Research Models

TDP-43 (A315T)

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Species: Mouse
Genes: TARDBP
Modification: TARDBP: Transgenic
Disease Relevance: Amyotrophic Lateral Sclerosis, Frontotemporal Dementia
Strain Name: N/A

Summary

Phenotype Characterization

When visualized, these models will distributed over a 18 month timeline demarcated at the following intervals: 1mo, 3mo, 6mo, 9mo, 12mo, 15mo, 18mo+.

Absent

  • Cortical Neuron Loss
  • Lower Motor Neuron Loss
  • Premature Death

No Data

  • NMJ Abnormalities
  • Muscle Atrophy
  • Body Weight

Cortical Neuron Loss

Not observed.

Lower Motor Neuron Loss

Not observed.

Cytoplasmic Inclusions

Cytoplasmic accumulation of TDP-43 was observed by 10 months of age in the spinal cord. Furthermore, cytoplasmic aggregates were observed and often co-localized with ubiquitin. These inclusions are not detected at three months of age.

Gliosis

Progressive gliosis of both astrocytes and microglia, starting at a young age (by 3 months) in the brain and spinal cord.

NMJ Abnormalities

Unknown.

Muscle Atrophy

Unknown.

Motor Impairment

At 38 weeks of age, mice develop impairments on the accelerating Rotarod relative to non-Tg littermates.

Body Weight

Unknown.

Premature Death

Not observed.

Last Updated: 06 Mar 2018

COMMENTS / QUESTIONS

No Available Comments

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Further Reading

No Available Further Reading

Research Models

Endogenous Sod1 D83G

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Species: Mouse
Genes: SOD1
Mutations: D83G in SOD1
Modification: SOD1: Other
Disease Relevance: Amyotrophic Lateral Sclerosis
Strain Name: B6(C3H)-Sod1m1H/J

Summary

In contrast to most SOD1 mouse models, which overexpress either mutant or wild-type human SOD1, this model has a mutation in the endogenous murine Sod1 gene (Joyce et al., 2015). Although human SOD1 is not present, the A>G point mutation carried by these mice corresponds to the D83G mutation in human SOD1, which causes familial ALS.

Phenotype Characterization

When visualized, these models will distributed over a 18 month timeline demarcated at the following intervals: 1mo, 3mo, 6mo, 9mo, 12mo, 15mo, 18mo+.

Absent

  • Cytoplasmic Inclusions

No Data

  • Muscle Atrophy

Cortical Neuron Loss

Neuronal numbers comparable to wild-type at 15 weeks, but about 20 percent loss of upper motor neurons by 29 weeks. Neurons in layer V of the motor cortex appeared selectively vulnerable.

Lower Motor Neuron Loss

Neuronal numbers comparable to wild-type at 6 weeks, but loss occurred by 15 weeks. The neuronal loss then stabilized; it was not more severe at 52 weeks of age.

Cytoplasmic Inclusions

Not observed.

Gliosis

Gliosis, of both astrocytes and microglia, was evident in the spinal cord by 15 weeks. It was further elevated at 52 weeks.

NMJ Abnormalities

Denervation of a hindlimb muscle, the extensor digitorum longus, was detected by 52 weeks of age, and a decreased number of motor units in the EDL muscle.

Muscle Atrophy

Muscle atrophy was not reported, although changes to the muscle composition and histochemistry were observed.

Motor Impairment

Both male and female mice develop a variety of progressive motor symptoms. Grip strength was reduced at 6 weeks of age. Tremors developed by about 5 months of age. Rotarod performance was impaired, at 23 weeks in females and 67 weeks in males.

Body Weight

Homozygous mice, both male and female, showed reduced body weight by 4 weeks of age in contrast to wild-type littermates. Loss of excessive body weight was the primary factor leading to euthanasia.

Premature Death

Males reach end-stage sooner than females (495 ± 22 versus 588 ± 24 days). Animals were sacrificed when weight loss exceeded 20 percent of maximum weight, in accordance with animal-use guidelines. This is likely explained by the development of hepatocellular carcinomas due to SOD1 loss of function.

