201 related articles for article (PubMed ID: 37628569)
1. Brain Targeted AAV1-GALC Gene Therapy Reduces Psychosine and Extends Lifespan in a Mouse Model of Krabbe Disease.
Herdt AR; Peng H; Dickson DW; Golde TE; Eckman EA; Lee CW
Genes (Basel); 2023 Jul; 14(8):. PubMed ID: 37628569
[TBL] [Abstract][Full Text] [Related]
2. Galactosylceramidase deficiency and pathological abnormalities in cerebral white matter of Krabbe disease.
Iacono D; Koga S; Peng H; Manavalan A; Daiker J; Castanedes-Casey M; Martin NB; Herdt AR; Gelb MH; Dickson DW; Lee CW
Neurobiol Dis; 2022 Nov; 174():105862. PubMed ID: 36113749
[TBL] [Abstract][Full Text] [Related]
3. Enzyme replacement therapy of a novel humanized mouse model of globoid cell leukodystrophy.
Matthes F; Andersson C; Stein A; Eistrup C; Fogh J; Gieselmann V; Wenger DA; Matzner U
Exp Neurol; 2015 Sep; 271():36-45. PubMed ID: 25956830
[TBL] [Abstract][Full Text] [Related]
4. Intravenous injection of AAVrh10-GALC after the neonatal period in twitcher mice results in significant expression in the central and peripheral nervous systems and improvement of clinical features.
Rafi MA; Rao HZ; Luzi P; Luddi A; Curtis MT; Wenger DA
Mol Genet Metab; 2015 Mar; 114(3):459-66. PubMed ID: 25533112
[TBL] [Abstract][Full Text] [Related]
5. CNS-targeted AAV5 gene transfer results in global dispersal of vector and prevention of morphological and function deterioration in CNS of globoid cell leukodystrophy mouse model.
Lin DS; Hsiao CD; Liau I; Lin SP; Chiang MF; Chuang CK; Wang TJ; Wu TY; Jian YR; Huang SF; Liu HL
Mol Genet Metab; 2011 Aug; 103(4):367-77. PubMed ID: 21620749
[TBL] [Abstract][Full Text] [Related]
6. β-Galactosylceramidase Deficiency Causes Upregulation of Long Pentraxin-3 in the Central Nervous System of Krabbe Patients and
Coltrini D; Chandran AMK; Belleri M; Poliani PL; Cominelli M; Pagani F; Capra M; Calza S; Prioni S; Mauri L; Prinetti A; Kofler JK; Escolar ML; Presta M
Int J Mol Sci; 2022 Aug; 23(16):. PubMed ID: 36012705
[TBL] [Abstract][Full Text] [Related]
7. Mechanism-based combination treatment dramatically increases therapeutic efficacy in murine globoid cell leukodystrophy.
Hawkins-Salsbury JA; Shea L; Jiang X; Hunter DA; Guzman AM; Reddy AS; Qin EY; Li Y; Gray SJ; Ory DS; Sands MS
J Neurosci; 2015 Apr; 35(16):6495-505. PubMed ID: 25904800
[TBL] [Abstract][Full Text] [Related]
8. Bone marrow transplantation increases efficacy of central nervous system-directed enzyme replacement therapy in the murine model of globoid cell leukodystrophy.
Qin EY; Hawkins-Salsbury JA; Jiang X; Reddy AS; Farber NB; Ory DS; Sands MS
Mol Genet Metab; 2012 Sep; 107(1-2):186-96. PubMed ID: 22704480
[TBL] [Abstract][Full Text] [Related]
9. Hematopoietic cell transplantation in murine globoid cell leukodystrophy (the twitcher mouse): effects on levels of galactosylceramidase, psychosine, and galactocerebrosides.
Ichioka T; Kishimoto Y; Brennan S; Santos GW; Yeager AM
Proc Natl Acad Sci U S A; 1987 Jun; 84(12):4259-63. PubMed ID: 2884662
[TBL] [Abstract][Full Text] [Related]
10. An Engineered Galactosylceramidase Construct Improves AAV Gene Therapy for Krabbe Disease in Twitcher Mice.
Pan X; Sands SA; Yue Y; Zhang K; LeVine SM; Duan D
Hum Gene Ther; 2019 Sep; 30(9):1039-1051. PubMed ID: 31184217
[TBL] [Abstract][Full Text] [Related]
11. Characterization and application of a disease-cell model for a neurodegenerative lysosomal disease.
Ribbens JJ; Moser AB; Hubbard WC; Bongarzone ER; Maegawa GH
Mol Genet Metab; 2014 Feb; 111(2):172-83. PubMed ID: 24094551
[TBL] [Abstract][Full Text] [Related]
12. Bone marrow transplantation augments the effect of brain- and spinal cord-directed adeno-associated virus 2/5 gene therapy by altering inflammation in the murine model of globoid-cell leukodystrophy.
