Gene Therapy for Fabry Disease: A Review of the Literature

Author:

Ruiz de Garibay Aritz Perez,Solinís María Ángeles,Rodríguez-Gascón Alicia

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),Pharmacology,General Medicine,Biotechnology

Reference81 articles.

1. Brady RO, Gal AE, Bradley RM, et al. Enzymatic defect in Fabry’s disease. N Engl J Med. 1967;276(21):1163–7.

2. Sweeley CC, Klionsky B. Fabry’s disease: classification as a sphingolipidosis and partial characterization of a novel glycolipid. J Biol Chem. 1963;238:3148–50.

3. Altarescu G, Moore DF, Pursley R, et al. Enhanced endothelium-dependent vasodilation in Fabry disease. Stroke. 2001;32(7):1559–62.

4. Desnick RJ, Ioannou YA, Eng CM. Alpha-galactosidase A deficiency: Fabry disease. In: Scriver CR, Beudet AL, Sly WS, Valle D, editors. The metabolic and molecular basis of inherited disease. 8th ed. NewYork: McGraw-Hill; 2001. p. 3733–74.

5. Spada M, Pagliardini S, Yasuda M, et al. High incidence of later-onset Fabry disease revealed by newborn screening. Am J Hum Genet. 2006;79(1):31–40.

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