21 results about "Huntingtons chorea" patented technology

Aspects of the disclosure relate to compositions and methods useful for treating Huntington's disease. In some embodiments, the disclosure provides interfering nucleic acids (e.g., artificial miRNAs) targeting the huntingtin gene (HTT) and methods of treating Huntington's disease using the same.

The present invention relates to the field of diagnostics. Specifically, it relates to a method for assessing Huntington's disease of the pre-manifest stage in a subject comprising the steps of determining at least one performance parameter from a dataset of fine motoric measurements from said subject, comparing the determined at least one performance parameter to a reference, and assessing Huntington's disease of the pre-manifest stage in the subject based on said comparison. Yet, the invention contemplates a device and a system for carrying out the aforementioned methods and the use of such device or system for assessing Huntington's disease of the pre-manifest stage in the subject.

The present invention addresses the problem of providing an agent for preventing or treating amyotrophic lateral sclerosis (ALS), Parkinson's disease (PD), Huntington's disease (HD), spinocerebellar ataxia (SCA), aging-related degenerative or neurological disease, brain aging, or diseases associated with brain aging, as well as a more stable antibody that exhibits an effect of preventing or treating these diseases, Alzheimer's disease (AD), or frontotemporal lobar degeneration (FTLD). A human monoclonal antibody that specifically binds to human HMGB1, wherein the human monoclonal antibody (anti-human HMGB1 antibody) comprises a heavy chain CDR1, heavy chain CDR2, and heavy chain CDR3 each consisting of a specific amino acid sequence and a light chain CDR1, light chain CDR2, and light chain CDR3 each consisting of a specific amino acid sequence, is used as an agent for preventing or treating ALS, PD, HD, SCA, aging-related degenerative or neurological disease, brain aging, or diseases associated with brain aging. An antibody in which the light chain complementarity determining region (CDR) 3 of the anti-human HMGB1 antibody has been modified is used.

The present description relates to a tablet formulation of 2-[3-(2,2,6,6-tetramethylpiperidin-4-yl)-3H-[1,2,3]triazolo[4,5-c]pyridazin-6-yl]-5-(2H-1,2,3-triazol-2-yl)phenol, a compound for use in treating Huntington's disease, and a method of making the same.

The invention relates to compounds, compositions, and methods for derepressing RE1 silencing transcription factor (REST) target genes are provided. In particular, a peptide having the sequence TEDLEPPEPPLPKEN (SEQ. ID NO: 1) and EDLEPPEPPLPK (SEQ. ID NO: 15), or the reversed sequences made of D-amino acids (retro inverted, RI) nekplppeppeldet (SEQ ID NO: 16) and kplppeppelde (SEQ ID NO: 17), are disclosed for inhibiting REST activity. The peptides are useful to treat, prevent, or ameliorate conditions such as traumatic brain injury, epilepsy, dementia, Huntington's Disease (HD), chronic pain, brain cancer (including glioblastoma multiforme), pancreatic cancer; diabetes, and peripheral nerve injury

Aspects of the present disclosure relate to compositions and methods useful for treating Huntington's disease. In some embodiments, the present disclosure provides interfering nucleic acids (e.g., artificial miRNAs) targeting the Huntingtin gene (HTT) and methods of using the same to treat Huntington's disease. Accordingly, in some aspects, the present disclosure provides an isolated nucleic acid comprising or encoding the sequence set forth in any one of SEQ ID NO: 1-SEQ ID NO: 22.

The present invention relates to the field of biotechnology and the use of chemically modified antisense oligonucleotides (AONs) for the deamination of one or more target adenosines in the transcript of (mutant) human Huntingtin (HTT) for use in the treatment, prevention, or delay of Huntington's disease (HD). In particular, the target adenosines are in the GAU codon coding for aspartic acid at position 572 that is part of a caspase-1 proteolytic cleavage site in the human HTT protein, and / or in the GAC codon coding for aspartic acid at position 586 that is part of a caspase-6 proteolytic cleavage site in the human HTT protein.

Terpenoid derivatives for use in the medicinal applications and compositions containing these derivatives. This new generation of compounds possesses selective anti-neurodegenerative properties on neuronal cells and tissues and can be particularly used in the treatment and prophylaxis of neurodegenerative disease, particularly in the treatment and prophylaxis of Parkinson's and Huntington's disease.

