BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

199 related articles for article (PubMed ID: 36541357)

  • 1. 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]  

  • 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. Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I.
    Belur LR; Temme A; Podetz-Pedersen KM; Riedl M; Vulchanova L; Robinson N; Hanson LR; Kozarsky KF; Orchard PJ; Frey WH; Low WC; McIvor RS
    Hum Gene Ther; 2017 Jul; 28(7):576-587. PubMed ID: 28462595
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Murine Mucopolysaccharidosis Type I.
    Belur LR; Romero M; Lee J; Podetz-Pedersen KM; Nan Z; Riedl MS; Vulchanova L; Kitto KF; Fairbanks CA; Kozarsky KF; Orchard PJ; Frey WH; Low WC; McIvor RS
    Front Mol Neurosci; 2021; 14():618360. PubMed ID: 34040503
    [TBL] [Abstract][Full Text] [Related]  

  • 5. 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]  

  • 6. 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]  

  • 7. 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]  

  • 8. 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]  

  • 9. 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]  

  • 10. Status of newborn screening and follow up investigations for Mucopolysaccharidoses I and II in Taiwan.
    Chuang CK; Lin HY; Wang TJ; Huang YH; Chan MJ; Liao HC; Lo YT; Wang LY; Tu RY; Fang YY; Chen TL; Ho HC; Chiang CC; Lin SP
    Orphanet J Rare Dis; 2018 May; 13(1):84. PubMed ID: 29801497
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I.
    Wolf DA; Lenander AW; Nan Z; Belur LR; Whitley CB; Gupta P; Low WC; McIvor RS
    Neurobiol Dis; 2011 Jul; 43(1):123-33. PubMed ID: 21397026
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector.
    Kobayashi H; Carbonaro D; Pepper K; Petersen D; Ge S; Jackson H; Shimada H; Moats R; Kohn DB
    Mol Ther; 2005 May; 11(5):776-89. PubMed ID: 15851016
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human alpha-L-iduronidase gene.
    Hartung SD; Frandsen JL; Pan D; Koniar BL; Graupman P; Gunther R; Low WC; Whitley CB; McIvor RS
    Mol Ther; 2004 Jun; 9(6):866-75. PubMed ID: 15194053
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Delivery of an Adeno-Associated Virus Vector into Cerebrospinal Fluid Attenuates Central Nervous System Disease in Mucopolysaccharidosis Type II Mice.
    Hinderer C; Katz N; Louboutin JP; Bell P; Yu H; Nayal M; Kozarsky K; O'Brien WT; Goode T; Wilson JM
    Hum Gene Ther; 2016 Nov; 27(11):906-915. PubMed ID: 27510804
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Long-term nonsense suppression therapy moderates MPS I-H disease progression.
    Gunn G; Dai Y; Du M; Belakhov V; Kandasamy J; Schoeb TR; Baasov T; Bedwell DM; Keeling KM
    Mol Genet Metab; 2014 Mar; 111(3):374-381. PubMed ID: 24411223
    [TBL] [Abstract][Full Text] [Related]  

  • 16. 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]  

  • 17. 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]  

  • 18. 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]  

  • 19. Improvement of CNS defects via continuous intrathecal enzyme replacement by osmotic pump in mucopolysaccharidosis type II mice.
    Sohn YB; Lee J; Cho SY; Kim SJ; Ko AR; Nam MH; Jin DK
    Am J Med Genet A; 2013 May; 161A(5):1036-43. PubMed ID: 23529876
    [TBL] [Abstract][Full Text] [Related]  

  • 20. 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]  

    [Next]    [New Search]
    of 10.