Genetic diseases seriously threaten human health and have always been one of the refractory conditions facing humanity. Currently, gene therapy drugs such as siRNA, shRNA, antisense oligonucleotide, CRISPR/Cas9 system, plasmid DNA and miRNA have shown great potential in biomedical applications. To avoid the degradation of gene therapy drugs in the body and effectively deliver them to target tissues, cells and organelles, the development of excellent drug delivery vehicles is of utmost importance. Viral vectors are the most widely used delivery vehicles for gene therapy
in vivo
and
in vitro
due to their high transfection efficiency and stable transgene expression. With the development of nanotechnology, novel nanocarriers are gradually replacing viral vectors, emerging superior performance. This review mainly illuminates the current widely used gene therapy drugs, summarizes the viral vectors and non-viral vectors that deliver gene therapy drugs, and sums up the application of gene therapy to treat genetic diseases. Additionally, the challenges and opportunities of the field are discussed from the perspective of developing an effective nano-delivery system.
Reactive oxygen species (ROS)‐mediated apoptosis is considered a crucial therapeutic mechanisms for artesunate (AS). As an Fe(II)‐dependent drug, the anticancer effect of AS is often limited due to insufficient Fe(II) concentration in targeted cells. To overcome this problem, a recombinant apoferritin nanocarrier containing ferriferous oxide (M‐HFn) is constructed to produce auxiliary exogenous Fe(II) when delivering AS to cancer cells. Here, the newly fabricated AS‐loaded M‐HFn nanoparticles (M‐HFn@AS NPs) can significantly improve the tumor‐specific targeting and intracellular uptake efficiency of AS in human cervical carcinoma cells. After being captured in the acidic cavity of endosomes, M‐HFn@AS NPs can simultaneously release Fe(II) and allow AS to activate satisfactory ROS‐mediated apoptosis. Furthermore, in vivo studies demonstrate that M‐HFn@AS NPs can selectively accumulate in tumors to efficiently inhibit tumor growth. Thus, M‐HFn@AS NPs are a promising system to enhance the therapeutic effect of Fe(II)‐dependent drugs.
Protean mesoporous silica nanoparticles are propitious candidates over decades for nanoscale drug delivery systems due to their unique characteristics, including changeable pore size, mesoporosity, high drug loading capacity and biodegradability.
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