Amyotrophic Lateral Sclerosis (ALS) Efficacy Studies

Amyotrophic Lateral Sclerosis (ALS), or Lou Gehrig’s disease, is a devastating and rapidly fatal disease marked by progressive neurodegeneration of nerve cells in the brain and spinal cord that lead to loss of voluntary muscle movement.

Efficacy studies performed by JAX® In Vivo Services for ALS feature:

  • Clinical observations including body weight collection, neurological scoring & survival curve analysis
  • Behavioral phenotyping including rotorod, wire hang & grip strength
  • Electrophysiological assessment of motor neuropathy
  • Biospecimen collection and histological assessment of neuromuscular junctions and femoral nerves

Amyotrophic Lateral Sclerosis (ALS) Solutions

  • JAX Solutions

  • Featured Models

JAX Solutions

{insert a sentence or two around the importance of SOD1 G93A models in drug development/efficacy testing}

Featured Models

STRAIN No. SIGNIFICANT MODELING STRAIN UTILITY
002726
B6SJL-Tg(SOD1*G93A)1Gur/J
  • Expresses a G93A mutant form of human SOD1
  • Mice become paralyzed due to motor neuron loss in the spinal cord
  • 50% of hemizygous mice live to ~130 days
 {why would someone want to use this model in their testing}
004435
B6.Cg-Tg(SOD1*G93A)1Gur/J
  • Expresses a G93A mutant form of human SOD1
  • Mice become paralyzed due to motor neuron loss in the spinal cord
  • 50% of hemizygous mice live to~130 days
 {why would someone want to use this model in their testing}

  • JAX Solutions

  • Featured Models

JAX Solutions

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Featured Models

STRAIN No. SIGNIFICANT MODELING STRAIN UTILITY
023099
C57BL/6J-Tg(C9orf72_i3)112Lutzy/J
  • Expresses several tandem copies of C9Orf72_3 transgene, with each copy encoding the human C9Orf72 with a hexanucleotide repeat expansion.
  • Each Tg expresses 100-1000 repeats.
  • RNA Foci and polyGPpeptides are observed by 3mo of age
  • Hom mice show overt paralysis by 16mo of age
 {why would someone want to use this model in their testing}
029099
FVB/NJ-Tg(C9orf72)500Lpwr/J
  • BAC transgenic that expresses a human C9Orf72 gene with ~500 hexanucleotide repeats
  • Reliably express polyGP DPRs
 {why would someone want to use this model in their testing}

  • JAX Solutions

  • Featured Models

JAX Solutions

{insert a sentence or two around the importance of TDP43models in drug development/efficacy testing}

Featured Models

STRAIN No. SIGNIFICANT MODELING STRAIN UTILITY
012836
B6;SJL-Tg(Thy1-TARDBP)4Singh/J
  • Thy1 promoter drivigexpression of human TDP-43 in neurons of brain and spinal cord
  • Degeneration of spinal & coritcal motor neuron, leading to paralysis(ALS) and nonmotor cortical neurosn (FTLD)
  • Accumulation of pTDP-43 & ubiquinated TDP43 in spinalcord & brain
  • Hemi mice show abnormal limb reflex by 14 months, motor performance issues by 15 months
 {why would someone want to use this model in their testing}
028412
B6;C3-Tg(NEFH-tTA)8Vle Tg(tetO-TARDBP*)4Vle/J
  • Regulatable overexpression of mutant hTDP-43-deltaNLS protein widespread in the brain & spinal cord
  • Expression is rapid, occurring 1 week off DOX; strain must be on DOX to prevent the phenotype from emerging
  • Significant spinal motor neuron loss & muscledevervation leading to rapid, robust phenotype (motor deficits, denervation of NMJs, motor neuron loss & premature death -8-20wks)
 {why would someone want to use this model in their testing}

  • JAX Solutions

  • Featured Models

JAX Solutions

{insert a sentence or two around the importance of TDP43models in drug development/efficacy testing}

Featured Models

STRAIN No. SIGNIFICANT MODELING STRAIN UTILITY
030568
C57BL/6N-Mdga2Tg(Prnp-PFN1*G118V)838Kiaei/J
  • Expresses human profilin 1 encoding the G118V mutation driven by the Prpto express in brain and spinal cord
  • Loss of lower & upper motor nuerons, mutant profilin 1 aggregation, abnormally ubiquinated proteins, reduced ChATexpression, fragmented mitochondria, glial cell activation, muscle atrophy, weight loss & shortened survival
  • Disease onset is 120 days -survival around 200 days
 {why would someone want to use this model in their testing}

AMYOTROPHIC LATERAL SCLEROSIS (ALS) Online Resources