2021 Nov; 29(11):3230-3242. PubMed ID: 33775910
    [TBL] [Abstract][Full Text] [Related]  

  • 5. 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]  

  • 6. Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of
    Smith MC; Belur LR; Karlen AD; Erlanson O; Podetz-Pedersen KM; McKenzie J; Detellis J; Gagnidze K; Parsons G; Robinson N; Labarre S; Shah S; Furcich J; Lund TC; Tsai HC; McIvor RS; Bonner M
    Hum Gene Ther; 2022 Dec; 33(23-24):1279-1292. PubMed ID: 36226412
    [TBL] [Abstract][Full Text] [Related]  

  • 7. 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]  

  • 8. Changes in glycogen and glycosaminoglycan levels in hepatocytes of iduronate-2-sulfatase knockout mice before and after recombinant iduronate-2-sulfatase supplementation.
    Lee JH; Choe YH; Kim SJ; Paik KH; Jin DK
    Yonsei Med J; 2011 Mar; 52(2):263-7. PubMed ID: 21319344
    [TBL] [Abstract][Full Text] [Related]  

  • 9. 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]  

  • 10. Extension of the molecular analysis to the promoter region of the iduronate 2-sulfatase gene reveals genomic alterations in mucopolysaccharidosis type II patients with normal coding sequence.
    Brusius-Facchin AC; Abrahão L; Schwartz IV; Lourenço CM; Santos ES; Zanetti A; Tomanin R; Scarpa M; Giugliani R; Leistner-Segal S
    Gene; 2013 Sep; 526(2):150-4. PubMed ID: 23707223
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Neurologic Recovery in MPS I and MPS II Mice by AAV9-Mediated Gene Transfer to the CNS After the Development of Cognitive Dysfunction.
    Podetz-Pedersen KM; Laoharawee K; Singh S; Nguyen TT; Smith MC; Temme A; Kozarsky K; McIvor RS; Belur LR
    Hum Gene Ther; 2023 Jan; 34(1-2):8-18. PubMed ID: 36541357
    [TBL] [Abstract][Full Text] [Related]  

  • 12. A novel mucopolysaccharidosis type II mouse model with an iduronate-2-sulfatase-P88L mutation.
    Mashima R; Ohira M; Okuyama T; Onodera M; Takada S
    Sci Rep; 2023 May; 13(1):7865. PubMed ID: 37188686
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Efficient engraftment of genetically modified cells is necessary to ameliorate central nervous system involvement of murine model of mucopolysaccharidosis type II by hematopoietic stem cell targeted gene therapy.
    Miwa S; Watabe AM; Shimada Y; Higuchi T; Kobayashi H; Fukuda T; Kato F; Ida H; Ohashi T
    Mol Genet Metab; 2020 Aug; 130(4):262-273. PubMed ID: 32631737
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Therapeutic Options for Mucopolysaccharidosis II (Hunter Disease).
    Kubaski F; Vairo F; Baldo G; de Oliveira Poswar F; Corte AD; Giugliani R
    Curr Pharm Des; 2020; 26(40):5100-5109. PubMed ID: 33138761
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Auditory characteristics and therapeutic effects of enzyme replacement in mouse model of the mucopolysaccharidosis (MPS) II.
    Hong SH; Chu H; Kim KR; Ko MH; Kwon SY; Moon IJ; Chung WH; Cho YS; Kim CH; Suh MW; Choi EW; Sohn YB; Park SW; Kim SH; Cho SY; Ko AR; Jin DK
    Am J Med Genet A; 2012 Sep; 158A(9):2131-8. PubMed ID: 22847837
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Characterization of Fluid Biomarkers Reveals Lysosome Dysfunction and Neurodegeneration in Neuronopathic MPS II Patients.
    Bhalla A; Ravi R; Fang M; Arguello A; Davis SS; Chiu CL; Blumenfeld JR; Nguyen HN; Earr TK; Wang J; Astarita G; Zhu Y; Fiore D; Scearce-Levie K; Diaz D; Cahan H; Troyer MD; Harris JM; Escolar ML
    Int J Mol Sci; 2020 Jul; 21(15):. PubMed ID: 32707880
    [TBL] [Abstract][Full Text] [Related]  

  • 17. 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]  

  • 18. A Blood-Brain-Barrier-Penetrating Anti-human Transferrin Receptor Antibody Fusion Protein for Neuronopathic Mucopolysaccharidosis II.
    Sonoda H; Morimoto H; Yoden E; Koshimura Y; Kinoshita M; Golovina G; Takagi H; Yamamoto R; Minami K; Mizoguchi A; Tachibana K; Hirato T; Takahashi K
    Mol Ther; 2018 May; 26(5):1366-1374. PubMed ID: 29606503
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Molecular diagnosis of 65 families with mucopolysaccharidosis type II (Hunter syndrome) characterized by 16 novel mutations in the IDS gene: Genetic, pathological, and structural studies on iduronate-2-sulfatase.
    Kosuga M; Mashima R; Hirakiyama A; Fuji N; Kumagai T; Seo JH; Nikaido M; Saito S; Ohno K; Sakuraba H; Okuyama T
    Mol Genet Metab; 2016 Jul; 118(3):190-197. PubMed ID: 27246110
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Non-invasive intravenous administration of AAV9 transducing iduronate sulfatase leads to global metabolic correction and prevention of neurologic deficits in a mouse model of Hunter syndrome.
    Laoharawee K; Podetz-Pedersen KM; Nguyen TT; Singh SM; Smith MC; Belur LR; Low WC; Kozarsky KF; McIvor RS
    Mol Genet Metab Rep; 2023 Mar; 34():100956. PubMed ID: 36704405
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 20.