Research Models

α-synuclein A53T Mouse (Tg)

Synonyms: G2-3(A53T), PrPsynA53T, A53TαS Tg mice (line G2-3), MoPrP-Huα-Syn(A53T), Hualpha-Syn(A53T), A53T aSyn Tg Mouse (Lee), alpha-synuclein A53T Mouse (Tg)

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Species: Mouse
Genes: SNCA
Mutations: SNCA A53T
Modification: SNCA: Transgenic
Disease Relevance: Parkinson's Disease
Strain Name: B6.Cg-2310039L15RikTg(Prnp-SNCA*A53T)23Mkle/J. Formerly: B6.Cg-Tg(Prnp-SNCA*A53T)23Mkle/J

Summary

These transgenic mice overexpress human α-synuclein with a PD-associated mutation (A53T). Hemizygous mice overexpress mutant α-synuclein in the brain at levels about sixfold higher than endogenous mouse α-synuclein. They develop severe progressive motor impairments around one year of age, but no overt neuronal loss (Lee et al., 2002). The data on this page refer to phenotypes observed in hemizygous animals; homozygous mice from this strain are not viable.

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

  • Neuronal Loss

No Data

Neuronal Loss

Overt neuronal loss was not reported in these mice.

Dopamine Deficiency

In symptomatic mice, striatal dopamine and metabolites DOPAC and HVA are comparable to wildtype, but at 5 months, striatal tyrosine hydroxylase is reduced.  Increased D1 receptors in the substantia nigra and decreased dopamine transporters in the nucleus accumbens and striatum have been reported.

α-synuclein Inclusions

Prior to motor deficits, these mice develop accumulations of α-synuclein in select neuronal populations, including the midbrain, cerebellum, brainstem, and spinal cord. The protein aggregates do not resemble Lewy bodies, but are thioflavin-S-positive, indicating fibrillar structure.

Neuroinflammation

In symptomatic mice, increased GFAP immunoreactivity was observed in select brain regions, including the dorsal midbrain, deep cerebellar nuclei, brainstem, and spinal cord. Cortex, hippocampus, and substantia nigra did not have increased reactivity compared with non-Tg controls.

Mitochondrial Abnormalities

At 11–14 months, mitochondria in brainstem neurons were enlarged and their co-localization with the mitochondrial fission protein Drp1 was reduced.

Motor Impairment

These mice develop severe motor impairment starting around 9-16 months of age. The deficits start out with mild hyperactivity at 7 months and progress to a wobbling movement, decreased activity, and ultimately paralysis and death.

Non-Motor Impairment

At 11–12 months, spatial memory was impaired as assessed by the Barnes circular maze.

Last Updated: 08 Feb 2019

COMMENTS / QUESTIONS

  1. This paper rigorously demonstrates that overexpression of one mutant form of alpha-synuclein results in synuclein aggregation, inclusions, and neurological disease while overexpression of wild-type human synuclein and another mutant does not. These mice should be very useful for further studying the pathophysiology of alpha-synucleinopathies.

    View all comments by David Holtzman

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Further Reading

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Research Models

α-synuclein E46K Rat (BAC Tg)

Synonyms: α-synuclein (E46K) rat, founder line 70, Human α-synuclein E46K rat, E46K aSyn Rat, Alpha-synuclein E46K Rat (BAC Tg)

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Species: Rat
Genes: SNCA
Mutations: SNCA E46K
Modification: SNCA: Transgenic
Disease Relevance: Parkinson's Disease
Strain Name: NTac:SD-Tg(SNCA*E46K)70CJLi

Summary

This rat model overexpresses mutant human α-synuclein carrying the E46K mutation associated with familial Parkinson’s disease. Hemizygous rats develop small α-synuclein aggregates in dopaminergic neurons, along with signs of oxidative stress. There is no overt loss of dopaminergic neurons. Behavioral characterization has not been reported (Cannon 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

  • Dopamine Deficiency
  • Motor Impairment
  • Neuronal Loss

No Data

  • Non-Motor Impairment
  • Neuroinflammation
  • Mitochondrial Abnormalities

Neuronal Loss

No overt loss of dopaminergic neurons out to 12 months of age.

Dopamine Deficiency

No dopamine deficiency in the striatum at 12 months of age. No serotonin deficiency in the striatum. Dopamine metabolites dihydroxyphenylacetic acid and homovanillic acid were reduced by approximately 25 percent and transmitter turnover was decreased.

