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In vitro and in vivo evaluation of liquid-crystalline nandispersions to deliver siRNA-TRP-1 as an alternative for the topical treatment of vitiligo

Grant number: 13/06559-0
Support Opportunities:Scholarships in Brazil - Master
Effective date (Start): November 01, 2013
Effective date (End): March 31, 2015
Field of knowledge:Health Sciences - Pharmacy - Pharmaceutical Technology
Principal Investigator:Maria Vitória Lopes Badra Bentley
Grantee:Larissa Bueno Tofani
Host Institution: Faculdade de Ciências Farmacêuticas de Ribeirão Preto (FCFRP). Universidade de São Paulo (USP). Ribeirão Preto , SP, Brazil


Gene therapy with RNA interference (RNAi) as an alternative for the treatment of various diseases has been increasingly studied and understood by many research groups around the world. Interference with RNAi is a process closely related to the gene expression; it can be used not only for functional genomics research but also as a valuable tool for the treatment of diseases occurring mutation or overexpression gene. Several studies have shown promising results with the use of RNAi for treating skin diseases involving overexpression or deregulation of genes. However, the challenge is in the development of a safe and effective delivery system that can enable the use of siRNAs for treatment of diseases. Vitiligo is an autoimmune disease of the skin characterized by progressive loss of cells responsible for the synthesis of the pigment melanin (melanocytes), resulting in melanocyte destruction by autoantibodies from the patient. A therapeutic target for gene therapy silencing can be the TRP-1 (Tyrosinase-related protein 1) since it is expressed in the membrane of melanocytes as a target for destruction, a condition shown in vitiligo. In this aspect, this project aims to evaluate in vivo and in vitro nanodispersões liquid-crystalline with siRNAi-TRP-1 specific as a carrier for the topical treatment of vitiligo.

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Scientific publications
(References retrieved automatically from Web of Science and SciELO through information on FAPESP grants and their corresponding numbers as mentioned in the publications by the authors)
TOFANI, LARISSA BUENO; DEPIERI, LIVIA VIEIRA; CAMPOS, PATRICIA MAZUREKI; RIUL, THALITA BACHELLI; ANTONIETTO, KAMILLA SWIECH; DE ABREU FANTINI, MARCIA CARVALHO; LOPES BADRA BENTLEY, MARIA VITORIA. In Vitro TyRP-1 Knockdown Based on siRNA Carried by Liquid Crystalline Nanodispersions: an Alternative Approach for Topical Treatment of Vitiligo. PHARMACEUTICAL RESEARCH, v. 35, n. 5, . (14/50928-2, 13/06559-0)
Academic Publications
(References retrieved automatically from State of São Paulo Research Institutions)
TOFANI, Larissa Bueno. Evaluation in vitro and in vivo of liquid crystalline nanodispersions for delivery of siRNA-TyRP-1 for topical treatment of vitiligo. 2015. Master's Dissertation - Universidade de São Paulo (USP). Faculdade de Ciências Farmacêuticas de Ribeirão Preto (PCARP/BC) Ribeirão Preto.

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