Search Results - amyotrophic+lateral+sclerosis

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Antisense oligonucleotide therapy and biomarker assay for C9ORF72 hexanucleotide expansion disorders
Therapy to Treat and Assay to Monitor Neurodegenerative Disease  JHU REF: [C12195]Invention novelty: This technology is the use of antisense oligonucleotides (ASO) as a therapeutic for specific neurodegenerative diseases. The technology could also be used in development of a biomarker assay to monitor the efficacy of the ASO therapy.   Value Proposition: Current...
Published: 5/9/2024   |   Inventor(s): Christopher Donnelly, C. Bennett, Susan Freier, Rita Sattler, Jeffrey Rothstein
Keywords(s): Amyotrophic Lateral Sclerosis, Assay, Biologics, Clinical Diagnostics, CNS and Neurological Disorders, Dementia, Disease Indication, In Vitro Diagnostics, Neurodegeneration, Nucleic Acid, Therapeutic Matter, Therapeutic Substance, Therapeutics
Category(s): Clinical and Disease Specializations, Technology Classifications > Therapeutic Modalities > Biologics, Technology Classifications > Therapeutic Modalities > Gene Therapies, Technology Classifications > Diagnostics > In Vitro Diagnostics, Technology Classifications > Research Tools > Assays, Technology Classifications > Diagnostics, Clinical and Disease Specializations > Neurology, Technology Classifications > Research Tools, Technology Classifications > Therapeutic Modalities, Clinical and Disease Specializations > Neurology > Amyotrophic Lateral Sclerosis, Clinical and Disease Specializations > Neurology > Dementia, Clinical and Disease Specializations > Neurology > Neurodegeneration
Tonabersat as a Neuroprotective Compound for Amyotrophic Lateral Sclerosis
Unmet NeedAmytrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disease that affects motor neurons, and results in muscle atrophy and loss of motor control. ALS affects approximately 17,000 people in the United States and 450,000 worldwide at any given time, and the average life expectancy from diagnosis to mortality is between 2-5 years....
Published: 5/9/2024   |   Inventor(s): Nicholas Maragakis
Keywords(s): Amyotrophic Lateral Sclerosis, Antagonists/Inhibitors, CNS and Neurological Disorders, Disease Indication, Non-novel, Predicted Novelty, Repurposed, Small Molecules, Therapeutic Matter, Therapeutic Substance, Therapeutics
Category(s): Technology Classifications > Therapeutic Modalities > Small Molecules, Technology Classifications > Therapeutic Modalities > Repurposed Drugs, Clinical and Disease Specializations > Neurology > Amyotrophic Lateral Sclerosis
Pharmacological Intervention of the Arachidonic Acid Pathway to Cure Amyotrophic Lateral Sclerosis
Unmet NeedAmyotrophic Lateral Sclerosis (ALS) affects as many as 30,000 people in the United States alone, with 5,000 new cases diagnosed every year. ALS is a fatal neuromuscular disease that is characterized by a progressive degeneration of the motor nerve cells and spinal cord. There are only two drugs approved by FDA for the treatment of ALS, and...
Published: 5/9/2024   |   Inventor(s): Gabsang Lee, Hyungjin Eoh, Hojae Lee, Thomas Lloyd, Nicholas Maragakis
Keywords(s): Amyotrophic Lateral Sclerosis, CNS and Neurological Disorders, Disease Indication, Natural compounds, Non-novel, Predicted Novelty, Target, Therapeutic Matter, Therapeutics
Category(s): Clinical and Disease Specializations > Neurology > Amyotrophic Lateral Sclerosis, Technology Classifications > Therapeutic Modalities > Targets, Technology Classifications > Therapeutic Modalities > Repurposed Drugs
Stimulation of Dendrite Growth and Spine Formation by Norrin and LGR6: A Target for Therapy in CNS Injury and Degeneration
Unmet NeedAstroglial dysfunction is associated with the development of numerous central nervous system (CNS) diseases, including amyotrophic lateral sclerosis (ALS), Parkinson’s disease (PD), and Huntington’s disease (HD). Astroglia are the homeostatic cells of the central nervous system and play key roles in the development and support of neuronal...
