A number of diseases can result from abnormal gene expression. One of the approaches for treating such diseases is gene therapy to inhibit expression of a particular gene in a specific cell population by RNA interference. Use of efficient delivery vehicles increases the safety and success of gene therapy. Here we report the development of functionalized biocompatible fluorescent nanoparticles f...

RNAi-mediated gene therapy is gradually becoming a conventional approach for the treatment of various diseases. One major challenges clinical translation RNAi to efficiently deliver siRNA into targeted tissues and cells. Herein, in article number 20200026 Yiyun Cheng, Jia Lv their co-workers have reviewed recent advances design functional polymers overcome multiple extracellular intracellular b...

The main obstacle in siRNA therapy is RNA delivery to the cytoplasm, where it can guide sequence-specific mRNA degradation. Attempts to develop effective nonviral vectors for in vivo delivery of nucleic acids through a systemic route are hampered by difficulties of combining high extracellular stability with ready availability of the nucleic acids following entry into cells. Other challenges wi...

application of therapeutic gene transfer in the treatment of genetic diseases is a notable progress but there are some disadvantages and limitations in it. the process of overcoming these barriers is a drastic change in gene delivery. recently, calcium phosphate nanoparticles alone, or in combination with viral and nonviral vectors, were found to have a positive effect on gene transfer especial...

The use of nanoparticles as anticancer cargo systems for drug delivery is a promising modality, they avoid the known toxicity drugs on healthy cells by multiple to target cells. Here, adsorption behavior cisplatin molecules in two different inorganic materials, silica and metallic gold, investigated mathematically. 6–12 Lennard-Jones potential, together with continuum approximation, adapted cal...

Gene targeting protocols for mammalian cells remain inefficient and labor intensive. Here we describe FASTarget, a rapid, fluorescent cell sorting based strategy to isolate rare gene targeting events in human somatic cells. A fluorescent protein is used as a means for direct selection of targeted clones obviating the need for selection and outgrowth of drug resistant clones. Importantly, the us...

Bmi1 gene overexpression is found in various human tumors and has been shown as a potential target for gene treatment. However, siRNA-based treatments targeting Bmi1 gene have been restricted to limited delivery, low bioavailability and hence relatively reduced efficacy. To overcome these barriers, we developed a folate receptor targeted co-delivery system folate-doxorubicin/Bmi1 siRNA liposome...

Gene therapy, to delivery of genetic material to a patient for therapeutic benefit, has significant promise for translating basic knowledge of disease mechanism into biomedical treatments. The clinical development of the field has been slowed, however, by the need for improvements in the properties and capabilities of gene delivery vehicles. Vehicles based on viruses offer the potential for eff...

Small interfering RNA (siRNA) has proved to be a powerful tool for target-specific gene silencing via RNA interference (RNAi). Its ability to control targeted gene expression gives new hope to gene therapy as a treatment for cancers and genetic diseases. However, siRNA shows poor pharmacological properties, such as low serum stability, off-targeting, and innate immune responses, which present a...