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GEN’s editor in chief, John Sterling, interviews life science academic and biotech industry leaders on important research, technology, and trends. These podcasts will keep you informed with all the important details you need.

GENE THERAPY RESTORES VISION TO BLIND MICE

10/30/2008

- Interview with Richard Masland, PhD, Director, Cellular Neurobiology Laboratory, Department of Neurosurgery, Massachusetts General Hospital

Massachusetts General Hospital researchers have used gene therapy to restore useful vision to mice with degeneration of the light-sensing retinal rods and cones, a common cause of human blindness. Their report, which appeared in the Oct. 14 issue of the Proceedings of the National Academy of Sciences, describes the effects of broadly expressing a light-sensitive protein in other neuronal cells found throughout the retina.


During this week's podcast, Dr. Richard Masland discusses the design of the study and what his team was actually able to demonstrate. He explains why melanopsin, which is usually produced in a subset of cells that are involved with establishing circadian rhythms, was chosen to do an experiment on vision. Dr. Masland also talks about the resultant levels of melanopsin expression in ganglion cells and how his research group was able to determine that the previously blind mice were now able to "see." He specifies the type of gene therapy method used in the research and offers his opinion on how this proof-of-principle study might be used to repair blindness in people with conditions like retinitis pigmentosa and macular degeneration.
Dr. Masland is the Charles A. Pappas Professor of Neuroscience at Harvard Medical School and Neurophysiologist in Neurosurgery at Massachusetts General Hospital, Boston. He received his A.B. degree from Harvard College and his Ph.D. degree from McGill University. His postdoctoral work was done at Stanford and Harvard Medical Schools. Among his awards is Harvard University’s Hoopes Prize, for excellence in teaching. Research in this laboratory concerns local cellular interactions within the retina. Mammalian retinas contain a surprising diversity of cell types. Amacrine cells, upon which we have especially concentrated, exist in atleast 20 different morphological subclasses. By fluorescent staining many of these classes can be visualized by distinct, reproducible populations in histological material or intact retinas in vitro during electrophysiological recording. The broad questions under investigation are: (1) why this diversity exists, i.e., what functions the many cells carry out; (2) how orderly structural relations among the cells are created and maintained; and (3) how inner retinal cell's local dendritic networks function.

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Genetic Engineering & Biotechnology News (GEN) has retained its position as the most widely read biotechnology publication around the globe since its launch in 1981. Published 21 times a year and with additional exclusive editorial content online, GEN's unique news and technology focus includes the entire bioproduct life cycle from early-stage R&D, to applied research including omics, biomarkers, as well as diagnostics, to bioprocessing and commercialization.

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