Research graph
References from Gene therapy in amyotrophic lateral sclerosis (ALS). Local targets link to admitted publications; unresolved targets remain external evidence.
Treatment of amyotrophic lateral sclerosis using a gene therapy approach
1995 · External reference
The genetics of amyotrophic lateral sclerosis: current insights
2016 · External reference
Gene therapy for ALS: a review
10.1016/j.ymthe.2021.04.008 · 2021 · External reference
C9orf72-mediated ALS and FTD: multiple pathways to disease
10.1038/s41582-018-0047-2 · 2018 · External reference
Unresolved reference
External reference
Unresolved reference
External reference
Unresolved reference
External reference
Narrative review of diagnosis, management and treatment of dysphagia and sialorrhea in amyotrophic lateral sclerosis
10.1007/s00415-024-12657-x · 2024 · External reference
Nearly 30 years of animal models to study amyotrophic lateral sclerosis: a historical overview and future perspectives
10.3390/ijms222212236 · 2021 · External reference
Wild-type and mutant SOD1 share an aberrant conformation and a common pathogenic pathway in ALS
10.1038/nn.2660 · 2010 · External reference
Amyotrophic lateral sclerosis
10.1056/nejmra1603471 · 2017 · External reference
Gene therapy for ALS-A perspective
10.3390/ijms20184388 · 2019 · External reference
Brain-derived neurotrophic factor attenuates cognitive impairment and motor deficits in a mouse model of Parkinson’s disease
10.1002/brb3.2251 · 2021 · External reference
The potential of the nose-to-brain delivery of PACAP for the treatment of neuronal disease
10.3390/pharmaceutics15082032 · 2023 · External reference
Delivery of AAV-IGF-1 to the CNS extends survival in ALS mice through modification of aberrant glial cell activity
10.1038/mt.2008.60 · 2008 · External reference
Unresolved reference
External reference
TBK1, a prioritized drug repurposing target for amyotrophic lateral sclerosis: evidence from druggable genome Mendelian randomization and pharmacological verification in vitro
10.1186/s12916-024-03314-1 · 2024 · External reference
Prognostic factors and epidemiology of amyotrophic lateral sclerosis in Southeastern United States
10.1016/j.mayocpiqo.2024.07.008 · 2024 · External reference
Gene therapy in amyotrophic lateral sclerosis
10.3390/cells11132066 · 2022 · External reference
Modeling sporadic ALS in iPSC-derived motor neurons identifies a potential therapeutic agent
10.1038/s41591-018-0140-5 · 2018 · External reference
Advances and challenges in gene therapy for neurodegenerative diseases: a systematic review
10.3390/ijms252312485 · 2024 · External reference
Poly(GP) proteins are a useful pharmacodynamic marker for C9ORF72-associated amyotrophic lateral sclerosis
10.1126/scitranslmed.aai7866 · 2017 · External reference
Prospects for gene replacement therapies in amyotrophic lateral sclerosis
10.1038/s41582-022-00751-5 · 2023 · External reference
Amyotrophic lateral sclerosis: an update for 2013 clinical features, pathophysiology, management and therapeutic trials
10.14336/ad.2013.0400295 · 2013 · External reference
Beta2-adrenergic suppression of neuroinflammation in treatment of parkinsonism, with relevance for neurodegenerative and neoplastic disorders
10.3390/biomedicines12081720 · 2024 · External reference
Gain of toxicity from ALS/FTD-linked repeat expansions in C9ORF72 is alleviated by antisense oligonucleotides targeting GGGGCC-containing RNAs
10.1016/j.neuron.2016.04.006 · 2016 · External reference
Improving clinical trial outcomes in amyotrophic lateral sclerosis
10.1038/s41582-020-00434-z · 2021 · External reference
Effects of noninvasive ventilation in amyotrophic lateral sclerosis: the complication of bulbar impairment
10.1177/2514183x20914183 · 2020 · External reference
DNA damage, defective DNA repair, and neurodegeneration in amyotrophic lateral sclerosis
10.3389/fnagi.2022.786420 · 2022 · External reference
Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis
10.1038/s41591-021-01615-z · 2022 · External reference
Pain in amyotrophic lateral sclerosis: a narrative review
10.12701/jyms.2022.00332 · 2022 · External reference
Gene therapy in ALS and SMA: advances, challenges and perspectives