The present disclosure provides the use of compounds of Formulae (I), (II), (III), (III-A), and (III-B) (e.g., metformin) in treating a neurological disease associated with repeat expansions and / or RAN protein accumulation, reducing the level of one or more repeat associated non-ATG (RAN) proteins, and reducing the accumulation of RAN proteins in a subject and / or biological sample. Also provided is the use of compounds of Formulae (I), (II), (III), (III-A), and (III-B) (e.g., metformin) in inhibiting RAN protein translation in a subject and in a biological sample (e.g., cells, tissue). Also provided in the present disclosure are pharmaceutical compositions, kits, and uses of compounds of Formulae (I), (II), (III), (III-A), and (III-B) (e.g., metformin) for treating diseases associated with repeat expansions. Exemplary diseases associated with repeat expansions include, but are not limited to, C9ORFf72 amyotrophic lateral sclerosis (ALS), or C9ORFf72 frontotemporal dementia; myotonic dystrophy type 1 (DM1) and myotonic dystrophy type 2 (DM2); spinocerebellar ataxia; Huntington's disease; Fragile X Tremor Ataxia Syndrome (FXTAS); and Fragile XE syndrome (FRAXE).

The invention relates to the expression of two molecules in viral vectors AAV / IGF2-HA and AAV / IGF2, the associated method and use thereof in the amelioration of protein misfolding-related diseases, such as Huntington's disease, as presented in the in vivo models in FIGS. 11 / 19.

The present disclosure relates to a transcription modulator molecule having a first end, a second end, and an oligomeric backbone, and methods for treating Huntington's disease (HD).

The present disclosure relates to compositions comprising trehalose and methods of using same for the treatment of Huntington's disease.

Described herein is 6-(6-(((1R,2R,3S,5S)-2-fluoro-9-azabicyclo[3.3.1]nonan-3-yl)(methyl)amino)pyridazin-3-yl)-2-methylbenzo[d]oxolan-5-ol (Structure B), a small molecule splicing modulator (SMSM) of mRNA encoded by a gene, such as a pre-mRNA, for use in the treatment of neurological and neurodegenerative diseases, such as Huntington's disease and brain cancer.

Described herein is a small molecule splicing modulator compound that modulates splicing of mRNA, such as pre-mRNA, encoded by genes, and methods of use of the small molecule splicing modulator compounds for modulating splicing and treating diseases and conditions.

The present invention provides KDM5 inhibitor. The compound disclosed herein represented by the general formula (I) : wherein all symbols have the same meanings as the definitions described in the specification; or a salt thereof is useful as a prophylactic and / or therapeutic agent for cancer, Huntington's disease, Alzheimer's disease and the like.

The present invention provides compositions and methods for inhibiting the autoimmune component of neurodegenerative diseases and thereby providing a therapeutic effect to patients suffering from such diseases. The compositions and methods comprise immunosuppressive moieties, such as regulatory T cells (Tregs) and proteins expressed by Tregs coupled to a chimeric antigen receptor or that specifically bind to one or more glial cell markers. Therapeutically effective doses of the compounds for treating neurodegenerative diseases, including progressive supranuclear palsy (PSP), Parkinson's disease (PD), Alzheimer's disease, Huntington's disease, amyotrophic lateral sclerosis (ALS), chronic traumatic encephalopathy (CTE), and prion diseases, are disclosed.

The application discloses an engineered intracellular antibody, which is fused by a Nef protein sequence of HIV-1 and an intracellular antibody sequence for targeting and degrading mutant huntingtin protein, and has simple structure and is easy to artificially synthesize. The application further discloses an exosome carrying the intracellular antibody, wherein a rabies virus glycoprotein peptide segment RVG is expressed on the membrane of the exosome, so that the exosome can specifically target nerve cells, and has high stability, low immunogenicity, and high penetration, and can be injected intravenously and cut to target and efficiently remove the existing mutant huntingtin protein and its aggregates in the brain of a patient, solve the off-target problem of gene therapy, and have important significance for removing the existing mutant huntingtin protein and its aggregates in the brain of a patient with late Huntington's disease.

The subject invention pertains to compositions comprising Poly(A) RNA polymerase D5 (PAPD5) small molecule inhibitors and methods of using said compositions to treat Huntington's Disease (HD). The PAPD5 small molecule inhibitor is, for example, BCH001 and RG7834. The PAPD5 small molecule inhibitor can mitigate the neuronal defects and cell death in HD.

The application discloses application of a gamma-aminobutyric acid derivative in treatment of TDP-43 proteinopathy, and discloses application of a gamma-aminobutyric acid derivative or an enantiomer thereof or a pharmaceutically acceptable salt thereof in preparation of a medicine for preventing and / or treating a TDP-43 abnormal aggregation related disease, wherein the TDP-43 abnormal aggregation related disease is amyotrophic lateral sclerosis, sporadic inclusion body myositis, Parkinson's disease, Alzheimer's disease, frontotemporal dementia and Huntington's disease.