Update on the Genetic Research at the University of Massachusetts
By Robert H. Brown, Jr., D.Phil., M.D.
The UMass Team has made progress in three activities in the laboratory. First, we have continued work on our program to find ways to turn off the gene that makes the mutant HSAN protein (serine palmitoyltransferase or SPT). This has involved developing a set of reagents that can interact with the working template of the gene, which is a string of RNA molecules copied from the DNA in the gene itself. Guided by Dr. Anastasia Khvorova and aided by a graduate student, Havisha Karnam, we have made starting tools – short strings of DNA molecules (known as oligonucleotides, or just “oligos”) that bind to the target RNA from the SPT gene. We have generated two types of the oligos and are very encouraged because they both successfully turn off the SPT gene in cells in a Petri dish.
In our second, parallel effort, we have also been studying the degree to which these oligos permeate the target sites in the nervous system (the dorsal root ganglia and spinal cord). We have addressed this in mice, using infusions into the spinal fluid. One of the oligos shows excellent penetration into the target tissues.