10.3390/ijms24021130 · 2023 · External reference
Fly for ALS: drosophila modeling on the route to amyotrophic lateral sclerosis modifiers
10.1007/s00018-021-03905-8 · 2021 · External reference
Therapeutic targeting of ALS pathways: refocusing an incomplete picture
10.1002/acn3.51887 · 2023 · External reference
Amyotrophic lateral sclerosis: a clinical review
10.1111/ene.14393 · 2020 · External reference
Translating the ALS genetic revolution into therapies: a review
10.1007/s11940-024-00781-y · 2024 · External reference
Amyotrophic lateral sclerosis estimated prevalence cases from 2022 to 2030, data from the national ALS registry
10.1080/21678421.2024.2447919 · 2025 · External reference
Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND)
10.1080/14660820310002601 · 2003 · External reference
Trial of antisense oligonucleotide tofersen for SOD1 ALS
10.1056/nejmoa2204705 · 2022 · External reference
SOD1 suppression with adeno-associated virus and MicroRNA in familial ALS
10.1056/nejmoa2005056 · 2020 · External reference
Pathogenesis of FUS-associated ALS and FTD: insights from rodent models
10.1186/s40478-016-0358-8 · 2016 · External reference
Trial of sodium phenylbutyrate–taurursodiol for amyotrophic lateral sclerosis
10.1056/nejmoa1916945 · 2020 · External reference
TDP-43: a key therapeutic target beyond amyotrophic lateral sclerosis
10.1021/acschemneuro.9b00026 · 2019 · External reference
Simple animal models for amyotrophic lateral sclerosis drug discovery
10.1080/17460441.2016.1196183 · 2016 · External reference
Neuronal dysfunction caused by FUSR521G promotes ALS-associated phenotypes that are attenuated by NF-κB inhibition
10.1186/s40478-023-01671-1 · 2023 · External reference
Emerging mechanisms of molecular pathology in ALS
10.1172/jci71601 · 2015 · External reference
Design and statistical innovations in a platform trial for amyotrophic lateral sclerosis
10.1002/ana.26714 · 2023 · External reference
State of play in amyotrophic lateral sclerosis genetics
10.1038/nn.3584 · 2014 · External reference
Janus kinase inhibitors are potential therapeutics for amyotrophic lateral sclerosis
10.1186/s40035-023-00380-y · 2023 · External reference
Clinical efficacy of edaravone for the treatment of amyotrophic lateral sclerosis
10.1080/14656566.2017.1319937 · 2017 · External reference
Unresolved reference
External reference
Cell therapy in ALS: an update on preclinical and clinical studies
10.1016/j.brainresbull.2023.01.008 · 2023 · External reference
Antisense oligonucleotide therapy for neurodegenerative disease
10.1172/jci25424 · 2006 · External reference
Genetics of amyotrophic lateral sclerosis: seeking therapeutic targets in the era of gene therapy
10.1038/s10038-022-01055-8 · 2023 · External reference
Decoding ALS: from genes to mechanism
10.1038/nature20413 · 2016 · External reference
Resting state fMRI analysis of pseudobulbar affect in amyotrophic lateral sclerosis (ALS): motor dysfunction of emotional expression
10.1007/s11682-022-00744-4 · 2023 · External reference
Safety, tolerability, and pharmacokinetics of antisense oligonucleotide BIIB078 in adults with C9orf72-associated amyotrophic lateral sclerosis: a phase 1, randomised, double blinded, placebo-controlled, multiple ascending dose study
10.1016/s1474-4422(24)00216-3 · 2024 · External reference
Amyotrophic lateral sclerosis
10.1016/s0140-6736(17)31287-4 · 2017 · External reference
ATAXIN-2 intermediate-length polyglutamine expansions elicit ALS-associated metabolic and immune phenotypes
10.1038/s41467-024-51676-0 · 2024 · External reference
Omics-based exploration and functional validation of neurotrophic factors and histamine as therapeutic targets in ALS
10.1016/j.arr.2020.101121 · 2020 · External reference
The heritability of amyotrophic lateral sclerosis in a clinically ascertained United States research registry
10.1371/journal.pone.0027985 · 2011 · External reference
Genetically modified large animal models for investigating neurodegenerative diseases
10.1186/s13578-021-00729-8 · 2021 · External reference
Pathological insights from amyotrophic lateral sclerosis animal models: comparisons, limitations, and challenges
