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ALS mouse models

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By Targets:	Apoptosis (1) DNA/RNA Synthesis (4) iGluR (2) Integrin (1) NF-κB (1) Reactive Oxygen Species (ROS) (1) RIBOTAC (2) SOD (3) Trk Receptor (1) TRP Channel (2)
By Research Areas:	Cancer (1) Inflammation/Immunology (1) Neurological Disease (12)
Oligonucleotides:	Antisense Oligonucleotides (2)

Inhibitors & Agonists

Inhibitory Antibodies

Natural
Products

Oligonucleotides

Targets Recommended: Acetolactate Synthase (ALS)

Cat. No.	Product Name	Target	Research Areas	Chemical Structure

HY-147410

Ulefnersen 1 Publications Verification ION-363	DNA/RNA Synthesis	Neurological Disease
Ulefnersen (ION363) is an Antisense Oligonucleotide (ASO) directed against the 6th intron of the fused-in sarcoma (FUS) transcript to silence FUS in a non-allele-specific manner. Ulefnersen can reduce postnatal levels of FUS protein in the brain and spinal cord in disease-relevant mouse model of ALS-FUS , delaying motor neuron degeneration. Ulefnersen can be used in the research of Amyotrophic Lateral Sclerosis (ALS) .

HY-141878

di-Ellipticine-RIBOTAC	RIBOTAC DNA/RNA Synthesis	Neurological Disease
di-Ellipticine-RIBOTAC is a RNase recruiting chimera (RIBOTAC) degrader, capable of specifically binding and degrading expanded G4C2 RNA repeat (r(G₄C₂) ^exp). di-Ellipticine-RIBOTAC selectively binds the three-dimensional (3D) structure formed by r(G₄C₂) ^exp and that recruits an endogenous ribonuclease (RNase) to cleave r(G₄C₂) ^exp. di-Ellipticine-RIBOTAC selectively degrades the mutant chromosome 9 open reading frame 72 (C9orf72) allele and reduces quantities of toxic dipeptide repeat proteins (DPRs) translated from r(G₄C₂) ^exp. di-Ellipticine-RIBOTAC significantly improves the pathological phenotype of amyotrophic lateral sclerosis/ frontotemporal dementia (c9ALS/FTD) in cells and mouse models. di-Ellipticine-RIBOTAC can be used for the study of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) .

HY-147410A

Ulefnersen sodium 1 Publications Verification ION-363 sodium	DNA/RNA Synthesis	Neurological Disease
Ulefnersen sodium (ION363) is an Antisense Oligonucleotide (ASO) directed against the 6th intron of the fused-in sarcoma (FUS) transcript to silence FUS in a non-allele-specific manner. Ulefnersen sodium can reduce postnatal levels of FUS protein in the brain and spinal cord in disease-relevant mouse model of ALS-FUS , delaying motor neuron degeneration. Ulefnersen sodium can be used in the research of Amyotrophic Lateral Sclerosis (ALS) .

HY-W611371

FP802	TRP Channel iGluR	Neurological Disease
FP802 is an orally active potent TwinF interface inhibitor that disrupts and detoxifies the NMDAR/TRPM4 death complex. FP802 exerts powerful neuroprotective effects in the 5xFAD mouse model of Alzheimer’s disease (AD) by preventing cognitive decline, preserving neuronal structural integrity, reducing amyloid-β plaque formation, and mitigating mitochondrial pathology . FP802 stops loss of motor neurons, reduces serum neurofilament light chain (NfL) levels, improves motor performance, and extends life in a mouse model of amyotrophic lateral sclerosis (ALS). FP802 can be used for AD and ALS research .

HY-138131

SOD1-Derlin-1 inhibitor-1	SOD	Neurological Disease
SOD1-Derlin-1 inhibitor-1 (compound 56-20) is an inhibitor of SOD1-Derlin-1 interaction. SOD1-Derlin-1 inhibitor-1 inhibits SOD1 ^G93A-Derlin-1 complex with an IC₅₀ value of 7.11 μM. SOD1-Derlin-1 inhibitor-1 can be used for the research of amyotrophic lateral sclerosis .

HY-123982

SOD1-Derlin-1 inhibitor-2	SOD	Neurological Disease
SOD1-Derlin-1 inhibitor-2 (compound 56-59) is an inhibitor of SOD1-Derlin-1 interaction. SOD1-Derlin-1 inhibitor-2 attenuates the interactions between Derlin-1 and SOD1 ^mut. SOD1-Derlin-1 inhibitor-2 can be used for the research of amyotrophic lateral sclerosis (ALS) .

