Cell-based treatments for huntington's disease

Abstract

In experimental rats, mice, and monkeys, transplantation of embryonic striatal cells into the striatum can repair the damage and alleviate the functional deficits caused by striatal lesions. Such strategies have been translated to striatal repair by cell transplantation in small numbers of patients with progressive genetic striatal degeneration in Huntington's disease. In spite of some encouraging preliminary data, the clinical results are to date neither as reliable nor as compelling as the broad extend of recovery observed in the animal models across motor, cognitive, and skill and habit learning domains. Strategies to achieve immediate and long-term improvements in the clinical applications include identifying and limiting the causes of complications, standardization and quality control of preparation and delivery, appropriate patient selection to match the cellular repair to specific profiles of cell loss and degeneration in individual patients and different neurodegenerative diseases, and improving the availability of alternative sources of donor cells and tissues.