Treatment for Degenerative Diseases on the Anvil After Novel Mouse Model Study

A new mouse that may lead to new therapies for degenerative diseases has been developed by a team of researchers. Degenerative disease is a condition in which the function or structure of the affected tissues or organs will progressively deteriorate over time. The "Mos-iCsp3" mouse (for "mosaic inducible caspase 3 mouse") is engineered so that administration of a drug initiates destruction of cells in specifically designated tissues, explains Dr. Edge. Selection of the cell type to be killed is achieved by mating the Mos-iCsp3 mouse with a "Cre" mouse in which an enzyme called Cre recombinase is contained in selected tissues. Any cell that contains the enzyme begins to produce caspase 3.

This protein, a so-called "cell death" protein, is subsequently kept in an inactive form until the mouse is treated with a drug that activates caspase 3. Upon treatment with the drug the selected cells die. Several hundred Cre mice exist and cover a broad array of cell types.

"The mouse provides a way to study degenerative diseases and a model organism in which to develop therapies for those diseases," said Albert Edge, principal investigator at the Massachusetts Eye and Ear Infirmary's Eaton-Peabody Laboratory and associate professor at Harvard Medical School.

"We targeted inner ear hair cells, beta cells in the pancreas, and epidermal cells. We found that whereas the beta cells and skin cells showed some regeneration in response to cellular loss, inner ear hair cells were not capable of regeneration and thus hair cell death caused partial deafness. The mouse will expedite our efforts to replace inner ear cells lost in deafness," he added.

The study was published recently in the Journal of Clinical Investigation.

Source-ANI