Gene Therapy of Anderson-Fabry Disease

Author(s): Antonino Tuttolomondo, Irene Simonetta*, Antonio Pinto.

Journal Name: Current Gene Therapy

Volume 19 , Issue 1 , 2019

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Tuttolomondo A, Simonetta I, Duro G, et al. Inter-familial and intra-familial phenotypic variability in three Sicilian families with Anderson-Fabry disease. Oncotarget 2017; 8(37): 61415-24.
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Tuttolomondo A, Pecoraro R, Simonetta I, et al. Neurological complications of Anderson-Fabry disease. Curr Pharm Des 2013; 19(33): 6014-30.
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Tuttolomondo A, Pecoraro R, Simonetta I, et al. Anderson-Fabry disease: A multiorgan disease. Curr Pharm Des 2013; 19(33): 5974-96.
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Palhais B, Dembic M, Sabaratnam R, et al. The prevalent deep intronic c. 639+919 G>A GLA mutation causes pseudoexon activation and Fabry disease by abolishing the binding of hnRNPA1 and hnRNP A2/B1 to a splicing silencer. Mol Genet Metab 2016; 119(3): 258-69.
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Biffi A, Montini E, Lorioli L, et al. Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science 2013; 341(6148): 1233158.
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Takahashi H, Hirai Y, Migita M, et al. Long-term systemic therapy of Fabry disease in a knockout mouse by adeno-associated virus-mediated muscle-directed gene transfer. Proc Natl Acad Sci USA 2002; 99(21): 13777-82.
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Li C, Ziegler RJ, Cherry M, et al. Adenovirus-transduced lung as a portal for delivering alpha-galactosidase A into systemic circulation for Fabry disease. Mol Ther 2002; 5(6): 745-54.
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Gori JL, Hsu PD, Maeder ML, Shen S, Welstead GG, Bumcrot D. Delivery and specificity of CRISPR-Cas9 genome editing technologies for human gene therapy. Hum Gene Ther 2015; 26(7): 443-51.
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Kaissarian N, Kang J, Shu L, Ferraz MJ, Aerts JM, Shayman JA. Dissociation of globotriaosylceramide and impaired endothelial function in α-galactosidase-A deficient EA.hy926 cells. Mol Genet Metab 2018; 125(4): 338-44.


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Article Details

VOLUME: 19
ISSUE: 1
Year: 2019
Page: [3 - 5]
Pages: 3
DOI: 10.2174/1566523219999190415160632

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