Register

Research Biology

ALS TDI: Gene Therapy The ALS Therapy Development Institute's scientists aim to discover and prioritize the most promising strategies to slow, stop, and reverse amyotrophic lateral sclerosis (ALS) disease progression. The Research Biology team dissects ALS pathology using broad genome-wide profiling technologies (trillions of data-points) integrated with high resolution studies of single proteins within single cells.

The "-omics"

"-omics" is a suffix usually applied to areas of large scale biological study. The ALS Therapy Development Institute uses genomics and proteomics to profile, or characterize, ALS tissues. The identification of differences in DNA sequences, RNA quantities, and the quantities and shapes of proteins in ALS related tissues when compared to healthy tissues leads to clearer understanding of the underlying biology of ALS and to the identification of promising ways to attack the disease. In 2007, ALS TDI completed the first phase of a comprehensive gene-expression (transcriptome) database for ALS with the profiling of the SOD1 mouse model. The second phase, profiling of human samples, is in process with new data generated continually. The databases, with their trillions of data points, are dynamic and are re-evaluated as new insights into the disease are generated by other types of experiments executed at ALS TDI and by the worldwide research community.

Microscopy and Imaging Technologies

Microscopy and imaging at the cellular scale allows for precise observation and description of biological events within a cell or cell types and also allows for characterization of interactions between cells. Specific imaging technologies in use at ALS TDI are:

  • Immunohistochemistry (IHC) is the technology by which a protein or proteins can be localized within a tissue are cell. The proteins are often labeled with fluorescent tags of many different colors making simultaneous visualization of multiple proteins possible.
  • In situ hybridization (ISH) is the technology by which specific pieces of DNA and RNA can be localized to specific cells. This is similar to IHC which is used to localize proteins. The ISH efforts at ALS TDI are executed in collaboration with the Allen Institute for Brain Science.