Research Grant Award

Rebecca L. Landsberg, PhD

Harvard Medical School
Genetic programs regulation cell fate decisions in the rhombic lip, a birth site of neurons affected in ataxias

Derangements in balance and coordination, the behavioral hallmarks of ataxia, result largely from the selective vulnerability of neurons comprising three major brain regions: the cerebellum, pons, and inferior olives. In general, cell type- or brain region-specific vulnerabilities that ultimately lead to disease, such as ataxia, typically reflect specific properties unique to those cells, properties typically acquired by those cells during their, development and differentiation. Thus, a powerful approach for attacking the molecular basis of ataxia, and therefore for identifying potential novel genetic diagnostics and therapeutic targets, is to first identify molecules critical to the development and differentiation of neurons comprising the cerebellum, pons, and inferior olives. Towards this goal, we have proposed novel molecular, genomic, and embryological studies directed at elucidating molecules critical for the development and differentiation of neurons in the pons, inferior olives, and associated neurons that collectively form what is called the pre-cerebellar system.