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183 related items for PubMed ID: 20652491
1. 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 [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 [Abstract] [Full Text] [Related]
3. 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 01; 15(7):1225-36. PubMed ID: 16505002 [Abstract] [Full Text] [Related]
4. 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 04; 26(4):1127-1136. PubMed ID: 29580682 [Abstract] [Full Text] [Related]
5. Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of Ex Vivo Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor Cells. 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 04; 33(23-24):1279-1292. PubMed ID: 36226412 [Abstract] [Full Text] [Related]
6. 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 04; 125(1-2):53-58. PubMed ID: 30064964 [Abstract] [Full Text] [Related]
7. 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 16; 1(9):e86696. PubMed ID: 27699273 [Abstract] [Full Text] [Related]
14. 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 16; 41(2):221-229. PubMed ID: 29168031 [Abstract] [Full Text] [Related]
19. 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 16; 52(2):263-7. PubMed ID: 21319344 [Abstract] [Full Text] [Related]
20. Preclinical studies of lymphocyte gene therapy for mild Hunter syndrome (mucopolysaccharidosis type II). Braun SE, Pan D, Aronovich EL, Jonsson JJ, McIvor RS, Whitley CB. Hum Gene Ther; 1996 Feb 10; 7(3):283-90. PubMed ID: 8835216 [Abstract] [Full Text] [Related] Page: [Next] [New Search]