These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.


BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

308 related articles for article (PubMed ID: 25761450)

  • 1. 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]  

  • 2. 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]  

  • 3. 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]  

  • 4. 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]  

  • 5. 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]  

  • 6. An empowered, clinically viable hematopoietic stem cell gene therapy for the treatment of multisystemic mucopolysaccharidosis type II.
    Das S; Rruga F; Montepeloso A; Dimartino A; Spadini S; Corre G; Patel J; Cavalca E; Ferro F; Gatti A; Milazzo R; Galy A; Politi LS; Rizzardi GP; Vallanti G; Poletti V; Biffi A
    Mol Ther; 2024 Mar; 32(3):619-636. PubMed ID: 38310355
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Enzyme augmentation therapy enhances the therapeutic efficacy of bone marrow transplantation in mucopolysaccharidosis type II mice.
    Akiyama K; Shimada Y; Higuchi T; Ohtsu M; Nakauchi H; Kobayashi H; Fukuda T; Ida H; Eto Y; Crawford BE; Brown JR; Ohashi T
    Mol Genet Metab; 2014 Feb; 111(2):139-46. PubMed ID: 24100247
    [TBL] [Abstract][Full Text] [Related]  

  • 8. 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]  

  • 9. 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]  

  • 10. Brain-targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms.
    Gleitz HF; Liao AY; Cook JR; Rowlston SF; Forte GM; D'Souza Z; O'Leary C; Holley RJ; Bigger BW
    EMBO Mol Med; 2018 Jul; 10(7):. PubMed ID: 29884617
    [TBL] [Abstract][Full Text] [Related]  

  • 11. 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]  

  • 12. Effect of donor chimerism to reduce the level of glycosaminoglycans following bone marrow transplantation in a murine model of mucopolysaccharidosis type II.
    Yokoi K; Akiyama K; Kaneshiro E; Higuchi T; Shimada Y; Kobayashi H; Akiyama M; Otsu M; Nakauchi H; Ohashi T; Ida H
    J Inherit Metab Dis; 2015 Mar; 38(2):333-40. PubMed ID: 25503568
    [TBL] [Abstract][Full Text] [Related]  

  • 13. A novel preclinical model of mucopolysaccharidosis type II for developing human hematopoietic stem cell gene therapy.
    Shimada Y; Ishii N; Higuchi T; Goto M; Ohashi T; Kobayashi H
    Gene Ther; 2023 Apr; 30(3-4):288-296. PubMed ID: 35835952
    [TBL] [Abstract][Full Text] [Related]  

  • 14. 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]  

  • 15. Reduction of GAG storage in MPS II mouse model following implantation of encapsulated recombinant myoblasts.
    Friso A; Tomanin R; Alba S; Gasparotto N; Puicher EP; Fusco M; Hortelano G; Muenzer J; Marin O; Zacchello F; Scarpa M
    J Gene Med; 2005 Nov; 7(11):1482-91. PubMed ID: 15966019
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Lentiviral Gene Therapy for Mucopolysaccharidosis II with Tagged Iduronate 2-Sulfatase Prevents Life-Threatening Pathology in Peripheral Tissues But Fails to Correct Cartilage.
    Catalano F; Vlaar EC; Dammou Z; Katsavelis D; Huizer TF; Zundo G; Hoogeveen-Westerveld M; Oussoren E; van den Hout HJMP; Schaaf G; Pike-Overzet K; Staal FJT; van der Ploeg AT; Pijnappel WWMP
    Hum Gene Ther; 2024 Apr; 35(7-8):256-268. PubMed ID: 38085235
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Fusion of Rabies Virus Glycoprotein or gh625 to Iduronate-2-Sulfatase for the Treatment of Mucopolysaccharidosis Type II.
    Wood SR; Chaudrhy A; Ellison S; Searle R; Burgod C; Tehseen G; Forte G; O'Leary C; Gleitz H; Liao A; Cook J; Holley R; Bigger BW
    Hum Gene Ther; 2024 Apr; 35(7-8):232-242. PubMed ID: 37212263
    [TBL] [Abstract][Full Text] [Related]  

  • 18. 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]  

  • 19. Non-myeloablative preconditioning with ACK2 (anti-c-kit antibody) is efficient in bone marrow transplantation for murine models of mucopolysaccharidosis type II.
    Yokoi T; Yokoi K; Akiyama K; Higuchi T; Shimada Y; Kobayashi H; Sato T; Ohteki T; Otsu M; Nakauchi H; Ida H; Ohashi T
    Mol Genet Metab; 2016 Nov; 119(3):232-238. PubMed ID: 27590924
    [TBL] [Abstract][Full Text] [Related]  

  • 20. 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]  

    [Next]    [New Search]
    of 16.