Alzpedia

PICALM

Synonyms: phosphatidylinositol binding clathrin assembly protein, clathrin assembly lymphoid-myeloid leukemia (CALM) protein, LAP, CLTH

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PICALM is an accessory protein in the endocytic pathway. Ubiquitously expressed, PICALM binds to clathrin and its adaptor proteins, which together regulate the formation of the clathrin lattice during endocytosis. PICALM was identified in one of the first large-scale genome-wide association studies (GWAS) for late-onset Alzheimer’s disease and remains one of the top 10 risk genes on AlzGene. Multiple single nucleotide polymorphisms (SNPs) within and around the PICALM gene have been associated with AD; however, the pathogenic variant(s) and the underlying mechanisms by which they affect a person’s risk of AD remain unknown.

PICALM is involved in many cellular processes, with many opportunities to influence AD pathogenesis. A prominent hypothesis holds that it affects internalization of APP and thus the production of Aβ. Additionally, PICALM could act through effects on the endocytosis and trafficking of other molecules important for neuronal function.

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Alzpedia

Complement Receptor 1 (CR1)

Synonyms: Complement component (3b/4b) receptor 1, CD35 antigen, C3b/C4b receptor, C3-binding protein, Knops blood group antigen

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The complement receptor 1 (CR1) gene encodes a transmembrane glycoprotein that functions in the innate immune system. CR1 is expressed on blood cells and microglia. As a receptor for the complement components C3b and C4b, CR1 helps regulate activation of the complement cascade and promotes phagocytosis of immune complexes and cellular debris, as well as Aβ. CR1 attracted attention in Alzheimer’s research when variants at the CR1 locus proved to be associated with risk of late-onset Alzheimer’s disease. Meta-analysis has confirmed several disease-associated single nucleotide polymorphisms (SNPs), as well as a copy-number variation (CNV). CR1 currently ranks among the top 10 risk genes on AlzGene. At least one risk SNP is also linked to intracerebral hemorrhage associated with cerebral amyloid angiopathy (CAA).

While the molecular underpinnings of CR1’s role in AD pathogenesis are not yet clear, accumulating evidence suggests a dysregulation of complement with effects on inflammation and amyloid accumulation. Clinically, a CR1 risk allele has been associated with faster cognitive decline and greater neuropathology burden in longitudinal aging cohorts. 

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Alzpedia

Clusterin

Synonyms: apolipoprotein J (ApoJ), sulfated glycoprotein-2 (SGP-2), secreted glycoprotein gp80, complement-associated protein SP-40, complement lysis inhibitor (CLI), testosterone- repressed prostate message 2 (TRPM-2)

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Clusterin is a highly conserved glycoprotein that functions primarily as an extracellular chaperone. Multiple large genome-wide association studies (GWAS) have uncovered clusterin variants that strongly associate with late-onset Alzheimer’s disease, earning clusterin a spot on AlzGene’s top 10 risk genes.

Clusterin levels are elevated in the AD brain, but how the protein affects AD pathogenesis is unknown. One idea holds that pathogenic clusterin variants weaken the brain’s ability to respond to stressors, another that they work through reduced expression of clusterin. Prevailing hypotheses revolve around clusterin’s ability to bind Aβ peptides and thereby influence their aggregation, deposition, and/or clearance, but the underlying mechanism(s) remain to be delineated. Functional studies are starting to associate clusterin variants with neurological phenotypes. Clusterin is detectable in blood and cerebrospinal fluid, but thus far it has not proven useful as a biomarker.

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Alzpedia

Bridging integrator 1 (BIN1)

Synonyms: amphiphysin 2, AMPH2, AMPHL, SH3P9

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Bridging integrator 1 (BIN1) is a widely expressed adaptor protein that is part of the Bin1/amphiphysin/RVS167 (BAR) family. BIN1 functions in clathrin-mediated endocytosis and endocytic recycling, as does the AD risk gene PICALM. Whereas brain-specific isoforms may be involved in the retrieval of synaptic vesicles, ubiquitous isoforms of BIN1 participate in apoptosis, inflammation and calcium homeostasis.

The bulk of the scientific literature on BIN1 concerns its initially identified roles in tumor suppression and muscle development, but in 2010, a large genome-wide association study (GWAS) discovered that BIN1 was a risk factor for late-onset Alzheimer’s disease. This association was confirmed in subsequent GWAS in different populations and in meta-analyses, and BIN1 remains near the top of AlzGene.

The pathogenic mechanism of BIN1 is unknown. A clustering of high-risk polymorphisms upstream of the gene points toward a potential effect on transcription, and AD patients carrying such BIN1 variants have been found to have elevated BIN1 expression in the brain. DNA methylation of the BIN1 promoter has been suggested as a possible epigenetic mechanism influencing AD risk.

BIN1 has also been proposed to modulate tau pathology, but the AD-associated variants known to date do not affect CSF tau levels. As an endocytic accessory protein, BIN1 could potentially affect BACE trafficking and APP metabolism, as well. The BIN1 risk locus has been reported to track with certain brain imaging features of AD.