α-synuclein Inclusions

By 12 months of age, intracellular aggregates were observed in dopaminergic neurons of the substantia nigra and ventral tegmental area. Aggregates noted to be fairly small compared to those observed in PD brain. In the striatum and cortex α -synuclein accumulation appeared primarily in neuronal processes.

Neuroinflammation

No data.

Mitochondrial Abnormalities

No data.

Motor Impairment

No overt motor differences out to 12 months of age, unless challenged with low-dose rotenone, upon which the rats exhibit bradykinesia, postural instability, and rigidity.

Non-Motor Impairment

No data.

Last Updated: 22 Mar 2017

COMMENTS / QUESTIONS

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Further Reading

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Research Models

DJ-1 KO Rat

Synonyms: DJ-1 knockout rat

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Species: Rat
Genes: Park7 (DJ1)
Modification: Park7 (DJ1): Knock-Out
Disease Relevance: Parkinson's Disease
Strain Name: LE-Park7em1sage; HsdSage:LE-Park7em1Sage; formerly LEH-Park7tm1sage

Summary

This knockout (KO) rat model was created at SAGE Labs (acquired first by Horizon Discovery, then by Envigo, and most recently by Inotiv) in collaboration with the Michael J. Fox Foundation. The rat carries a disrupted Park7 gene, which encodes the protein DJ-1. Homozygous DJ-1 KO rats develop motor (e.g., gait, strength) impairments and loss of dopaminergic neurons in the substantia nigra; however, levels of striatal dopamine are high (Dave et al., 2014).

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

  • Dopamine Deficiency
  • α-synuclein Inclusions

No Data

  • Neuroinflammation

Neuronal Loss

Age-related decreases in TH-positive dopaminergic neurons were reported in the substantia nigra and locus coeruleus reaching approximately 50 percent by 8 months of age. No change was found in TH-immunoreactivity in the ventral tegmental area or striatum.

Dopamine Deficiency

Striatal dopamine level was increased 2-3 fold in KO rats compared to wild-type levels at 8 months of age. Dopaminergic innervation of the dorsal striatum was intact in DJ-1 KO rats at 4 and 6 months of age compared to wild-type rats. Basal levels of dopamine metabolites and evoked levels of dopamine in the striatum were not different between KO and wild-type rats.

α-synuclein Inclusions

Staining for α-synuclein revealed no increase in the striatum or in any other brain region assessed.

Neuroinflammation

No data.

Mitochondrial Abnormalities

At 3 months of age, the mitochondrial proteome in DJ-1 KO rats was differentially expressed compared to wild-type rats. Mitochondrial respiration was also increased in KO versus wild-type rats

Motor Impairment

Abnormalities in gait and strength, vocalizations, and tongue movements were observed. By 4 months, the rats exhibited abnormal paw positioning and a shorter stride. Males showed impaired licking, longer and more frequent ultrasonic vocalizations, and an accelerated decrease in average call intensity with age. Fine motor skills were also impaired in KO versus wild-type rats by 7 months of age.

Non-Motor Impairment

Olfactory detection enhanced (16 mos). Short-term memory abnormal (4.5, 15 mos). Appetitive instrumental learning normal (4, 6, 8 mos). Coping behavior (forced-swim test) impaired (6 mos). No anxiety-like behavior (elevated plus maze; 4, 8, 17 mos), less anxiety on light-dark box (6, 8 mos). No sucrose preference at 9 mos. Sensorimotor function (adhesive removal) unaffected (4, 7, 13 mos).

Last Updated: 26 Sep 2023

COMMENTS / QUESTIONS

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Further Reading

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Research Models

APP/PS1/rTg21221

Synonyms: APPswe/PSEN1dE9/MAPT, APPswe/PSEN1dE9/CaMKIIa-tTa/TRE-Tg21221

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Species: Mouse
Genes: APP, PSEN1, MAPT
Mutations: PSEN1: deltaE9
Modification: APP: Transgenic; PSEN1: Transgenic; MAPT: Transgenic
Disease Relevance: Alzheimer's Disease
Strain Name: N/A

To investigate the influence of human tau (hTau) on amyloid pathology, APPswe/PSEN1dE9 (APP/PS1) mice were crossed with the CaMKIIa-tTA and Tg21221 lines to create the APP/PS1/rTg21221 mouse. The transgene from the Tg21221 mouse encodes wild-type hTau with four microtubule binding domains and lacking amino terminal inserts (4R/0N) under control of a tetracycline responsive elements (TRE) promotor (Hoover et al., 2010).