Last Updated: 20 Jun 2018

COMMENTS / QUESTIONS

No Available Comments

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Further Reading

No Available Further Reading

Research Models

C9-BACexp (Baloh/Lutz)

Synonyms: C9-BAC[GGGGCC]100-1000, Tg(C9orf72_3) line 112, Line F112

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Species: Mouse
Genes: C9orf72
Mutations: Hexanucleotide repeat in C9ORF72
Modification: C9orf72: Transgenic
Disease Relevance: Amyotrophic Lateral Sclerosis, Frontotemporal Dementia
Strain Name: C57BL/6J-Tg(C9orf72_i3)112Lutzy/J

Summary

Phenotype Characterization

When visualized, these models will distributed over a 18 month timeline demarcated at the following intervals: 1mo, 3mo, 6mo, 9mo, 12mo, 15mo, 18mo+.

Absent

  • Motor Impairment
  • Cortical Neuron Loss
  • Lower Motor Neuron Loss
  • NMJ Abnormalities
  • Muscle Atrophy
  • Body Weight
  • Premature Death
  • Gliosis

No Data

Cortical Neuron Loss

Not observed.

Lower Motor Neuron Loss

Not observed.

Cytoplasmic Inclusions

RNA foci throughout the nervous system starting at 3 months of age. Foci comprised of RNA transcripts in both the sense and antisense directions. Age-associated formation of dipeptide aggregates, e.g., poly-GP.

Gliosis

No increase in GFAP staining in the brain and spinal cord compared with non-Tg controls, even at 18 months of age.

NMJ Abnormalities

Not observed.

Muscle Atrophy

Not observed.

Motor Impairment

No abnormalities in grip strength, Rotarod performance, or open-field testing at a young age (3 months) or advanced age (18 months), compared with non-Tg controls.

Body Weight

No abnormalities in body weight at a young age (3 months) or advanced age (18 months) compared with non-Tg controls.

Premature Death

Normal lifespan.

Last Updated: 06 Mar 2018

COMMENTS / QUESTIONS

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Further Reading

No Available Further Reading

Research Models

PrP-hFUS (WT)

Synonyms: PWT17, PrP-hFUS(WT) line PWT17

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Species: Mouse
Genes: FUS
Modification: FUS: Transgenic
Disease Relevance: Amyotrophic Lateral Sclerosis, Frontotemporal Dementia
Strain Name: B6.Cg-Tg(Prnp-FUS)17Ljh/J

Summary

Phenotype Characterization

When visualized, these models will distributed over a 18 month timeline demarcated at the following intervals: 1mo, 3mo, 6mo, 9mo, 12mo, 15mo, 18mo+.

Absent

  • Motor Impairment
  • Cytoplasmic Inclusions
  • NMJ Abnormalities

No Data

  • Cortical Neuron Loss
  • Lower Motor Neuron Loss
  • Muscle Atrophy
  • Body Weight
  • Gliosis

Cortical Neuron Loss

No data.

Lower Motor Neuron Loss

No data.

Cytoplasmic Inclusions

Not observed.

Gliosis

No data.

NMJ Abnormalities

Not observed.

Muscle Atrophy

No data.

Motor Impairment

Not observed.

Body Weight

No data.

Premature Death

Hemizygous mice die prematurely following a brief illness characterized by poor feeding. Mean survival of hemizygotes ~203 days.

Last Updated: 06 Mar 2018

COMMENTS / QUESTIONS

No Available Comments

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Further Reading

No Available Further Reading

Research Models

PrP-hFUS (R495X)

Synonyms: PrP-hFUS*R495X transgenic line PX78, PrP-hFUS(R495X) line PX78

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Species: Mouse
Genes: FUS
Mutations: FUS R495X
Modification: FUS: Transgenic
Disease Relevance: Amyotrophic Lateral Sclerosis, Frontotemporal Dementia
Strain Name: B6.Cg-Tg(Prnp-FUS*R495X)78Ljh/J

Summary

Phenotype Characterization

When visualized, these models will distributed over a 18 month timeline demarcated at the following intervals: 1mo, 3mo, 6mo, 9mo, 12mo, 15mo, 18mo+.