Published: 5/9/2024   |   Inventor(s): Sean Miller, Jeffrey Rothstein
Keywords(s): Alzheimer's Disease, Amyotrophic Lateral Sclerosis, Biologics, CNS and Neurological Disorders, Dementia, Disease Indication, Huntington's Disease, Parkinson's Disease, Protein, Target, Therapeutic Matter, Therapeutic Substance, Therapeutics
Category(s): Clinical and Disease Specializations > Neurology > Neurodegeneration, Clinical and Disease Specializations > Neurology > Amyotrophic Lateral Sclerosis, Clinical and Disease Specializations > Neurology > Dementia, Technology Classifications > Therapeutic Modalities > Proteins, Clinical and Disease Specializations > Neurology > Alzheimer's Disease, Technology Classifications > Therapeutic Modalities > Targets, Clinical and Disease Specializations > Neurology > Huntington's Disease, Clinical and Disease Specializations > Neurology > Parkinson’s Disease
Target Validation of Splicing Repression, a Major Function of TDP-43 in the Motor Neuron
Unmet NeedAmyotrophic lateral sclerosis (ALS) is a rare but debilitating disease characterized by the death of motor neurons that eventually leads to difficulty moving, speaking, swallowing, and breathing. The fundamental cause of ALS is unknown and as a result treatment options are primarily restricted to physical therapy to help prolong muscle strength....
Published: 5/9/2024   |   Inventor(s): Philip Wong, Aneesh Donde, Jonathan Ling, Liam Chen, Mingkuan Sun
Keywords(s): Amyotrophic Lateral Sclerosis, CNS and Neurological Disorders, Disease Indication, Target, Therapeutic Matter, Therapeutics
Category(s): Clinical and Disease Specializations > Neurology > Amyotrophic Lateral Sclerosis, Technology Classifications > Therapeutic Modalities > Targets
TDP-43 Suppression of Non-conserved Cryptic Exons is Compromised in Human Neurodegenerative Diseases
C13621: Suppression of Non-conserved Cryptic Exons is Compromised in Human Neurodegenerative DiseasesAbstractA novel mechanism underlying the suppression of non conserved cryptic exons. This mechanism is essential to maintain intron integrity, and is compromised in ALS-FTD, indicating that loss of function underlies pathogenesis of disease. Publication...
Published: 5/9/2024   |   Inventor(s): Philip Wong, Jonathan Ling
Keywords(s): Alzheimer's Disease, Amyotrophic Lateral Sclerosis, Basic Research Biomarker, CNS and Neurological Disorders, Dementia, Discovery/Research Tools, Disease Indication, In Vivo Research Tool, Knock Out Mouse Model, Mouse Model
Category(s): Clinical and Disease Specializations, Clinical and Disease Specializations > Neurology, Technology Classifications > Research Tools > Animal Models, Technology Classifications > Research Tools, Clinical and Disease Specializations > Neurology > Alzheimer's Disease, Clinical and Disease Specializations > Neurology > Amyotrophic Lateral Sclerosis, Clinical and Disease Specializations > Neurology > Dementia
NG2-cDTa mice
C11678: Transgenic Mouse Line with Inducible Diphtheria Toxin GeneNovelty: A novel line of transgenic mice expressing stable inducible diphtheria toxin gene under the control of an NG-2 promoter. This mouse line can be used in neuroscience studies to induce a model state of chronic demyelination.Value Proposition: Neuronal cell studies are an important...
