14 results about "Huntingtin" patented technology

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 provides small molecule drugs and pharmaceutical compositions for the treatment and prevention of diseases related to the formation of certain types of oligomers in a subject. More specifically, the drugs and compositions reduce or prevent the formation of oligomers formed from tau protein, TDP-43, Huntingtin's protein and / or alpha-synuclein. It further provides a method of reducing formation of or disrupting TDP-43, alpha-synuclein, Huntingtin's protein and / or tau protein oligomers in a subject, the method comprising the step of administering to the subject in need thereof a therapeutically effective amount of a pharmaceutical composition.

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.

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).

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.

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 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.