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Alzpedia

APOE

Synonyms: Apolipoprotein E, LPG, LDLCQ5, AD2

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Apolipoprotein E is a secreted apolipoprotein involved in lipid metabolism. It complexes with lipids to form lipoprotein particles that transport cholesterol and triglycerides via the bloodstream and facilitate the transfer of cholesterol and phospholipids between cells in the brain. In the periphery, ApoE is expressed by hepatocytes and macrophages. In the brain, it is produced and secreted primarily by astrocytes and activated microglia.

ApoE has three isoforms—ApoE2, E3, and E4—with unique cysteine/arginine combinations at positions 130 and 176. Every person has an APOE genotype determined by homozygous or heterozygous pairs of the alleles encoding these isoforms—for example, APOE3/E3 or APOE3/E4. The APOE4 allele is thought to be the ancestral form of the APOE gene which gave rise to APOE2 and APOE3.

While APOE4 increases the risk for late-onset Alzheimer’s disease, APOE2 reduces it. The largest datasets from studies of Caucasian populations show APOE4 increasing AD risk in a dose-dependent manner with one allele raising risk about threefold, and two alleles elevating it 10- to 15-fold relative to APOE3. In contrast, one APOE2 allele cuts AD risk down to about half of that of carriers of two APOE3 alleles, and two alleles reduce it to 13 percent.

Overall, the population risk that can be attributed to APOE4 is approximately 20 percent, although its effects vary between populations of different ancestries. APOE4 also modifies the age of AD onset, with carriers’ symptoms emerging about five years earlier than those of non-carriers. Whether and under what circumstances APOE4 affects disease progression remains uncertain, although most studies suggest it accelerates it.

ApoE4 initiates a cascade of early alterations which can lead to later pathological changes associated with AD, including both gain- and loss-of-function effects relative to ApoE3. It appears to contribute to both hallmark AD pathologies, amyloid plaques and neurofibrillary tangles. In addition, it has been blamed for altering lipid transport and metabolism in brain cells and fueling neuroinflammation, abnormal cellular stress responses, and blood-brain barrier breakdown. ApoE4 has also been reported to reduce neurogenesis, disrupt endocytosis and intracellular trafficking, impair mitochondrial function and energy metabolism, as well as modify transcription. At a tissue level, it appears to alter brain glucose metabolism and cause neural network dysfunction.

Multiple approaches to therapeutically counter the harmful effects of ApoE4 are being examined, ranging from directly targeting the protein to mitigating its downstream effects.

In addition to the three common APOE alleles—APOE2, E3, and E4—genetic variants in other positions, many of them rare, have also been studied (see APOE genetic variant dataset). Some have been tied to AD or to other neurological disorders, and/or to peripheral conditions including alterations of lipid metabolism, cardiovascular health, and kidney function.

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Research Models

TMHT (Thy-1 mutated human tau)

Synonyms: TAU 441, hTAU441, TAU441 V337M R406W

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Species: Mouse
Genes: MAPT
Modification: MAPT: Transgenic
Disease Relevance: Alzheimer's Disease
Strain Name: N/A

Neuropathology

Increased total tau and phosphorylated tau species (Thr181, Ser199, Thr231) in the amygdala and hippocampus starting at three months.

Cognition/Behavior

Spatial memory deficits starting at five months by the Morris water maze. Olfactory deficits at five months indicated by the buried food test. No motor deficits (by rotarod, beam walk) or depressive behavior as indicated by the forced swim test.

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

  • Plaques

No Data

Plaques

Absent.

Tangles

Tangles at 4 months and progress with age.

Cognitive Impairment

Cognitive impairment by 5 months as measured by the Morris Water Maze.

Last Updated: 03 Apr 2024

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Research Models

mThy1-hAPP751 (TASD41)

Synonyms: Line 41, hAPPSL, hAPP-SL, AβPP751, mThy1-hAβPP751 Swe Lon (line 41), APP751SL, hAPPlon/swe line 41, APP41

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Species: Mouse
Genes: APP
Modification: APP: Transgenic
Disease Relevance: Alzheimer's Disease
Strain Name: mThy1-hAβPP751 Swe Lon

Summary

These transgenic mice express mutant human APP with both the Swedish (K670N/M671L) and London (V717I) mutations. They develop age-associated amyloid plaques and cognitive impairment.

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
  • Neuronal Loss

No Data

  • Changes in LTP/LTD

Plaques

Plaques start at 3-6 months in the frontal cortex and become widespread with age, affecting the piriform and olfactory cortices, hippocampus, and thalamus (Rockenstein et al., 2001; Havas et al., 2011).

Tangles

Absent.

Synaptic Loss

Dystrophic neurites and synaptic loss starting at 12 months.

Neuronal Loss

Absent.

Gliosis

Inflammation related to activated microglia (increased CD11) and reactive astrocytes (increased GFAP) is significant by 6 months and increases with age.

Changes in LTP/LTD

Unknown.

