Amyotrophic Lateral Sclerosis (ALS) Efficacy Studies

Amyotrophic Lateral Sclerosis (ALS) or Lou Gehrig's disease is a devastating and rapidly fatal disease with currently only one available, FDA-approved, modestly effective treatment. This disease is marked by progressive neurodegeneration of nerve cells in the brain and spinal cord that lead to loss of voluntary muscle movement.

ALS Efficacy Studies

Efficacy studies performed by JAX® In Vivo Services for ALS typically feature clinical observations, body weights, progressive neurological scoring, electrophysiological assessment of motor neuropathy, survival, tissue/blood collection and histological assessment of neuromuscular junctions and femoral nerves.

B6SJL-Tg(SOD1G93A)1Gur/J (002726), also called G93A-SOD1, express a human transgene that carries the G93A mutant form of human SOD1 on a mixed C57BL/6J and SJL/J genetic background.
B6.Cg-Tg(SOD1G93A)1Gur/J (004435) mice carry the same transgene as 002726 but on a C57BL/6J congenic background. They develop similar phenotypes as the mixed background strain, but have a slightly longer life span.
B6.Cg-Tg(Prnp-TARDBPQ331K)103Dwc/J (017933) mice, also called Prp-TDP-43Q331K line 103, express a mutant human TAR DNA binding protein associated with familial ALS. The Prp-TDP-43Q331K [~1.5x] mice have moderate overexpression levels in total TDP-43 mRNA/protein, with an ~2.5-fold increase in total TDP-43 expression (huTDP-43Q331K levels ~1.5-fold greater) compared to endogenous TDP-43 in non-transgenic mice. By three months of age, Prp-TDP-43Q331K [~1.5x] mice develop adult-onset motor dysfunction (as measured by rotarod performance) accompanied by a loss of hindlimb-grip strength and the appearance of muscle fasciculations.