10.1186/s40035-023-00377-7 · 2023 · External reference
Therapeutic targeting of ALS pathways: refocusing an incomplete picture
10.1002/acn3.51887 · ExternalCitation · doi-reference
Design and statistical innovations in a platform trial for amyotrophic lateral sclerosis
10.1002/ana.26714 · ExternalCitation · doi-reference
Brain-derived neurotrophic factor attenuates cognitive impairment and motor deficits in a mouse model of Parkinson’s disease
10.1002/brb3.2251 · ExternalCitation · doi-reference
Fly for ALS: drosophila modeling on the route to amyotrophic lateral sclerosis modifiers
10.1007/s00018-021-03905-8 · ExternalCitation · doi-reference
Narrative review of diagnosis, management and treatment of dysphagia and sialorrhea in amyotrophic lateral sclerosis
10.1007/s00415-024-12657-x · ExternalCitation · doi-reference
Resting state fMRI analysis of pseudobulbar affect in amyotrophic lateral sclerosis (ALS): motor dysfunction of emotional expression
10.1007/s11682-022-00744-4 · ExternalCitation · doi-reference
Translating the ALS genetic revolution into therapies: a review
10.1007/s11940-024-00781-y · ExternalCitation · doi-reference
Omics-based exploration and functional validation of neurotrophic factors and histamine as therapeutic targets in ALS
10.1016/j.arr.2020.101121 · ExternalCitation · doi-reference
Cell therapy in ALS: an update on preclinical and clinical studies
10.1016/j.brainresbull.2023.01.008 · ExternalCitation · doi-reference
Prognostic factors and epidemiology of amyotrophic lateral sclerosis in Southeastern United States
10.1016/j.mayocpiqo.2024.07.008 · ExternalCitation · doi-reference
Gain of toxicity from ALS/FTD-linked repeat expansions in C9ORF72 is alleviated by antisense oligonucleotides targeting GGGGCC-containing RNAs
10.1016/j.neuron.2016.04.006 · ExternalCitation · doi-reference
Gene therapy for ALS: a review
10.1016/j.ymthe.2021.04.008 · ExternalCitation · doi-reference
Amyotrophic lateral sclerosis
10.1016/s0140-6736(17)31287-4 · ExternalCitation · doi-reference
Safety, tolerability, and pharmacokinetics of antisense oligonucleotide BIIB078 in adults with C9orf72-associated amyotrophic lateral sclerosis: a phase 1, randomised, double blinded, placebo-controlled, multiple ascending dose study
10.1016/s1474-4422(24)00216-3 · ExternalCitation · doi-reference
TDP-43: a key therapeutic target beyond amyotrophic lateral sclerosis
10.1021/acschemneuro.9b00026 · ExternalCitation · doi-reference
Delivery of AAV-IGF-1 to the CNS extends survival in ALS mice through modification of aberrant glial cell activity
10.1038/mt.2008.60 · ExternalCitation · doi-reference
Decoding ALS: from genes to mechanism
10.1038/nature20413 · ExternalCitation · doi-reference
Wild-type and mutant SOD1 share an aberrant conformation and a common pathogenic pathway in ALS
10.1038/nn.2660 · ExternalCitation · doi-reference
State of play in amyotrophic lateral sclerosis genetics
10.1038/nn.3584 · ExternalCitation · doi-reference
Genetics of amyotrophic lateral sclerosis: seeking therapeutic targets in the era of gene therapy
10.1038/s10038-022-01055-8 · ExternalCitation · doi-reference
ATAXIN-2 intermediate-length polyglutamine expansions elicit ALS-associated metabolic and immune phenotypes
10.1038/s41467-024-51676-0 · ExternalCitation · doi-reference
C9orf72-mediated ALS and FTD: multiple pathways to disease
10.1038/s41582-018-0047-2 · ExternalCitation · doi-reference
Improving clinical trial outcomes in amyotrophic lateral sclerosis
10.1038/s41582-020-00434-z · ExternalCitation · doi-reference
Prospects for gene replacement therapies in amyotrophic lateral sclerosis
10.1038/s41582-022-00751-5 · ExternalCitation · doi-reference
Modeling sporadic ALS in iPSC-derived motor neurons identifies a potential therapeutic agent
10.1038/s41591-018-0140-5 · ExternalCitation · doi-reference
Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis
10.1038/s41591-021-01615-z · ExternalCitation · doi-reference
Trial of sodium phenylbutyrate–taurursodiol for amyotrophic lateral sclerosis
10.1056/nejmoa1916945 · ExternalCitation · doi-reference
SOD1 suppression with adeno-associated virus and MicroRNA in familial ALS
