There are currently no cures available for the devastating and progressive neurodegenerative disease amyotrophic lateral sclerosis (ALS). Progress is being made, however, thanks to genetic studies that have led to the identification of a number of genes that seem to be implicated in the disease. One interesting line of enquiry relates to the genetic concomitants of inflammation and ALS where research has uncovered that genes involved in the inflammatory process in ALS are selectively activated. Read More
Adeno-associated virus (AAV)-based systems are ideally suited for delivering gene therapy. After the viral vector infects the cell, the DNA doesn’t integrate into the host genome, instead existing primarily in an episomal form, which allows for long-term expression in nonreplicating cells without DNA damage to the host cells and the risk of inducing malignancy. Read More