Page 64 - 8_04
P. 64
Solid lipid nanoparticles for gene therapy 75. Shen JS, Meng XL, Schiffmann R, Brady RO, Kaneski
CR. Establishment and characterization of Fabry
Terapia génica aplicada al tratamiento de disease endothelial cells with an extended lifespan.
enfermedades degenerativas de la retina. Industria
Farmacéutica 2008;139:98-105. Mol Genet Metab 2007; 92: 137-44.
61. Delgado D, del Pozo-Rodríguez A, Solinís MA, et al. 76. Shu L, Shayman JA. Caveolin-associated
Dextran and protamine-based solid lipid nanoparticles accumulation of globotriaosylceramide in the vascular
as potential vectors for the treatment of X-linked
juvenile retinoschisis. Hum Gene Ther 2012; 23: 345- endothelium of alpha-galactosidase A null mice. J
55.
Biol Chem 2007; 282: 20960-7.
62. Apaolaza PS, del Pozo-Rodríguez A, Solinís MA, et al.
Structural recovery of the retina in a retinoschisin- 77. Hepatitis C. Fact sheet. World Health Organization.
deficient mouse after gene replacement therapy by
solid lipid nanoparticles Biomaterials 2015; 90: 40-9. 2016. Disponible en:
63. Barbado Hernández FJ. Biografía de la enfermedad de (http://www.who.int/mediacentre/factsheets/fs164/en/)
Fabry. Med Clin Monogr 2010;11:3-6.
.
64. Quinta R, Rodrigues D, Assunção M, et al. Reduced
glucosylceramide in the mouse model of Fabry 78. Pungpapong S, Aqel B, Leise M, et al. Multicenter
disease: correction by successful enzyme replacement experience using simeprevir and sofosbuvir with or
therapy. Gene 2014; 536:97-104.
without ribavirin to treat hepatitis C genotype 1 after
65. Ramaswami U. Update on role of agalsidase alfa in
management of Fabry disease. Drug Des Devel Ther liver transplant. Hepatology 2015; 61:1880-6.
2011; 15: 155-173
79. Romano KP, Ali A, Aydin C, et al. The Molecular
66. Hoffman B, Garcia de Lorenzo A, Mehta A, et al. Basis of Drug Resistance against Hepatitis C Virus
Effects of enzyme replacement therapy on pain and
health related quality of life in patients with Fabry NS3/4A Protease Inhibitors. PLoS Pathog 2012; 8:
disease: data from FOS (Fabry Outcome Survey). J
Med Genet 2005; 42: 247-252. e1002832.
67. Germain DP, Charrow J, Desnick RJ, et al. Ten-year 80. Preciado MV, Valva P, Escobar-Gutierrez A, et al.
outcome of enzyme replacement therapy with Hepatitis C virus molecular evolution: transmission,
agalsidase beta in patients with Fabry disease. J Med
Genet 2015; 52: 353-8. disease progression and antiviral therapy. World J
Gastroenterol 2014; 20: 15992-6013.
68. Seydelmann N, Wanner C, Störk S, Ertl G, Weidemann
F. Fabry disease and the heart. Best Pract Res Clin 81. Chandra PK, Kundu AK, Hazari S, et al. Inhibition of
Endocrinol Metab 2015; 29: 195-204.
hepatitis C virus replication by intracellular delivery
69. Wilcox WR, Linthorst GE, Germain DP, et al. Anti-
alpha galactosidase A antibody response toa galsidase of multiple siRNAs by nanosomes. Mol Ther 2012;
beta treatment: data from de Fabry Registry. Mol 20: 1724-36.
Genet Metab 2012; 105: 443-9.
82. Huang PI, Lo WL, Cherng JY, Chien Y, Chiou GY,
70. Hopkin RJ, Cabrera G, Charrow J. Risk factors for
severe clinical events in male and female patients with Chiou SH. Non-viral delivery of RNA interference
Fabry disease treated with agalsidase beta enzyme
replacement therapy: Data from the Fabry Registry. targeting cancer cells in cancer gene therapy. Curr
Mol Genet Metab 2016; 119: 151-9. Gene Ther 2012; 12:275-84.
71. Vedder AC, Linthorst GE, Houge G, et al. Treatment 83. González-Alegre P. ARN de interferencia terapéutico
of Fabry disease: outcome of a comparative trial with
agalsidase alfa or beta at a dose of 0.2 mg/Kg. PLoS para enfermedades neurodegenerativas. Rev Neurol
One 2007; 2: e598.
2008; 47: 641-7.
72. Hollak CE, Vedder AC, Linthorst GE, Aerts JM. Novel
therapeutic targests for the treatment of Fabry disease. 84. Torrecilla J, Rodríguez-Gascón A, Solinís MÁ, del
Expert Opin Ther Targets 2007; 11: 821-33. Pozo-Rodríguez A. Lipid nanoparticles as carriers for
73. Ruiz de Garibay A, Solinís MA, Rodríguez-Gascón A. RNAi against viral infections: current status and
Gene therapy for Fabry disease: a Review of the
literature. BioDrugs 2013; 27: 237-46. future perspectives. Biomed Res Int 2014; 161794.
74. Ruiz de Garibay AP, Delgado D, Del Pozo-Rodríguez 85. Moon JS, Lee SH, Kim EJ, et al. Inhibition of Hepatitis
A, Solinís MÁ, Gascón AR. Multicomponent C Virus in Mice by a Small Interfering RNA
nanoparticles as nonviral vectors for the treatment of
Fabry disease by gene therapy. Drug Des Devel Ther Targeting a Highly Conserved Sequence in Viral
2012; 6: 303-310. IRES Pseudoknot. PLoS One 2016; 11:e0146710.
@Real Academia Nacional de Farmacia. Spain 86. Komar AA, Hatzoglou M. Exploring Internal
Ribosome Entry Sites as Therapeutic Targets. Front
Oncol 2015; 5: 233.
87. Lohmann V, Körner F, Koch J, Herian U, Theilmann
L, Bartenschlager R. Replication of subgenomic
hepatitis C virus RNAs in a hepatoma cell line.
Science 1999; 285:110-3.
423