Abstract: The application of RNA interference-based gene silencing technologies has the potential to treat a variety of illness. Preclinical studies and some early clinical trials have already demonstrated the utility of small interfering RNAs (siRNAs) as a potential novel therapy for the treatment of cancer, viral infections, as well as a wide range of additional diseases. To be effective, an siRNA must be taken up by specific cells, enter the cytoplasm, and be loaded onto the Argonaute protein, the catalytic core of the RNA induced silencing complex (RISC) to direct the cleavage of the homologous transcripts. To meet this need, a variety of novel siRNA delivery strategies have been developed. As our understanding of the molecular mechanisms underlying the RNAi pathway has increased so has the ability to rationally design effective silencing and delivery strategies. This review will examine the latest advances in non-viral delivery of siRNA, with special reference to targeted siRNA delivery to specific target tissues and cell types in vivo in preclinical animal models. ... Read more

Abstract: Diabetic retinopathy, the most common long-term complication of diabetes mellitus, remains one of the leading causes of blindness worldwide. Strict metabolic control, tight blood pressure control, laser photocoagulation, and vitrectomy remain the standard care for diabetic retinopathy. Focal/grid photocoagulation is a better treatment than intravitreal triamcinolone acetonide in eyes with diabetic macular edema and should be considered as the first-line therapeutic option. The current evidence suggests that intravitreal triamcinolone acetonide or anti-vascular endothelial growth factor agents result in a temporary improvement of visual acuity and a short-term reduction of central macular thickness in patients with refractory diabetic macular edema and are effective adjunctive treatment to laser photocoagulation or vitrectomy. However, triamcinolone is associated with risks of elevated intraocular pressure and cataract. Vitrectomy with removal of the posterior hyaloid without internal limiting membrane peeling seems to be effective in eyes with persistent diffuse diabetic macular edema, particularly in eyes with associated vitreomacular traction. Emerging therapies include islet cell transplantation, fenofibrate, ruboxistaurin, pharmacologic vitreolysis, rennin-angiotensin system blockers, and peroxisome proliferator-activated receptor gamma agonists. ... Read more

Abstract: Identification of pathogenetically relevant autoantigen targets is a major goal in the study of autoimmune diseases. Indeed it may allow the development of new specific diagnostic tools and facilitate the understanding of the pathogenesis of a disease in order to individualize possible novel treatments. The random peptide library is a molecular biology method that consists of the display of random peptides on live microorganisms. The screening of the library with pooled immunoglobulins obtained from patients affected by an autoimmune disease may lead to the identification of novel autoantigens and of antibodies that are able to bind such antigens with high affinity. Testing patients' sera for the presence of these antibodies may be helpful in the diagnosis of the disease. Moreover such antibodies can be investigated for their functional activity and therefore provide new insights into the pathogenesis of the disease. ... Read more

Abstract: According to the World Health Organization, approximately 220 million people worldwide have type 2 diabetes mellitus. Patients with type 2 diabetes not only have a chronic disease to cope with, they are also at increased risk for coronary heart disease, peripheral vascular disease, retinopathy, nephropathy, and neuropathy. The exact causes of type 2 diabetes are still not clear. Since the 17^th century, it has been suggested that emotional stress plays a role in the etiology of type 2 diabetes mellitus. So far, review studies have mainly focused on depression as a risk factor for the development of type 2 diabetes mellitus. Yet, chronic emotional stress is an established risk factor for the development of depression. The present review provides an overview of mainly prospective epidemiological studies that have investigated the associations between different forms of emotional stress and the development of type 2 diabetes mellitus. Results of longitudinal studies suggest that not only depression but also general emotional stress and anxiety, sleeping problems, anger, and hostility are associated with an increased risk for the development of type 2 diabetes. Conflicting results were found regarding childhood neglect, life events, and work stress. It is important to emphasize that publication-bias may have occurred, resulting from "fishing-expeditions," where authors search their data for significant associations. Publication bias may also be caused by the tendency of reviewers and Editors to reject manuscripts with negative results for publication. It is therefore essential that research groups, who aim to conduct a new epidemiological cohort study, prospectively describe and publish the design of their study. Future research should focus on identifying mechanisms linking different forms of stress and incident type 2 diabetes. ... Read more

Abstract: Human gene therapy has made substantial progress since the initiation of the first clinical trials 20 years ago. Here, we summarized important applications of gene transfer protocols in the treatment of various human diseases using different viral vectors. Recent successful trials on the treatment of ocular diseases and inherited immune deficiencies are particularly encouraging and have raised hopes that human gene therapy as a standard treatment option will finally become a reality. While immune responses and insertional mutagenesis pose obstacles for this novel form of molecular medicine, continuous progress suggests that a wider range of diseases can be treated with gene therapy in the future. ... Read more

Abstract: Most uveitics enjoy good vision despite potentially sight-threatening complications including cataract development. In those patients who develop cataracts, successful surgery stems from educated patient selection, careful surgical technique, and aggressive preoperative and postoperative control of inflammation. While commonly accepted in the adult patient population, recent investigations reflect the increased tolerance for primary intraocular lens placement in the pediatric cohort. The role of absolute control of inflammation continues with greater focus on immunomodulatory therapies. However, these agents bear their own side effect and complication profiles. Cataract extraction with intraocular lens implantation in the setting of meticulous control of inflammation can optimize visual outcome in adults and children with uveitis. ... Read more

Abstract: Age-related Macular Degeneration (AMD) is the major cause of vision loss after age 50 in the United States. Although an important association of the complement cascade with AMD has recently been made, we still do not understand the pathogenesis of the disease. AMD is characterized by loss of the retinal pigment epithelium (RPE) within the macula (i.e., the center of the retina), and in turn, loss of the overlying foveal photoreceptors. Since RPE and photoreceptors can both be generated from stem cells using cell culture, there is hope for future cell replacement therapy. But, aging changes in Bruch's membrane, the scaffold on which the RPE are anchored, may complicate such therapy, and require surgical repair of Bruch's membrane to provide a suitable environment for cell survival and function. We have referred to such a multipronged approach of surgical reconstruction of the macular architecture in conjunction with cell transplantation as Maculoplasty. ... Read more

Abstract: Gene therapy for inherited retinal degeneration has made major advances toward the ultimate goal of reversing blindness in human patients. With significant advances in recombinant viral vector design, safety and efficacy profiles have greatly improved. Although these recent advances have been applied to many different retinal diseases, one retinal degenerative disease, Leber congenital amaurosis, appears to have the greatest potential for reversing blindness. In pre-clinical animal studies, gene therapy for Leber congenital amaurosis has demonstrated visual recovery. Recently, in landmark clinical trials, preliminary results have indicated safety and efficacy for the use of gene therapy in Leber congenital amaurosis, thus laying the foundation for continued use of gene therapy in other forms of inherited blinding disease. ... Read more

Abstract: Can one conveniently treat an autoimmune disease such as rheumatoid arthritis by ingesting the very protein that triggers the disease in the first place? The answer is maybe. Active studies are performed on both animals and humans and met with both success and failure. ... Read more

After a deep freeze, biotech Initial Public Offerings (IPOs) emerged strongly in the second half of last year and have accelerated during the first four months of this year. A generally healthy capital market and an outstanding performance by some biotech companies has provided a friendly backdrop and propelled this acceleration.

In just over four months, 17 companies — from biotech and pharmaceuticals to surgical centers and medical equipment — have gone public, according to Renaissance Capital and its website. That accounts for 38% of the IPOs so far this year, and there are many more such companies looking ... Read more