Cognitive Impairment

Cognitive impairment observed by 6 months by Morris Water Maze (Rockenstein et al., 2005).

Last Updated: 15 Apr 2024

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Further Reading

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Research Models

J20 (PDGF-APPSw,Ind)

Synonyms: PDGF-hAPP695,751,770V171F, KM670/671NL, hAPPJ20, hAPP, Mucke mice

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Species: Mouse
Genes: APP
Modification: APP: Transgenic
Disease Relevance: Alzheimer's Disease
Strain Name: B6.Cg-Zbtb20Tg(PDGFB-APPSwInd)20Lms/2Mmjax

Summary

This popular mouse model overexpresses human APP with two mutations linked to familial Alzheimer's disease (the Swedish and Indiana mutations). Transgene expression is driven by the PDGF-β promoter and immunoreactivity is detected in neurons throughout the brain with highest levels in the neocortex and hippocampus.

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

Plaques

At 5-7 months of age diffuse amyloid-β plaques deposit in the dentate gyrus and neocortex. Amyloid deposition is progressive with widespread plaques by 8-10 months. Aβ puncta are deposited in the hippocampus as early as 1 month (Hong et al., 2016).

Tangles

Absent.

Synaptic Loss

Age-dependent loss of synaptophysin, synaptotagmin, PSD-95, and homer immunoreactivity in the hippocampus by 3 months; synapse loss was confirmed by electron microscopy. No significant difference was seen at 1 month (Hong et al., 2016).

Neuronal Loss

Cell loss varies by brain region. No significant neuronal loss was observed in the CA3 region of the hippocampus at 6, 12, 24 and 36 weeks of age nor in the CA1 region at 6 weeks; however, at 12, 24, and 36 weeks significant neuronal loss was observed in the CA1 region compared to age-matched wild-type animals (Wright et al., 2013).

Gliosis

At 24 and 36 weeks a significant increase in the number of reactive GFAP+ astrocytes and CD68+ microglia was observed in the hippocampi of J20 mice compared to age-matched wild-type controls. No significant difference was observed at 6 and 12 weeks (Wright et al., 2013).

Changes in LTP/LTD

Basal synaptic transmission is impaired between 3-6 months; extracellularly recorded field EPSPs at the Schaffer collateral to CA1 synapse in acute hippocampal slices were on average smaller in amplitude than those seen in wild-type mice. Significant deficits in LTP at the Schaffer collateral–CA1 synapse compared with control mice at 3-6 months (Saganich et al., 2006).

Cognitive Impairment

Deficits in spatial memory and learning appear as the mice age. By 4 months, J20 mice demonstrate spatial reference memory deficits as measured by the radial arm maze (Wright et al., 2013) and Morris water maze (Cheng et al., 2007).

Last Updated: 03 Nov 2017

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Therapeutics

Varenicline

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Overview

Name: Varenicline
Synonyms: Champix™, Chantix™, Varenicline tartrate, CP-526,555, Alpha4 beta2 nicotinic receptor agonist
Chemical Name: 7,8,9,10-Tetrahydro-6,10-methano-6H-pyrazino[2,3-h][3]benzazepin
Therapy Type: Small Molecule (timeline)
Target Type: Cholinergic System (timeline)
Condition(s): Alzheimer's Disease
U.S. FDA Status: Alzheimer's Disease (Discontinued)
Company: Pfizer
Approved for: Smoking cessation

Background

Varenicline is a medication approved in the United States and many other countries to aid smoking cessation. It is a partial agonist of the α4β2 subtype of the nicotinic acetylcholine receptor (nAChR). It also acts on α3β4 and α7 nACHRs.  Acting as a partial agonist, varenicline stimulates the α4β2 receptor without producing the full effect of the nicotine ligand.  Its competitive binding on these receptors blocks the ability of nicotine to stimulate the mesolimbic dopamine system, hence its indicated use. Varenicline also acts as an agonist at 5-HT3 serotonine receptors, which are thought to contribute to its mood-altering effects. Side effects of vareniclin include as changes in behavior, agitation, depressed mood, and suicidal ideation, both in people with and without pre-existing psychiatric conditions. Vareniclin is one of several α4β2 nACHRs agonists that have been tested as treatments for Alzheimer's disease.

Findings

In 2009 and 2010, Pfizer compared varenicline to placebo in a six-week Phase 2 trial in 66 patients with mild to moderate Alzheimer's disease for its ability to improve cognition as measured by the ADAS-Cog. The multicenter trial was conducted in South Korea. The trial assessed varenicline's safety, tolerability, and ability to improve cognition as measured by the ADAS-Cog. Fifty-four people completed the trial. Varenicline did not improve cognition, nor did it have a benefit on most of the study's secondary outcomes. It did, however, worsen participants' neuropsychriatic state and caused gastrointestinal side effects. Development of varenicline for Alzheimer's therapy was discontinued in 2011. For results of this trial see clinicaltrials.gov.

Last Updated: 11 Jan 2016

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