RNA interference (RNAi) is the sequence-specific gene silencing induced by double-stranded RNA (dsRNA). Being a highly specific and efficient knockdown technique, RNAi not only provides a powerful tool for functional genomics but also holds a promise for gene therapy. The key player in RNAi is small RNA (~22-nt) termed siRNA. Small RNAs are involved not only in RNAi but also in basic cellular processes, such as developmental control and heterochromatin formation. The interesting biology as well as the remarkable technical value has been drawing widespread attention to this exciting new field.
Animals ; Gene Therapy/*methods/trends ; *Genomics ; Human ; *RNA Interference

High-intensity focused ultrasound therapy is a novel, emerging, therapeutic modality that uses ultrasound waves, propagated through tissue media, as carriers of energy. This completely non-invasive technology has great potential for tumor ablation as well as hemostasis, thrombolysis and targeted drug/gene delivery. However, the application of this technology still has many drawbacks. It is expected that current obstacles to implementation will be resolved in the near future. In this review, we provide an overview of high-intensity focused ultrasound therapy from the basic physics to recent clinical studies with an interventional radiologist's perspective for the purpose of improving the general understanding of this cutting-edge technology as well as speculating on future developments.
Drug Delivery Systems ; Gene Targeting ; Hemostatic Techniques ; Humans ; Thrombolytic Therapy/methods ; *Ultrasonic Therapy/methods

The field of gene therapy has been increasingly studied in the last four decades, and its clinical application has become a reality in the last 15 years. Traditional Chinese medicine (TCM), an important component of complementary and alternative medicine, has evolved over thousands of years with its own unique system of theories, diagnostics and therapies. TCM is well-known for its various roles in preventing and treating infectious and chronic diseases, and its usage in other modern clinical practice. However, whether TCM can be applied alongside gene therapy is a topic that has not been systematically examined. Here we provide an overview of TCM theories in relation to gene therapy. We believe that TCM theories are congruent with some principles of gene therapy. TCM-derived drugs may also act as gene therapy vehicles, therapeutic genes, synergistic therapeutic treatments, and as co-administrated drugs to reduce side effects. We also discuss in this review some possible approaches to combine TCM and gene therapy.
Gene Expression ; drug effects ; Genes ; drug effects ; Genetic Therapy ; methods ; Humans ; Medicine, Chinese Traditional ; methods

Animals ; Gene Transfer Techniques ; Genetic Therapy ; methods ; Humans ; Kidney Diseases ; genetics ; therapy

Recently there have been increasing interests in ultrasound-mediated gene delivery techniques. This paper reviews the general concept of sonoporation, the effect of contrast agents, and the in vitro and in vivo applications of such techniques. Factors which contribute to the efficiency of gene delivery were also discussed. The technology is promising and has great potential in targeted drug delivery and gene delivery applications.
DNA ; administration & dosage ; Drug Delivery Systems ; Gene Transfer Techniques ; instrumentation ; Genetic Therapy ; Humans ; Microbubbles ; Transfection ; methods ; Ultrasonic Therapy ; methods ; Ultrasonics

Naturally occurring microRNAs (miRNAs), small non-coding RNAs of 19 to 24 nucleotides (nt), are encoded in the genomes of invertebrates, vertebrates, and plants. miRNAs act as regulators of gene expression during development and differentiation at the transcriptional, posttranscriptional, and/or translational levels, although most target genes are still elusive. Many miRNAs are conserved in sequence between distantly related organisms, suggesting that these molecules participate in essential processes. In this review, we present principles related to the basic and translational research that has emerged in the last decade, a period that can be truly considered the "miRNA revolution" in molecular oncology. These principles include the regulation mechanism of miRNA expression, functions of miRNAs in cancers, diagnostic values and therapeutic potentials of miRNAs. Furthermore, we present a compendium of information about the main miRNAs that have been identified in the last several years as playing important roles in cancers. Also, we orient the reader to several additional reviews that may provide a deeper understanding of this new and exciting field of research.
Animals ; Drug Delivery Systems ; methods ; Gene Expression Profiling ; Gene Expression Regulation, Neoplastic ; Genetic Therapy ; methods ; Humans ; MicroRNAs ; genetics ; metabolism ; physiology ; Mutation ; Neoplasms ; genetics ; therapy ; RNA, Long Noncoding ; metabolism

Gene therapy, as a therapeutic treatment for genetic or acquired diseases, is attracting much interest in the research community, leading to noteworthy developments over the past two decades. Although this field is still dominated by viral vectors, novel nonviral gene delivery systems based on nanoparticle technology have recently received an ever increasing attention in order to overcome the safety problems of viral vectors as well as the cytotoxicity of conventional nonviral vectors. This review presented the aspects of bionanotechnology involved in the gene delivery process and explored the recent developments and achievements of inorganic nanodelivery systems for gene transfection.
Gene Targeting ; methods ; trends ; Gene Transfer Techniques ; trends ; Genetic Therapy ; trends ; Genetic Vectors ; Humans ; Nanoparticles ; therapeutic use ; Nanotechnology ; methods ; Transfection ; methods

Animals ; CRISPR-Cas Systems ; Cystic Fibrosis ; genetics ; therapy ; Gene Editing ; ethics ; Genetic Therapy ; ethics ; methods ; Humans ; Mice ; Neoplasms ; genetics ; therapy

The concept of cell transplantation using tissue engineering techniques has provided numerous possibilities in the area of urologic tissue reconstruction. Tissue engineering applications in the genitourinary tract system have been investigated in almost every tissue in order to improve, restore and replace existing tissue function. Although most reconstructive efforts still remain in the experimental stage, several technologies have been transferred to the bedside with satisfactory outcome. In this article, we describe tissue engineering approaches attempted in the genitourinary system for reconstruction.
Animal ; Biomedical Engineering* ; Bladder ; Fetus ; Gene Therapy/methods ; Genitalia ; Human ; Kidney ; Ureter ; Urethra ; Urogenital System*

The efficacy of recombinant adeno-associated virus (rAAV) vector-mediated gene delivery to the gastrointestinal tract has been paid a considerable attention over the last 10 years, since our first report on the oral gene pill strategy in Nature Medicine, even though there are still several potential obstacles for this route to overcome in order to obtain efficient gene delivery. The preclinical results of oral rAAV gene medicine are summarized in this review, and special attention is paid on its pharmacokinetic and pharmacodynamic aspects with an emphasis on drug delivery, absorption, distribution and transduction. The rAAV based vectors have been shown promising results in human clinical trials with fewer safety concerns over other gene medicines. However, the underlying mechanisms and biopharmaceutical features of oral rAAV gene medicine remain to be explored extensively and intensively to develop this novel technology as a treatment for a wider range of diseases.
Administration, Oral ; Dependovirus ; genetics ; Drug Carriers ; Gene Transfer Techniques ; Genetic Therapy ; methods ; Genetic Vectors ; Humans