10.1056/nejmoa2005056 · ExternalCitation · doi-reference
Trial of antisense oligonucleotide tofersen for SOD1 ALS
10.1056/nejmoa2204705 · ExternalCitation · doi-reference
Amyotrophic lateral sclerosis
10.1056/nejmra1603471 · ExternalCitation · doi-reference
Clinical efficacy of edaravone for the treatment of amyotrophic lateral sclerosis
10.1080/14656566.2017.1319937 · ExternalCitation · doi-reference
Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND)
10.1080/14660820310002601 · ExternalCitation · doi-reference
Simple animal models for amyotrophic lateral sclerosis drug discovery
10.1080/17460441.2016.1196183 · ExternalCitation · doi-reference
Amyotrophic lateral sclerosis estimated prevalence cases from 2022 to 2030, data from the national ALS registry
10.1080/21678421.2024.2447919 · ExternalCitation · doi-reference
Amyotrophic lateral sclerosis: a clinical review
10.1111/ene.14393 · ExternalCitation · doi-reference
Poly(GP) proteins are a useful pharmacodynamic marker for C9ORF72-associated amyotrophic lateral sclerosis
10.1126/scitranslmed.aai7866 · ExternalCitation · doi-reference
Antisense oligonucleotide therapy for neurodegenerative disease
10.1172/jci25424 · ExternalCitation · doi-reference
Emerging mechanisms of molecular pathology in ALS
10.1172/jci71601 · ExternalCitation · doi-reference
Effects of noninvasive ventilation in amyotrophic lateral sclerosis: the complication of bulbar impairment
10.1177/2514183x20914183 · ExternalCitation · doi-reference
TBK1, a prioritized drug repurposing target for amyotrophic lateral sclerosis: evidence from druggable genome Mendelian randomization and pharmacological verification in vitro
10.1186/s12916-024-03314-1 · ExternalCitation · doi-reference
Genetically modified large animal models for investigating neurodegenerative diseases
10.1186/s13578-021-00729-8 · ExternalCitation · doi-reference
Pathological insights from amyotrophic lateral sclerosis animal models: comparisons, limitations, and challenges
10.1186/s40035-023-00377-7 · ExternalCitation · doi-reference
Janus kinase inhibitors are potential therapeutics for amyotrophic lateral sclerosis
10.1186/s40035-023-00380-y · ExternalCitation · doi-reference
Pathogenesis of FUS-associated ALS and FTD: insights from rodent models
10.1186/s40478-016-0358-8 · ExternalCitation · doi-reference
Neuronal dysfunction caused by FUSR521G promotes ALS-associated phenotypes that are attenuated by NF-κB inhibition
10.1186/s40478-023-01671-1 · ExternalCitation · doi-reference
Pain in amyotrophic lateral sclerosis: a narrative review
10.12701/jyms.2022.00332 · ExternalCitation · doi-reference
The heritability of amyotrophic lateral sclerosis in a clinically ascertained United States research registry
10.1371/journal.pone.0027985 · ExternalCitation · doi-reference
Amyotrophic lateral sclerosis: an update for 2013 clinical features, pathophysiology, management and therapeutic trials
10.14336/ad.2013.0400295 · ExternalCitation · doi-reference
DNA damage, defective DNA repair, and neurodegeneration in amyotrophic lateral sclerosis
10.3389/fnagi.2022.786420 · ExternalCitation · doi-reference
Beta2-adrenergic suppression of neuroinflammation in treatment of parkinsonism, with relevance for neurodegenerative and neoplastic disorders
10.3390/biomedicines12081720 · ExternalCitation · doi-reference
Gene therapy in amyotrophic lateral sclerosis
10.3390/cells11132066 · ExternalCitation · doi-reference
Gene therapy for ALS-A perspective
10.3390/ijms20184388 · ExternalCitation · doi-reference
Nearly 30 years of animal models to study amyotrophic lateral sclerosis: a historical overview and future perspectives
10.3390/ijms222212236 · ExternalCitation · doi-reference
Gene therapy in ALS and SMA: advances, challenges and perspectives
10.3390/ijms24021130 · ExternalCitation · doi-reference
Advances and challenges in gene therapy for neurodegenerative diseases: a systematic review
10.3390/ijms252312485 · ExternalCitation · doi-reference
The potential of the nose-to-brain delivery of PACAP for the treatment of neuronal disease
10.3390/pharmaceutics15082032 · ExternalCitation · doi-reference