Using a gene-therapy delivery system developed in the laboratory of Inder Verma at the Salk Institute for Biological Studies, an international research team successfully treated two boys from Spain suffering from adrenoleukodystrophy (ALD), the rare, inherited disease that was the focus of the Hollywood film "Lorenzo's Oil." The genetic disorder, in which the fatty insulation of nerve cells degenerates, leads to progressive brain damage and results in death. Two years after mixed gene-therapy and bone marrow transplants, the boys remain disease-free.
Verma pioneered the use of stripped-down versions of HIV, the virus that causes AIDS, to ferry intact versions of genes that are defective or missing to cells throughout the body.
Before this innovation, viruses used in gene delivery could only infect actively dividing cells, drastically limiting its utility. Verma's modified HIV virus is capable of infecting nondividing cells and delivering genes efficiently into a wide variety of cells.
The virus was produced by CellGenesys under a license agreement with the Salk Institute and the treatment administered by a French research team. The findings appear in the journal Science (http://bit.ly/2Yk9aC).
Monitoring oil spills
One day, swarms of inexpensive, miniature underwater robotic devices could predict where ocean currents will carry oil spills. Such "drifter" devices are being designed, built and deployed by researchers at Scripps Institution of Oceanography and the Department of Mechanical and Aerospace Engineering at UCSD.
Critical to the technology's success is the development of robotic control systems. This research area has been given a boost by a $1.5 million National Science Foundation grant awarded to engineers at the UCSD Jacobs School of Engineering (http://bit.ly/2v64N5).
Underwater ocean currents are poorly characterized despite their importance for understanding marine protected areas, algal blooms, oil spills and the path sewage takes after it is pumped into the ocean. Autonomous underwater robotic explorers will provide insights in fundamental oceanographic mechanisms that can be used to determine underwater ocean currents on the order of a few kilometers.
Potential to slow ALS?
In animal studies, researchers in the Department of Cellular and Molecular Medicine at UCSD have shown that a chemical cousin of the drug-activated protein C (APC) — currently used to treat sepsis — can dramatically slow the progression of a form of amyotrophic lateral sclerosis (ALS), better known as Lou Gehrig's disease. Not only were researchers able to significantly extend the lifespan of mice with an aggressive form of ALS, the compound reduced the pace of muscle wasting, thereby extending the length of time mice were able to function well despite showing some symptoms of the disease.
While more research must be done before being tested in people, researchers are encouraged that the work involves a compound that has already been proven to be safe and is currently given to patients for another condition. The team hopes to test the enzyme as an ALS treatment in patients within five years. The finding appears in the online edition of Journal of Clinical Investigation (http://bit.ly/20DtAv).
Lynne Friedmann is a science writer based in Solana Beach.