Absent

  • Motor Impairment
  • Cortical Neuron Loss
  • Lower Motor Neuron Loss
  • Cytoplasmic Inclusions

No Data

  • Muscle Atrophy
  • Body Weight
  • Gliosis

Cortical Neuron Loss

Not observed.

Lower Motor Neuron Loss

Not observed.

Cytoplasmic Inclusions

Despite high levels of cytoplasmic FUS, neuronal inclusions were not observed.

Gliosis

No data.

NMJ Abnormalities

A number of abnormalities were detected in the hindlimb musculature by electromyography (EMG). These phenotypes were detectable by 8-12 months of age and included fibrillation potentials, muscle denervation, and a reduction in the number of motor units.

Muscle Atrophy

No data.

Motor Impairment

Not observed.

Body Weight

No data.

Premature Death

Hemizygous mice sporadically developed intestinal swelling leading to premature death (mean survival 118 days). Homozygous mice were more severely affected (50 percent of the original cohort died around 59 days of age).

Last Updated: 06 Mar 2018

COMMENTS / QUESTIONS

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Further Reading

No Available Further Reading

Research Models

hFUS (+/+) (PrP-hFUS)

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Species: Mouse
Genes: FUS
Modification: FUS: Transgenic
Disease Relevance: Amyotrophic Lateral Sclerosis, Frontotemporal Dementia
Strain Name: Tg (Prnp-FUS)WT3Cshw/J

Summary

This transgenic mouse model of ALS overexpresses wild-type human FUS. Motor symptoms appear at a young age, accompanied by degeneration of spinal motor neurons. End-stage disease occurs by 12 weeks of age in homozygous mice, and is characterized by severe motor dysfunction that includes hindlimb paralysis (Mitchell et al., 2013).

Phenotype Characterization

When visualized, these models will distributed over a 18 month timeline demarcated at the following intervals: 1mo, 3mo, 6mo, 9mo, 12mo, 15mo, 18mo+.

Absent

  • Cortical Neuron Loss

No Data

Cortical Neuron Loss

In the brain, overt neuronal loss was absent at end stage (~11 weeks).

Lower Motor Neuron Loss

By end stage (~11 weeks), homozygous mice had lost about 60 percent of α-motor neurons in the anterior horn of the lumbar spinal cord.

Cytoplasmic Inclusions

By end stage, cytoplasmic FUS inclusions, described as “granular” and “globular,” develop in the spinal cord of homozygous mice. Ubiquitin-positive inclusions also develop, but do not co-localize with FUS inclusions. Neurons in the brain also develop “granular” and “skein-like” FUS inclusions.

Gliosis

By end stage (~11 weeks), homozygous mice had microgliosis and astrogliosis in the anterior horn of the spinal cord and in the white matter of the dorsal columns. Reactive gliosis was absent in the brain, despite cytoplasmic inclusions of FUS in some neurons.

NMJ Abnormalities

Homozygous mice had about 20 percent fewer functional motor units in the extensor digitorum longus muscle as estimated by neurophysiological assessment.

Muscle Atrophy

Muscle histology from end stage (~11 weeks) homozygous mice showed focal muscle atrophy in hindlimb muscles.

Motor Impairment

Homozygous mice exhibited motor symptoms at four weeks of age, starting with a tremor and mild signs of hindlimb dysfunction, including gait abnormalities. Motor symptoms progressed quickly, ultimately developing into hindlimb paralysis.

Body Weight

Homozygous mice fail to gain weight normally starting about four weeks of age. Their body weight declines in subsequent weeks, often precipitating euthanasia.

Premature Death

Homozygous mice were euthanized between 10 and 13 weeks of age when they developed severe motor impairment or lost 25 percent of their body weight. Average survival was 82 ± 12 days.

Last Updated: 05 Jun 2019

COMMENTS / QUESTIONS

No Available Comments

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Further Reading

No Available Further Reading

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