252 related articles for article (PubMed ID: 24100247)
1. 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]
2. 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]
3. 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]
4. 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]
5. First experience of enzyme replacement therapy with idursulfase in Spanish patients with Hunter syndrome under 5 years of age: case observations from the Hunter Outcome Survey (HOS).
Alcalde-Martín C; Muro-Tudelilla JM; Cancho-Candela R; Gutiérrez-Solana LG; Pintos-Morell G; Martí-Herrero M; Munguira-Aguado P; Galán-Gómez E
Eur J Med Genet; 2010; 53(6):371-7. PubMed ID: 20709629
[TBL] [Abstract][Full Text] [Related]
6. 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]
7. 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]
8. 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]
9. 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]
10. 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]
11. 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]
12. Long-term efficacy of hematopoietic stem cell transplantation on brain involvement in patients with mucopolysaccharidosis type II: a nationwide survey in Japan.
Tanaka A; Okuyama T; Suzuki Y; Sakai N; Takakura H; Sawada T; Tanaka T; Otomo T; Ohashi T; Ishige-Wada M; Yabe H; Ohura T; Suzuki N; Kato K; Adachi S; Kobayashi R; Mugishima H; Kato S
Mol Genet Metab; 2012 Nov; 107(3):513-20. PubMed ID: 23022072
[TBL] [Abstract][Full Text] [Related]
13. Bone marrow transplantation in Hunter syndrome (mucopolysaccharidosis type II): two-year follow-up of the first Italian patient and review of the literature.
Coppa GV; Gabrielli O; Zampini L; Pierani P; Giorgi PL; Jezequel AM; Orlandi F; Miniero R; Busca A; De Luca T
Pediatr Med Chir; 1995; 17(3):227-35. PubMed ID: 7567644
[TBL] [Abstract][Full Text] [Related]
14. Enzyme Replacement Therapy with Pabinafusp Alfa for Neuronopathic Mucopolysaccharidosis II: An Integrated Analysis of Preclinical and Clinical Data.
Giugliani R; Martins AM; Okuyama T; Eto Y; Sakai N; Nakamura K; Morimoto H; Minami K; Yamamoto T; Yamaoka M; Ikeda T; So S; Tanizawa K; Sonoda H; Schmidt M; Sato Y
Int J Mol Sci; 2021 Oct; 22(20):. PubMed ID: 34681597
[TBL] [Abstract][Full Text] [Related]
15. Preclinical dose ranging studies for enzyme replacement therapy with idursulfase in a knock-out mouse model of MPS II.
Garcia AR; DaCosta JM; Pan J; Muenzer J; Lamsa JC
Mol Genet Metab; 2007 Jun; 91(2):183-90. PubMed ID: 17459751
[TBL] [Abstract][Full Text] [Related]
16. A method for measuring disease-specific iduronic acid from the non-reducing end of glycosaminoglycan in mucopolysaccharidosis type II mice.
Shimada Y; Wakabayashi T; Akiyama K; Hoshina H; Higuchi T; Kobayashi H; Eto Y; Ida H; Ohashi T
Mol Genet Metab; 2016 Feb; 117(2):140-3. PubMed ID: 26051019
[TBL] [Abstract][Full Text] [Related]
17. 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]
18. 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]
19. Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy.
Wraith JE; Scarpa M; Beck M; Bodamer OA; De Meirleir L; Guffon N; Meldgaard Lund A; Malm G; Van der Ploeg AT; Zeman J
Eur J Pediatr; 2008 Mar; 167(3):267-77. PubMed ID: 18038146
[TBL] [Abstract][Full Text] [Related]
20. 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]
[Next] [New Search]
