Development and characterization of thermoreversible topical delivery system for delivery of siRNA in ocular gene therapy

• Grant number: 10/14457-4
• Support Opportunities: Scholarships in Brazil - Master
• Start date: March 01, 2011
• End date: August 31, 2012
• Field of knowledge: Health Sciences - Pharmacy - Pharmaceutical Technology
• Principal Investigator: Maria Vitória Lopes Badra Bentley
• Grantee: Thais Fedatto Abelha
• Host Institution: Faculdade de Ciências Farmacêuticas de Ribeirão Preto (FCFRP). Universidade de São Paulo (USP). Ribeirão Preto , SP, Brazil

Abstract

The ocular gene therapy has brought new perspective to the treatment of many diseases. The use of siRNA allows the interruption of production of harmful proteins which cause metabolic disorders and several diseases. The study and development of a formulation to deliver siRNA accordingly to cornea cells, has the potential to treat several diseases that impair vision. There are anatomical and physiological barriers that must be overcomed and new strategies to transpose these obstacles need to be explored. Therefore, the use of raw materials such as polymer thermoreversible Poloxamer 407 in combination with Carbopol and cationic polymer poly(ethyleneimine) can enhance the application, penetration and uptake of siRNA in the cornea. Thus, the purpose of this study is to obtain and characterize a in situ gel formulation and to do in vitro release studies of siRNA. The investment in the research of topical ocular delivery systems for siRNA is warranted because this molecule has the potential to treat incurable diseases, so far, and it's therapeutic application has already being tested in clinical practice, moreover, there are few studies of in vitro release of siRNA in the cornea.

Academic Publications

(References retrieved automatically from State of São Paulo Research Institutions)

ABELHA, Thais Fedatto. Development and characterization of topical siRNA drug delivery systems for corneal gene therapy: thermoreversible gel and liquid crystalline nanodispersions. 2012. Master's Dissertation - Universidade de São Paulo (USP). Faculdade de Ciências Farmacêuticas de Ribeirão Preto (PCARP/BC) Ribeirão Preto.