Retroviruses are naturally occurring viruses with an RNA genome. A retroviral vector is a modified infectious virus derived from murine or avian species that can be used to introduce nonviral DNAs into a target cell. There are two prominent features of retroviralmediated DNA transductio ...

Over the past decade, there has been spectacular growth in our understanding of the molecular genetics of eye development and ocular disease. Although this is primarily caused by developments in recombinant DNA technology, it is also caused in large part by advances in, and the spread of, transg ...

The RHO gene, which encodes rhodopsin, was the first gene in which mutations were found that were associated with an inherited retinal disease (1). RHO was examined by exon screening in a large population of patients with autosomal dominant retinitis pigmentosa (adRP), because a linkage study in ...

The ability to culture pluripotent cell lines, introduce into them targeted gene mutations, and then use these mutant cell lines to generate chimeric animals (1-4), has had a major impact on many fields; not least on ophthalmology and vision research (5-8). By generating animals lacking in the pro ...

Molecular biology has significantly contributed to our understanding of inherited retinal disease, in particular to that of monogenic disorders. The discovery of the responsible gene, the elucidation of its function, and the increasing understanding of the pathogenesis of inher ...

Retinoblastoma (Rb) is an intraocular tumor usually diagnosed in children under four years of age (1). The tumor rises when both alleles of the Rb tumor suppressor gene become inactivated in a retinal precursor cell during development (2,3). The first retinoblastoma cell line to be established ...

Molecular biology was first referred to as the study of the chemical and physical structure of biological macromolecules such as nucleic acids and proteins. Nucleic acids, deoxyribonucleic acid (DNA), and ribonucleic acid (RNA) are polymers that consist of nucleotides. Proteins are po ...

It is often desirable to localize a specific protein within a layer, cell, or subcellular organelle in the retina. A variety of immunocytochemical techniques are available to achieve this goal. These techniques all utilize an antibody that binds to the protein of interest and is detected by a label ...

Antisense DNA technology is a method to inhibit or downregulate the production of a target protein by using antisense DNA or RNA molecules. An antisense sequence is a DNA or RNA that is perfectly complementary to the target nucleotide sequence present in the cell. There are two possible mechanisms ...

RNA enzymes, or ribozymes, can be defined as RNA molecules that promote a variety of reactions involving RNA and DNA molecules. These include site-specific cleavage, ligation, polymerization, and phosphoryl exchange (1). The use of ribozymes for medical therapy was recognized soon after ...

Tissue factor (TF) plays a crucial role in the pathogenesis of thrombotic, vascular, and inflammatory disorders, and thus, the inhibition of this membrane protein provides a unique therapeutic approach for prophylaxis and/or treatment of various diseases. In recent years, tissue-fa ...

The role of cell adhesion molecules (CAM) and extracellular matrix (ECM) proteins in various pathological processes, including angiogenesis, thrombosis, apoptosis, cell migration, and proliferation, is well-documented. These processes can lead to both acute and chronic disea ...

Animal models have played a crucial role in the development of new antithrombotic drugs during the past few decades. Through the use of these animal models, the differentiation between the anticoagulant and antithrombotic effects was first recognized. Drugs that were unable to produce a p ...

Of more than 2 million deaths in the United States each year (from all causes), nearly 1 million are caused by cardiovascular disease (CVD). CVD claims more lives each year than the next seven leading causes combined. The prevalence of CVD in the United States is indicated by the statistics: 50 million peo ...

The synthetic pentasaccharide Arixtra or Fondaparinux is an indirect anti-Xa that effectively inhibits thrombin generation via its binding to anti-thrombin, the co-factor for Xa. A well-executed and designed clinical development of Fondaparinux in venous thrombosis prophyl ...

Preclinical and initial clinical experiences with the direct oral thrombin inhibitor ximelagatran demonstrated both a short and long-term potential utility for this agent in the prevention and treatment of various thromboembolic disorders associated with both arterial and v ...

The term pharmacogenetics, first introduced by Vogel in 1959 (1) is defined as the analysis of inherited factors that define an individual’s response to a drug, and generally refers to monogenetic variants that affect drug response. Pharmacogenetics refers to the monogenetic variants t ...

Thrombosis is still the leading cause of morbidity and mortality, and thus, effective antithrombotic strategies remain a critical therapeutic objective. The past decade has witnessed considerable progress in the development of newer anticoagulants, antiplatelets, and thro ...

Venous thromboembolism (VTE) is a term that includes both deep venous thrombosis (DVT) and pulmonary embolism (PE). Considerable progress has been made in the understanding of the risk factors for VTE. The clinical applications of molecular techniques have facilitated identificat ...

Despite recent major pharmacological and device advances, percutaneous coronary intervention (PCI) remains a costly procedure with significant periprocedural risk. Heparin has maintained the foundation of procedural anticoagulation, but heparin anticoagulation is ...