Our Techniques
Gene Therapy
Gene Therapy is a highly targeted therapeutic approach designed to insert modified
genes into cells in order to produce a potentially therapeutic protein or reduce
production of a potentially toxic target. In a neurodegenerative disease like ALS
there are multiple cell types potentially involved in disease including motor neurons,
glial cells in the central nervous system, skeletal muscle, and peripheral immune
cells. In order to reduce potential toxicity it is often necessary to target the
gene therapy construct to specific tissues or even specific cell types within a
given tissue.
Research Biology
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.
Validation & Biomarkers
In vitro validation uses living cells grown in nutrient solutions for experiments
that confirm the activity of promising therapeutics prior to initiation of longer
and more costly in vivo (animal) studies. Many of the same technologies used to
evaluate in vivo studies are also employed to interpret in vitro results. Biomarkers
are biological signals that can be used to definitively diagnose a condition or
disease and can also correlate with disease progression. Biomarkers can come from
blood sampling, tissue biopsy, or less invasive techniques like skin elasticity
measurement.
Testing Therapeutics
In vivo validation involves testing therapeutics in an animal model of disease.
ALS TDI tests small molecules, gene therapies, biologics and stem cell products
in its state-of-the-art research facility. Established in 1995, the SOD1 mouse model
is internationally accepted as a robust model for ALS research. In 2008, ALS TDI
published guidelines for the model's use and interpretation of experimental results.
Schedule a Lab Tour
We take our open-door philosophy and commitment to transparency very seriously. And, we love to show off.
Click here if you’d like to visit ALS TDI and get an up-close-and-personal look
at the lab.
Virtual Lab Tour
Check outALS TDI's Virtual Lab Tour.
You'll be able to explore several areas of therapeutics that are part of the world's most
comprehensive ALS research program.