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

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

DNA ; administration & dosage ; Gene Transfer Techniques ; trends ; Genetic Therapy ; methods ; Genetic Vectors ; Humans

RNA interference (RNAi) is a powerful endogenous process initiated by short double stranded RNAs, which results in sequence-specific posttranscriptional gene silencing. Because any protein that causes or contributes to a disease is susceptible to RNAi, the RNAi has high potential for therapeutic treatments. In a clinical setting, however, there are many obstacles to targeted delivery of small interfering RNA (siRNA) in vivo, specificity and stability of the RNAi reagents. In this review, we focus on recent progress in the development of efficient siRNA delivery vehicles to help the application of siRNA to in vivo therapy.
Drug Carriers ; Gene Transfer Techniques ; Humans ; Molecular Targeted Therapy ; methods ; trends ; RNA Interference ; RNA, Small Interfering ; administration & dosage ; genetics ; metabolism ; Transfection

Numerous studies and clinical trials have demonstrated the efficacy of recombinant adeno-associated virus gene delivery vectors. However, prior to expression, it is necessary to convert the single-stranded DNA genome into double-stranded DNA, which hinders the efficiency of these vectors. We can entirely circumvent this step through the use of self-complementary recombinant adeno-associated virus vector (scrAAV). ScrAAV packages an inverted repeat genome that can fold into double-stranded DNA without the requirement for DNA synthesis or base-pairing between multiple vector genomes. By using scrAAV, we could increase expression efficiency and reduce immune response caused by vectors themselves. Therefore, it is a promising vector for gene therapy. So far, it has been used in the treatment of hepatic diseases, central nervous system diseases, and eye diseases. It has also been used in the modifications of stem cells and as vectors for siRNA/miRNA and ribozymes. In this review, we focused on the preparation, expression and location of scrAAV both in vitro and in vivo. We mainly introduced the recent progress of scrAAV based therapy of Hemophilia B, in order to elucidate the potential and prospects of scrAAV in gene therapy.
Animals ; Base Sequence ; DNA ; genetics ; DNA, Complementary ; genetics ; DNA, Single-Stranded ; genetics ; Dependovirus ; genetics ; metabolism ; Gene Transfer Techniques ; Genetic Therapy ; methods ; trends ; Genetic Vectors ; genetics ; Hemophilia B ; therapy ; Humans ; Molecular Sequence Data