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JOSÉ	
  LUIS	
  VILA	
  
	
  

parámetros	
  farmacocinéticos,	
  farmacodinámicos	
  y	
  de	
  seguridad	
  están	
  permitiendo	
  
desarrollar	
  terapéuticas	
  que	
  pasen	
  a	
  fase	
  clínica	
  para	
  cáncer	
  y	
  otras	
  patologías.	
  

        	
  

8. REFERENCIAS

     1. Fire, et al. (1998) Potent and specific genetic interference by double-stranded RNA in
           Caenorhabditis elegans. Nature. 391: 806-811.

     2. A Dose Escalation Trial of an Intravitreal Injection of Sirna-027 in Patients With Subfoveal
             Choroidal Neovascularization (CNV) Secondary to Age-Related Macular Degeneration
             (AMD). ClinicalTrials.gov.

     3. A Study Using Intravitreal Injections of a Small Interfering RNA in Patients With Age-Related
             Macular Degeneration. ClinicalTrials.gov.

     4. Safety and Efficacy Study of Small Interfering RNA Molecule (Cand5) to Treat Diabetic
             Macular Edema. ClinicalTrials.gov.

     5. Safety & Efficacy Study Evaluating the Combination of Bevasiranib & Lucentis Therapy in Wet
             AMD (CARBON) ClínicalTrials.gov.

     6. Safety and efficacy study of small interfering ribonucleic acid (RNA) molecule (Cand5) to treat
             wet age-related macular degeneration.ClinicalTrials.gov.

     7. Durcan, N., Murphy, C. & Cryan, A. (2008) Inhalable siRNA: potent as a therapeutic agent in
             the lugs. Molecular Pharm. 5(4): 559-566.

     8. Phase II Clinical Trial of ALN-RSV01 demonstrates statistically significant anti-viral efficacy.
             Estudio GEMINI: www.alnylam.com.

     9. Immunotherapy of melanoma with tumor antigen RNA and small inhibitory RNA transfected
             autologous dendritic cells. ClinicalTrial.gov.

     10. Use of SV40 vectors to treat chronic myeloid leukemia (CML): ClinicalTrials.gov.
     11. Safety Study of CALAA-01 to Treat Solid Tumor Cancers: ClinicalTrials.gov.
     12. Davies, M. E. (2009) The first targeted delivery of siRNA in humans via a self-assembling,

             cyclodextrin polymer-based nanoparticle: from concept to clinic. Mol. Pharm. 6(3): 659-668.
     13. Study with Atu027 in patients with advanced solid cancer. ClinicalTrials.gov.
     14. The Role of Aromatic Hydrocarbon Receptor in the Tumorigenesis of Neuroblastoma and Its

             Relationship With MYCN Expression: ClinicalTrials.gov.
     15. Single Dose Study of siG12D LODER (Local Drug EluteR) in Patients With Operable

             Adenocarcinoma of the Pancreas Phase I - Multiple Dose Study of siG12D LODER (Local
             Drug EluteR) in Patients With Adenocarcinoma of the Pancreas. ClinicalTrials. gov.
     16. The Role of Glycosyltransferases in the Oncogenesis of Neuroblastoma. ClinicalTrials.gov.
     17. Study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD)
             of liposomal siRNA in subjects with high cholesterol. ClinicalTrials.gov.
     18. A dose escalation and safety study of I5NP to prevent AKI in patients undergoing major
             cardiovascular surgery (QRK.002): ClinicalTrials.gov.
     19. I5NP for prophylaxis of delayed graft function in kidney transplantation: ClinicalTrials.gov.
     20. Leachman, S. A., Hickerson, R. P., Schwartz, M. E., Bullough, E. E., Hutcherson, S. L.,
             Boucher, K. M., Hansen, C. D., Eliason, M. J., Srivatsa, G. S., Kornbrust, D. J., Smith, F. J.,
             McLean, W. I., Milstone, L. M. & Kaspar, R. L. (2010) First-in-human Mutation-targeted
             siRNA Phase Ib Trial of an Inherited Skin Disorder. Mol. Ther. 18(2): 442-446.
     21. NUC B1000: http://www.nucleonicsinc.com/clinical/index.html
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     23. SYL040012. Tolerance and Effect on Intraocular Pressure in Subjects With Intraocular Pressure
             (IOP) >= 21 mm Hg. ClincalTrials gov.
     24. Ray, R. B. & Kanda, T. (2009) Inhibition of HCV replication by small interfering RNA.
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     25. Kim, S. I., Shin, D., Lee, H., Ahn, B. Y., Yoon, Y. & Kim, M. (2009) Targeted delivery of
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     26. Caldwell, D. J. & Evans J. D. (2008) Developing clinical role of a CCR5 coreceptor antagonist
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