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Items: 1 to 20 of 60

1.

Treatment of adult MPSI mouse brains with IDUA-expressing mesenchymal stem cells decreases GAG deposition and improves exploratory behavior.

da Silva FH, Pereira VG, Yasumura EG, Tenório LZ, de Carvalho LP, Lisboa BC, Matsumoto PK, Stilhano RS, Samoto VY, Calegare BF, Brandão Lde C, D'Almeida V, Filippo TR, Porcionatto M, Toma L, Nader HB, Valero VB, Camassola M, Nardi NB, Han SW.

Genet Vaccines Ther. 2012 Apr 20;10(1):2. doi: 10.1186/1479-0556-10-2.

2.

Mesenchymal stem cells do not prevent antibody responses against human α-L-iduronidase when used to treat mucopolysaccharidosis type I.

Martin PK, Stilhano RS, Samoto VY, Takiya CM, Peres GB, da Silva Michelacci YM, da Silva FH, Pereira VG, D'Almeida V, Marques FL, Otake AH, Chammas R, Han SW.

PLoS One. 2014 Mar 18;9(3):e92420. doi: 10.1371/journal.pone.0092420.

3.

Nonviral in vivo gene transfer in the mucopolysaccharidosis I murine model.

Camassola M, Braga LM, Delgado-Cañedo A, Dalberto TP, Matte U, Burin M, Giugliani R, Nardi NB.

J Inherit Metab Dis. 2005;28(6):1035-43.

PMID:
16435197
4.

Treatment of the mouse model of mucopolysaccharidosis I with retrovirally transduced bone marrow.

Zheng Y, Rozengurt N, Ryazantsev S, Kohn DB, Satake N, Neufeld EF.

Mol Genet Metab. 2003 Aug;79(4):233-44.

PMID:
12948739
5.

Reversal of lysosomal storage in brain of adult MPS-I mice with intravenous Trojan horse-iduronidase fusion protein.

Boado RJ, Hui EK, Lu JZ, Zhou QH, Pardridge WM.

Mol Pharm. 2011 Aug 1;8(4):1342-50. doi: 10.1021/mp200136x.

PMID:
21667973
6.

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.

7.

Limited transgene immune response and long-term expression of human alpha-L-iduronidase in young adult mice with mucopolysaccharidosis type I by liver-directed gene therapy.

Di Domenico C, Di Napoli D, Gonzalez Y Reyero E, Lombardo A, Naldini L, Di Natale P.

Hum Gene Ther. 2006 Nov;17(11):1112-21.

PMID:
17044753
8.

Chromosomal localization of the human alpha-L-iduronidase gene (IDUA) to 4p16.3.

Scott HS, Ashton LJ, Eyre HJ, Baker E, Brooks DA, Callen DF, Sutherland GR, Morris CP, Hopwood JJ.

Am J Hum Genet. 1990 Nov;47(5):802-7.

9.

α- L-iduronidase gene-based therapy using the phiC31 system to treat mucopolysaccharidose type I mice.

Stilhano RS, Martin PK, de Melo SM, Samoto VY, Peres GB, da Silva Michelacci YM, da Silva FH, Pereira VG, D'Almeida V, da Cruz AT, Jasiulionis MG, Han SW.

J Gene Med. 2015 Jan-Feb;17(1-2):1-13. doi: 10.1002/jgm.2818.

PMID:
25597593
10.

Retroviral-vector-mediated gene therapy to mucopolysaccharidosis I mice improves sensorimotor impairments and other behavioral deficits.

Baldo G, Wozniak DF, Ohlemiller KK, Zhang Y, Giugliani R, Ponder KP.

J Inherit Metab Dis. 2013 May;36(3):499-512. doi: 10.1007/s10545-012-9530-x.

11.
12.

Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice.

Watson G, Bastacky J, Belichenko P, Buddhikot M, Jungles S, Vellard M, Mobley WC, Kakkis E.

Gene Ther. 2006 Jun;13(11):917-25.

PMID:
16482204
13.

Lipid composition of whole brain and cerebellum in Hurler syndrome (MPS IH) mice.

Heinecke KA, Peacock BN, Blazar BR, Tolar J, Seyfried TN.

Neurochem Res. 2011 Sep;36(9):1669-76. doi: 10.1007/s11064-011-0400-y.

PMID:
21253856
14.
15.

Intraperitoneal implant of recombinant encapsulated cells overexpressing alpha-L-iduronidase partially corrects visceral pathology in mucopolysaccharidosis type I mice.

Baldo G, Mayer FQ, Martinelli B, Meyer FS, Burin M, Meurer L, Tavares AM, Giugliani R, Matte U.

Cytotherapy. 2012 Aug;14(7):860-7. doi: 10.3109/14653249.2012.672730.

PMID:
22472038
16.

Increased longevity and metabolic correction following syngeneic BMT in a murine model of mucopolysaccharidosis type I.

Wolf DA, Lenander AW, Nan Z, Braunlin EA, Podetz-Pedersen KM, Whitley CB, Gupta P, Low WC, McIvor RS.

Bone Marrow Transplant. 2012 Sep;47(9):1235-40. doi: 10.1038/bmt.2011.239.

17.

Genotypic and bioinformatic evaluation of the alpha-l-iduronidase gene and protein in patients with mucopolysaccharidosis type I from Colombia, Ecuador and Peru.

Pineda T, Marie S, Gonzalez J, García AL, Acosta A, Morales M, Correa LN, Vivas R, Escobar X, Protzel A, Barba M, Ospina S, Corredor C, Mansilla S, Velasco HM.

Mol Genet Metab Rep. 2014 Oct 30;1:468-473.

18.

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.

19.

Recombinant encapsulated cells overexpressing alpha-L-iduronidase correct enzyme deficiency in human mucopolysaccharidosis type I cells.

Baldo G, Quoos Mayer F, Burin M, Carrillo-Farga J, Matte U, Giugliani R.

Cells Tissues Organs. 2012;195(4):323-9. doi: 10.1159/000327532.

PMID:
21778683
20.

alpha-L-Iduronidase transport in neurites.

Chen F, Vitry S, Hocquemiller M, Desmaris N, Ausseil J, Heard JM.

Mol Genet Metab. 2006 Apr;87(4):349-58.

PMID:
16439176
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