Published: 5/9/2024   |   Inventor(s): Shin Kang, Dwight Bergles
Keywords(s): Amyotrophic Lateral Sclerosis, Amyotrophic Laterial Sclerosis (ALS), Basic Research Biomarker, CNS and Neurological Disorders, Discovery/Research Tools, Disease Indication, In Vivo Research Tool, Knock In Mouse Model, Motor Neuron Diseases, Mouse Model, Mouse Model Xenograft, Neurodegeneration
Category(s): Clinical and Disease Specializations, Clinical and Disease Specializations > Autoimmunity, Clinical and Disease Specializations > Neurology > Amyotrophic Lateral Sclerosis, Clinical and Disease Specializations > Neurology > Motor Neuron Diseases, Clinical and Disease Specializations > Neurology > Neurodegeneration, Clinical and Disease Specializations > Rare Diseases, Technology Classifications > Research Tools > Animal Models, Clinical and Disease Specializations > Neurology, Technology Classifications > Research Tools, Clinical and Disease Specializations > Immunology
Germline and Conditional Deletion of Tardbp
C11168: A Gene Linked to Obesity, and Alters Body Fat Metabolism Value Proposition: • Identifies novel therapeutic target to control obesity and diabetes. • Helps to understand incurable diseases: frontal temporal dementia and amyotrophic lateral sclerosis. Technical Details: JHU scientists have developed conditional knockout mice...
Published: 5/9/2024   |   Inventor(s): Donald Price, Po-Min Chiang, Philip Wong
Keywords(s): Amyotrophic Lateral Sclerosis, Basic Research Biomarker, Biomarker, Cell Model, Clinical Diagnostics, CNS and Neurological Disorders, Dementia, Diabetes, Discovery/Research Tools, Disease Indication, In Vitro Diagnostics, In Vitro Research Tool, In Vivo Research Tool, Knock Out Mouse Model, Mechanism-of-action Biomarker, Metabolic Disorders, Mouse Model, Neurodegeneration, Obesity, Stem Cells
Category(s): Clinical and Disease Specializations, Clinical and Disease Specializations > Diabetes, Clinical and Disease Specializations > Metabolic Diseases, Clinical and Disease Specializations > Metabolic Diseases > Obesity, Clinical and Disease Specializations > Neurology > Amyotrophic Lateral Sclerosis, Clinical and Disease Specializations > Neurology > Dementia, Clinical and Disease Specializations > Neurology > Neurodegeneration, Technology Classifications > Diagnostics > Biomarkers, Technology Classifications > Diagnostics > In Vitro Diagnostics, Technology Classifications > Therapeutic Modalities > Targets, Technology Classifications > Diagnostics, Clinical and Disease Specializations > Neurology, Technology Classifications > Therapeutic Modalities, Technology Classifications > Research Tools > Animal Models, Technology Classifications > Research Tools > Stem Cells
Hybridoma Clone G6
C10552: Hybridoma Secreting Anti-Excitatory Amino Acid Transporter 2 (EAAT2) AntibodyNovelty: Hybridoma cell line that secretes monoclonal antibody to GLT1/EAAT2 protein.Value Proposition: Mutations or decreased expression of the GLT1/EAAT2 protein are associated with amyotrophic lateral sclerosis and thus the detection of protein can be used as an...
Published: 5/9/2024   |   Inventor(s): Jeffrey Rothstein
Keywords(s): Amyotrophic Lateral Sclerosis, Basic Research Biomarker, Cell Lines, Cell Model, CNS and Neurological Disorders, Discovery/Research Tools, Disease Indication, Human Cell Lines, Hybridomas, In Vitro Research Tool, Translational Research Biomarker
Category(s): Technology Classifications > Research Tools > Antibodies, Technology Classifications > Research Tools > Cell Lines, Technology Classifications > Research Tools, Clinical and Disease Specializations > Rare Diseases, Clinical and Disease Specializations > Neurology > Amyotrophic Lateral Sclerosis, Technology Classifications > Research Tools > Human Cell Lines
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