HY-170409

BNN27	Trk Receptor Apoptosis	Neurological Disease Inflammation/Immunology
BNN27 is the agonist for TrkA receptor and p75NTR receptor, that exhibits neurotrophic and anti-apoptotic effects. BNN27 increases the levels of glutamate, GABA, and glutamine in the rat hippocampus and prefrontal cortex, improves glutamate turnover. BNN27 exhibits neuroprotective efficacy in mouse amyotrophic lateral sclerosis (ALS) model, exhibits anti-inflammatory efficacy in experimental autoimmune encephalomyelitis (EAE) model, exhibits retinal protective efficacy in rat diabete models. BNN27 is blood-brain barrier penetrable .

HY-W611371A

FP802 dihydrochloride	TRP Channel iGluR	Neurological Disease
FP802 dihydrochloride is an orally active potent TwinF interface inhibitor that disrupts and detoxifies the NMDAR/TRPM4 death complex. FP802 dihydrochloride exerts powerful neuroprotective effects in the 5xFAD mouse model of Alzheimer’s disease (AD) by preventing cognitive decline, preserving neuronal structural integrity, reducing amyloid-β plaque formation, and mitigating mitochondrial pathology . FP802 dihydrochloride stops loss of motor neurons, reduces serum neurofilament light chain (NfL) levels, improves motor performance, and extends life in a mouse model of amyotrophic lateral sclerosis (ALS) . FP802 dihydrochloride can be used for AD and ALS research .

HY-N2707

6-Deoxyjacareubin	Reactive Oxygen Species (ROS)	Neurological Disease Cancer
6-Deoxyjacareubin is a natural xanthone, that can be isolated from the leaves of Vismia latifolia. 6-Deoxyjacareubin protects against non-apoptotic cell death by inhibiting ROS production. 6-Deoxyjacareubin ameliorates neurodegeneration in a mouse model of familial amyotrophic lateral sclerosis (ALS) .

HY-141878A

di-Ellipticine-RIBOTAC TFA	RIBOTAC DNA/RNA Synthesis	Neurological Disease
di-Ellipticine-RIBOTAC TFA is a RNase recruiting chimera (RIBOTAC) degrader, capable of specifically binding and degrading expanded G4C2 RNA repeat (r(G₄C₂) ^exp). di-Ellipticine-RIBOTAC TFA selectively binds the three-dimensional (3D) structure formed by r(G₄C₂) ^exp and that recruits an endogenous ribonuclease (RNase) to cleave r(G₄C₂) ^exp. di-Ellipticine-RIBOTAC TFA selectively degrades the mutant chromosome 9 open reading frame 72 (C9orf72) allele and reduces quantities of toxic dipeptide repeat proteins (DPRs) translated from r(G₄C₂) ^exp. di-Ellipticine-RIBOTAC TFA significantly improves the pathological phenotype of amyotrophic lateral sclerosis/ frontotemporal dementia (c9ALS/FTD) in cells and mouse models. di-Ellipticine-RIBOTAC TFA can be used for the study of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) .

HY-P992436

PAS-003	Integrin	Neurological Disease
PAS-003 is a humanized monoclonal antibody targerting α5β1 integrin. PAS-003 modulates immune cell migration, immune cell adhesion, improves behavior, and improves survival in ALS mouse models. PAS-003 can be used for the research of amyotrophic lateral sclerosis .

HY-182446

SRI-22818	NF-κB SOD	Neurological Disease
SRI-22818 is a NF-κB activator with an EC₅₀ of 0.5 μM. SRI-22818 upregulates the expression of NF-κB and promotes its activation. SRI-22818 increases the level of SOD2. SRI-22818 exhibits activity in the SOD1 ^G93Amodel of amyotrophic lateral sclerosis (ALS). SRI-22818 can be used for research related to amyotrophic lateral sclerosis .

Cat. No.	Product Name	Target	Research Area	Image

HY-P992436

PAS-003	Integrin	Neurological Disease
PAS-003 is a humanized monoclonal antibody targerting α5β1 integrin. PAS-003 modulates immune cell migration, immune cell adhesion, improves behavior, and improves survival in ALS mouse models. PAS-003 can be used for the research of amyotrophic lateral sclerosis .

