Current Gene Therapy - Volume 7, Issue 2, 2007

The type I glycogen storage diseases (GSD-I) are a group of related diseases caused by a deficiency in the glucose- 6-phosphatase-α (G6Pase-α ) system, a key enzyme complex that is essential for the maintenance of blood glucose homeostasis between meals. The complex consists of a glucose-6-phosphate transporter (G6PT) that translocates glucose- 6-phosphate from the cytoplasm into the lumen of the endoplasmic r Read More

Heme oxygenase-1 (HO-1) is regarded as a sensitive and reliable indicator of cellular oxidative stress. Studies on carbon monoxide (CO) and bilirubin, two of the three (iron is the third) end products of heme degradation have improved the understanding of the protective role of HO against oxidative injury. CO is a vasoactive molecule and bilirubin is an antioxidant, and an increase in their production through an increa Read More

Parkinson's disease is the second most common neurodegenerative disease. It is charaterized by a progressive loss of dopamine (DA) producing neurons in the midbrain, which result in a decline of DA innervations present in the forebrain, in particular, the striatum. The disease leads to appearance of motor symptoms involving akinesia/bradykinesia, gait disturbances, postural imbalance and tremor. Oral administration Read More

Extracellular signaling molecules have been implicated in the progression of Retinal Degeneration (RD). Gene regulatory events linked to the maintenance of retinal structure and function incorporate signaling cascades that may serve as therapeutic targets for some forms of blindness. This review shall focus on the evidence for non-cell-autonomous mechanisms that affect the pattern of degeneration seen in retinal dyst Read More

The endothelium has an important regulatory role in the maintenance of vascular homeostasis, vascular tone, blood flow, and in preserving a non-thrombogenic blood-tissue interface. Injury to the vascular wall with subsequent endothelial dysfunction alters these important regulatory functions leading to a state of abnormal endothelial function. In this paper, we review the pathophysiology of endothelial dysfunction and how this Read More