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

  • Tangles

No Data

  • Changes in LTP/LTD
  • Cognitive Impairment

Plaques

Cortical plaques observed between 8-10 months. Plaques larger than in control mice not expressing human tau.

Tangles

No tangles. Aggregates of misfolded and phosphorylated tau observed between 8-10 months.

Synaptic Loss

Decreased synapse density adjacent to plaques relative to more distal areas.

Neuronal Loss

Neuronal loss observed adjacent to plaques relative to more distal areas.

Gliosis

Increased astrocytosis adjacent to plaques relative to more distal areas.

Changes in LTP/LTD

No data.

Cognitive Impairment

No data.

Last Updated: 13 Apr 2018

COMMENTS / QUESTIONS

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Further Reading

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Research Models

hPFN1-G118V

Synonyms: PrP-PFN1-G118V

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Species: Mouse
Genes: PFN1
Mutations: PFN1 G118V
Modification: PFN1: Transgenic
Disease Relevance: Amyotrophic Lateral Sclerosis
Strain Name: N/A

Mutations in profilin, including G118V, are linked to rare familial cases of ALS. The profilin protein is best known for its role in polymerizing actin, but has also been shown to aggregate with RNA and proteins commonly associated with stress granules.

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

No Data

Cortical Neuron Loss

At 202 days, there was a decrease in the number of corticospinal neurons of the motor cortex.

Lower Motor Neuron Loss

Progressive loss of ventral horn neurons from 165 through 202 days of age.

Cytoplasmic Inclusions

Spinal cord motor neurons had TDP-43 puncta.

Gliosis

Astrocytosis and microgliosis were observed in the spinal cord at end stage.

NMJ Abnormalities

Denervation of gastrocnemius muscle at end stage. Muscle action potential also had reduced amplitude.

Muscle Atrophy

At 165 days, hind limb muscle atrophy was observed.

Motor Impairment

Progressive motor impairments began ~ 120 days. Mice demonstrated tremors, limb clasping, muscle weakness, gait abnormalities, as well as reduced locomotion and decreased performance on the Rotarod. 

Body Weight

Body weight peaked ~ 150 days and then progressively decreased.

Premature Death

Mice were sacrificed at an average of 202 days when they were unable to right themselves. Females on average reached 191 days while males attained 213 days.

Last Updated: 03 Mar 2017

COMMENTS / QUESTIONS

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Further Reading

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Research Models

PFN1-C71G

Synonyms: Thy1.2-PFN1C71G/Prp-PFN1C71G

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Species: Mouse
Genes: PFN1
Mutations: PFN1 C71G
Modification: PFN1: Multi-transgene
Disease Relevance: Amyotrophic Lateral Sclerosis
Strain Name: FVB/N-Tg(Prnp-PFN1*C71G)22Zxu Tg(Thy1-PFN1*C71G)67Zxu/J

Summary

Mutations in profilin, including C71G, are linked to rare familial cases of ALS. The profilin protein is best known for its role in polymerizing actin, but has also been shown to aggregate with RNA and proteins commonly associated with stress granules.

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

No neuronal loss in the cortex but neurodegeneration in medulla.

Lower Motor Neuron Loss

By 4 months there was a loss of cervical motor neurons and an increase in degenerating axons.

Cytoplasmic Inclusions

Cytoplasmic inclusions of PFN1, ubiquitin, and p62 in motor neurons around 6 months.

Gliosis

Microgliosis and astrogliosis observed in the dorsal horn by 5 months.

NMJ Abnormalities

Denervation of gastrocnemius muscle occurs by 5 months.

Muscle Atrophy

Muscle atrophy in lower hind limb occurs by 6 months.

Motor Impairment

By 4 months, mice began showing minor gate changes and at 5-6 months they began demonstrating progressive deficits in Rotorod performance, vertical behaviors, and grip strength. 

Body Weight

Body weight peaked at 4-6 months and then progressively decreased.

Premature Death

Mice were sacrificed when they were incapable of locomotion following the paralysis of two or more limbs which occurred around 7 months of age.