6-Deoxyjacareubin	Structural Classification Garcinia intermedia (Pittier) Hammel Xanthones Guttiferae Phenols Plants Source Classification	Reactive Oxygen Species (ROS)
6-Deoxyjacareubin is a natural xanthone, that can be isolated from the leaves of Vismia latifolia. 6-Deoxyjacareubin protects against non-apoptotic cell death by inhibiting ROS production. 6-Deoxyjacareubin ameliorates neurodegeneration in a mouse model of familial amyotrophic lateral sclerosis (ALS) .

Cat. No.	Product Name		Classification

HY-147410

Ulefnersen 1 Publications Verification ION-363		Antisense Oligonucleotides
Ulefnersen (ION363) is an Antisense Oligonucleotide (ASO) directed against the 6th intron of the fused-in sarcoma (FUS) transcript to silence FUS in a non-allele-specific manner. Ulefnersen can reduce postnatal levels of FUS protein in the brain and spinal cord in disease-relevant mouse model of ALS-FUS , delaying motor neuron degeneration. Ulefnersen can be used in the research of Amyotrophic Lateral Sclerosis (ALS) .

HY-147410A

Ulefnersen sodium 1 Publications Verification ION-363 sodium		Antisense Oligonucleotides
Ulefnersen sodium (ION363) is an Antisense Oligonucleotide (ASO) directed against the 6th intron of the fused-in sarcoma (FUS) transcript to silence FUS in a non-allele-specific manner. Ulefnersen sodium can reduce postnatal levels of FUS protein in the brain and spinal cord in disease-relevant mouse model of ALS-FUS , delaying motor neuron degeneration. Ulefnersen sodium can be used in the research of Amyotrophic Lateral Sclerosis (ALS) .

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BODIPY 493/503 purchased from MedChemExpress. Usage Cited in: Nat Commun. 2025 Feb 1;16(1):1241. [Abstract]

MedchemExpress Validation 01

Western blot analysis of extracts from THP-1(lane 2(20μg), Jurkat (lane 3(20μg) and NIH3T3(lane 4(20μg) using FOXO1A (HY-P80132) Rabbit mAb. Proteins were transferred to a PVDF membrane and blocked with 5% non-fat milk in TBST for 2 hour at room temperature. The primary antibody (1/1000) and Loading control antibody (Beta Actin, HY-P80438, 1/10000) was used in 5% non-fat milk in TBST at 4°C overnight. Goat Anti-Mouse/Rabbit IgG-HRP Secondary Antibody (1/10000) was used for 1 hour at room temperature. Western blot analysis of extracts from THP-1(lane 2(20μg), Jurkat (lane 3(20μg) and NIH3T3(lane 4(20μg) using FOXO1A (HY-P80132) Rabbit mAb. Proteins were transferred to a PVDF membrane and blocked with 5% non-fat milk in TBST for 2 hour at room temperature. The primary antibody (1/1000) and Loading control antibody (Beta Actin, HY-P80438, 1/10000) was used in 5% non-fat milk in TBST at 4°C overnight. Goat Anti-Mouse/Rabbit IgG-HRP Secondary Antibody (1/10000) was used for 1 hour at room temperature. Western blot analysis of extracts from THP-1(lane 2(20μg), Jurkat (lane 3(20μg) and NIH3T3(lane 4(20μg) using FOXO1A (HY-P80132) Rabbit mAb. Proteins were transferred to a PVDF membrane and blocked with 5% non-fat milk in TBST for 2 hour at room temperature. The primary antibody (1/1000) and Loading control antibody (Beta Actin, HY-P80438, 1/10000) was used in 5% non-fat milk in TBST at 4°C overnight. Goat Anti-Mouse/Rabbit IgG-HRP Secondary Antibody (1/10000) was used for 1 hour at room temperature.

BODIPY 493/503 purchased from MedChemExpress. Usage Cited in: Nat Commun. 2025 Feb 1;16(1):1241. [Abstract]

MedchemExpress Validation 01

MedchemExpress Validation 03

Western blot analysis of extracts from THP-1(lane 2(20μg), Jurkat (lane 3(20μg) and NIH3T3(lane 4(20μg) using FOXO1A (HY-P80132) Rabbit mAb. Proteins were transferred

MedchemExpress Validation 04

MedchemExpress Validation

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