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Liposomes - Hollow Nanospheres for Gene Therapy - Medical / Health Care
The use of genetic material as therapeutic compounds to adjust dysfunctional cell behaviors is a revolutionary approach that has gained more attention in recent years in the medical field. Up or downregulation of genetic expression leads to an increased or decreased production of proteins respectively. The main challenge that gene therapy faces with naked genetic material is the low transfection rates and thus, therapeutic efficacy. Their physical properties as big sizes and polarity, difficult the permeation thought cell and nucleus membranes as well as are susceptible to enzymatic degradation.
Liposomes and other lipid based nanovesicles are able to encapsulate DNA, RNA, siRNA, antisense oligonucleotides, aptamers, DNAzymes, plasmid DNA protecting them along the way until the cell nucleus. The main advantage over the traditional viral vectors is that there are less safety concerns regarding their use. This concept has already been applied to multiple diseases as cystic fibrosis, anemia, immune system deficiencies, viral transmissible diseases, neurological diseases, and hemophilia.