Last Updated: 03 Mar 2017

COMMENTS / QUESTIONS

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Further Reading

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Research Models

hTau-AT (hTau40-AT)

Synonyms: Ala152T-Tau, A152T-Tau, Tau/A152T

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Species: Mouse
Genes: MAPT
Modification: MAPT: Knock-In
Disease Relevance: Alzheimer's Disease, Frontotemporal Dementia, Other Tauopathy
Strain Name: N/A

In humans, the A152T MAPT mutation appears to act as a risk modifier to multiple neurodegenerative diseases including AD, FTD, and DLB. In contrast, the majority of pathogenic mutations in MAPT are causally linked to FTD. To investigate the consequences of the A152T mutation, this mouse model overexpresses the 2N4R isoform of human tau with the A152T mutation.

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

  • Changes in LTP/LTD

No Data

  • Plaques

Plaques

Unknown.

Tangles

Tangles in hippocampus, cortex, and spinal cord starting at 3 months with age-dependent increases. Hyperphosphorylation, conformation changes, and mislocalization.

Synaptic Loss

Synaptophysin, but not PSD95, decreased in hippocampus and cortex at 12 months. By Golgi staining, spines unchanged in CA1 at 10 months, increased in CA3 at 12 months, and decreased in CA1 and CA3 at 16 months.

Neuronal Loss

Neuron loss in the hippocampus and cortex at 12 months.

Gliosis

Astrocytosis and microgliosis at 10 months.

Changes in LTP/LTD

Unchanged at 12 months.

Cognitive Impairment

No change at 10 months but at 16 months deficits in learning and memory (Morris water maze).

Last Updated: 10 Feb 2017

COMMENTS / QUESTIONS

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Further Reading

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Research Models

hTau-A152T

Synonyms: CaMKII-tTA/TRE-hTau-A152T

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Species: Mouse
Genes: MAPT
Modification: MAPT: Transgenic
Disease Relevance: Alzheimer's Disease, Frontotemporal Dementia, Other Tauopathy
Strain Name: C57BL/6-Tg(tetO-MAPT*A152T)L1Lms/J

In humans, the A152T MAPT mutation appears to act as a risk modifier to multiple neurodegenerative diseases, including AD, FTD, and DLB. In contrast, the majority of pathogenic mutations in MAPT are causally linked to FTD. To investigate the consequences of the A152T mutation, this mouse model overexpresses the 1N4R isoform of human tau with the A152T mutation.

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

  • Tangles
  • Changes in LTP/LTD

No Data

  • Plaques
  • Synaptic Loss

Plaques

No data.

Tangles

Abnormal accumulations of soluble tau were observed, but not tangles or tangle-like structures.

Synaptic Loss

No data.

Neuronal Loss

Neuron loss in the hippocampus was observed by 20 months.

Gliosis

Astrocytosis, but no differences in microglia.

Changes in LTP/LTD

Unchanged at 20 months.

Cognitive Impairment

In the Morris water maze, performance was impaired after 17 months of age. Nest building was impaired at 10-14 months. Social interaction, anxiety, exploratory behavior, and motor functions were unaltered.

Last Updated: 30 Nov 2018

COMMENTS / QUESTIONS

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Further Reading

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Research Models

Tau35

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Species: Mouse
Genes: MAPT
Modification: MAPT: Transgenic
Disease Relevance: Progressive Supranuclear Palsy, Alzheimer's Disease, Frontotemporal Dementia, Other Tauopathy
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

  • Gliosis

No Data

  • Plaques
  • Neuronal Loss
  • Changes in LTP/LTD

Plaques

Unknown.

Tangles

Abnormally phosphorylated tau detected at two months and by eight months tau was mislocalized and misfolded and dystrophic neurites were observed. Tangle-like structures observed in the hippocampus by 14 months.

Synaptic Loss

At 14 months synapsin1 protein levels were decreased but synaptophysin levels remained at wild-type levels.

Neuronal Loss

Cell death was not formally assessed, however, overt neuronal death was not seen in the hippocampus.

Gliosis

Gliosis was not observed at 14 months.

Changes in LTP/LTD

Unknown.

Cognitive Impairment

In the Morris water maze, Tau35 had the same performance as wild-type animals at six months but developed progressive deficits by eight months. 

Last Updated: 03 Feb 2017

COMMENTS / QUESTIONS

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Further Reading

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