Reddy AS; Kim JH; Hawkins-Salsbury JA; Macauley SL; Tracy ET; Vogler CA; Han X; Song SK; Wozniak DF; Fowler SC; Klein RS; Sands MS
J Neurosci; 2011 Jul; 31(27):9945-57. PubMed ID: 21734286
[TBL] [Abstract][Full Text] [Related]
13. Krabbe disease: involvement of connexin43 in the apoptotic effects of sphingolipid psychosine on mouse oligodendrocyte precursors.
Graziano AC; Parenti R; Avola R; Cardile V
Apoptosis; 2016 Jan; 21(1):25-35. PubMed ID: 26459425
[TBL] [Abstract][Full Text] [Related]
14. Efficacy and Safety of a Krabbe Disease Gene Therapy.
Hordeaux J; Jeffrey BA; Jian J; Choudhury GR; Michalson K; Mitchell TW; Buza EL; Chichester J; Dyer C; Bagel J; Vite CH; Bradbury AM; Wilson JM
Hum Gene Ther; 2022 May; 33(9-10):499-517. PubMed ID: 35333110
[TBL] [Abstract][Full Text] [Related]
15. Intraventricular administration of recombinant adenovirus to neonatal twitcher mouse leads to clinicopathological improvements.
Shen JS; Watabe K; Ohashi T; Eto Y
Gene Ther; 2001 Jul; 8(14):1081-7. PubMed ID: 11526455
[TBL] [Abstract][Full Text] [Related]
16. AAV2/5 vector expressing galactocerebrosidase ameliorates CNS disease in the murine model of globoid-cell leukodystrophy more efficiently than AAV2.
Lin D; Fantz CR; Levy B; Rafi MA; Vogler C; Wenger DA; Sands MS
Mol Ther; 2005 Sep; 12(3):422-30. PubMed ID: 15996520
[TBL] [Abstract][Full Text] [Related]
17. Neuron-specific ablation of the Krabbe disease gene galactosylceramidase in mice results in neurodegeneration.
Kreher C; Favret J; Weinstock NI; Maulik M; Hong X; Gelb MH; Wrabetz L; Feltri ML; Shin D
PLoS Biol; 2022 Jul; 20(7):e3001661. PubMed ID: 35789331
[TBL] [Abstract][Full Text] [Related]
18. Genetic ablation of acid ceramidase in Krabbe disease confirms the psychosine hypothesis and identifies a new therapeutic target.
Li Y; Xu Y; Benitez BA; Nagree MS; Dearborn JT; Jiang X; Guzman MA; Woloszynek JC; Giaramita A; Yip BK; Elsbernd J; Babcock MC; Lo M; Fowler SC; Wozniak DF; Vogler CA; Medin JA; Crawford BE; Sands MS
Proc Natl Acad Sci U S A; 2019 Oct; 116(40):20097-20103. PubMed ID: 31527255
[TBL] [Abstract][Full Text] [Related]
19. Waning efficacy in a long-term AAV-mediated gene therapy study in the murine model of Krabbe disease.
Heller GJ; Marshall MS; Issa Y; Marshall JN; Nguyen D; Rue E; Pathmasiri KC; Domowicz MS; van Breemen RB; Tai LM; Cologna SM; Crocker SJ; Givogri MI; Sands MS; Bongarzone ER
Mol Ther; 2021 May; 29(5):1883-1902. PubMed ID: 33508430
[TBL] [Abstract][Full Text] [Related]
20. Generation of a mouse with low galactocerebrosidase activity by gene targeting: a new model of globoid cell leukodystrophy (Krabbe disease).
Luzi P; Rafi MA; Zaka M; Curtis M; Vanier MT; Wenger DA
Mol Genet Metab; 2001 Jul; 73(3):211-23. PubMed ID: 11461188
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
