Translational siRNA therapeutics using liposomal carriers: prospectschallenges
Autor: | Uma Maheswari Krishnan, Dhiraj Bhavsar, Krishnakumar Subramanian, Swaminathan Sethuraman |
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Rok vydání: | 2012 |
Předmět: |
Small interfering RNA
Liposome Chitosan Gene Transfer Techniques High cell Computational biology Genetic Therapy Pharmacology Biology Lipids Molecular therapy Cations Drug Discovery Liposomes Genetics Molecular Medicine Gene silencing Humans Delivery system Gene Silencing RNA Small Interfering Molecular Biology Genetics (clinical) |
Zdroj: | Current gene therapy. 12(4) |
ISSN: | 1875-5631 |
Popis: | Gene silencing has emerged as a promising strategy for molecular therapy of various malignant, viral, hereditary and inflammatory disorders. However, its translation from lab to clinic is yet to gain momentum due to the numerous problems that plague its development. A multi–functional siRNA delivery system with desired properties such as enhanced immune compatibility, target specificity, high cell uptake and excellent silencing efficiency is required to understand the challenges involved in the selection and modification of small interfering RNA (siRNA), factors influencing the complexation process and the response of the biological system to the formulation. Liposomes have been used as delivery systems due to its versatility in handling different types of drugs, tunable size, charge and surface functionalities that improve its effectiveness in vivo. This review highlights the challenges involved in gene silencing and describes the progression of liposomal systems used in gene silencing. The rationale in introducing chemical modifications in siRNA, synthesizing designer cationic lipids and evolution of hybrid liposomal systems has been elaborated, emphasizing their merits and short–comings. Finally, a description of the current state of clinical trials involving liposomal formulations has been included to provide an unbiased perspective of the future of liposomal systems and gene silencing tools as therapeutic tools. |
Databáze: | OpenAIRE |
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