Drug delivery, especially gene delivery to the lung, has been a challenge. Numerous gene delivery systems to the lung have been developed and tested in preclinical studies. However, only a few of them have been successfully tested in the clinic and shown promise. The reasons for failure to translate ...
Retroviral vectors have been widely used for research and clinical trials in gene therapy because of their high transduction efficiency. Retroviruses interact with target cells through their surface molecules (i.e., envelope proteins) and cellular receptors, which limit the susc ...
Bone tissue engineering has emerged as a promising strategy for the repair of critical-sized skeletal fractures. However, the clinical application of this approach has been limited by the availability of a robust mineralizing cell source. Non-osteogenic cells, such as skin fibroblas ...
RNA interference mediated by small interfering RNAs (siRNAs) is a powerful tool for evaluating gene function in vivo. In particular it should be able to provide tissue-specific and developmental stage-specific knockdown of target genes in physiological contexts. However, there are f ...
Skin is an attractive target for gene modification to treat skin diseases, wound healing, or even systemic disorders. Although retroviral transduction results in permanent genetic modification, differentiation and eventually loss of the transduced cells from the epidermis and in ...
Helper-dependent adenoviral vectors (HDAd) are deleted of all viral coding sequences and have demonstrated tremendous potential for gene therapy by providing long-term, high-level transgene expression in the absence of chronic toxicity. Thus, HDAd are superior to early generat ...
Retroviral vectors were the first viral vectors to enter clinical trials and continue to be attractive candidates for applications where integration of the transgene is required. While these vectors are versatile and are used widely in the research setting, large-scale production for ...
Adenoviruses (Ads) possess several features that make them attractive mammalian gene transfer vectors. They can efficiently infect a wide variety of quiescent and proliferating cell types from various species to direct high level viral gene expression, their 36 kb double-stranded D ...
Current and future demands of viral vectors for the development of successful pre-clinical and clinical studies in human gene therapy and possible commercialization of gene therapy products require well-established large-scale production processes. One of the most promising v ...
Virus vectors have been employed as gene transfer vehicles for various pre-clinical and clinical gene therapy applications. Replication-competent herpes simplex virus (HSV) vectors that replicate specifically in actively dividing glial tumor cells have been used in Phase I–II h ...
Advances in molecular biology and genetics have provided an abundance of descriptive biological data. Ascribing physiologic roles to these data is an important task to translate this information into improvements in human health. Non-viral transgene delivery of a transgene is a prom ...
Inefficient delivery of antisense oligonucleotides (AO) to target cell nuclei remains as the foremost limitation to their usefulness. Copolymers of cationic poly(ethylene imine) (PEI) and polyethylene glycol (PEG) are extremely well-studied compounds that markedly improve ...
Recent advances in genomics and proteomics have led to the evolution of DNA-based therapeutics and the use of gene therapy for treatment of a wide range of human diseases. However, poor cellular uptake and rapid in vivo degradation of DNA-based therapeutics are the major drawbacks of gene therap ...
Although the majority of microarray studies have been directed toward RNA expression profiling (functional genomics) and increasingly toward proteomics, a steady increase in the use of microarrays as platforms for DNA genotyping has occurred over the past 5 yr. Multiple array-based c ...
A large number of human genetic diseases, bacterial drug resistances, and single-nucleotide polymorphisms are caused by gene mutations. Rapid and high-throughput mutation detection methods are urgently demanded. A protein chip method for detection of single-base mismatches a ...
A quantitative screening method was developed to evaluate the quality of cDNA libraries constructed by suppression subtraction hybridization (SSH) or other enrichment techniques. The SSH technique was adapted to facilitate screening of the resultant library on a small number of gl ...
Automated target preparation for gene expression analysis eliminates the time-consuming and labor-intensive manual process, which is error prone and diverts scientists from value added activities. Target preparation methods were developed, on the fully integrated ArrayP ...
Tumor-stroma interactions are of great importance not only for the development and progression of primary prostate carcinoma but probably also for the establishment of metastasis. Fibroblasts are an important stromal cell type encountered by metastatic tumor cells at different s ...
Small molecules, such as metabolites and hormones, interact with proteins to regulate numerous biological pathways, which are often aberrant in disease. Small molecule drugs have been successfully exploited to specifically perturb such processes and thereby, decrease and even e ...
Small molecule detection poses special problems during analysis whether hormones in a clinical setting or pesticides from environmental monitoring. Traditional analysis involves procedures like high-pressure liquid chromatography, gas chromatography, or mass spect ...