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(SOD1*G93A)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(SOD1*G93A)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- TARDBP*A315T)95Balo/J (010700) mice, also called Prp-TDP43^A315T, express a mutant human TAR DNA binding protein associated with familial ALS. Hemizygous mice develop a progressive and fatal disease associated with neurodegeneration of the myenteric plexus and exhibit cellular brain pathology reminiscent of both ALS and frontotemporal lobar degeneration with ubiquitin aggregates.

Guide: Working with Amyotrophic Lateral Sclerosis (ALS) Mouse Models

This guide summarizes the guidelines for preclinical testing and colony management of ALS mice discusses considerations for preclinical studies using SOD1 mice, colony management, QPCR protocol for determining copy number, and more.

Upcoming Education