19 results about "Amyloid Protein Precursor" patented technology

An L-form ribonucleic acid (L-RNA) aptamer-antisense oligonucleotide (ASO) conjugate comprise the L-RNA aptamer, which comprises a ribonucleic acid sequence. The ASO comprises a deoxyribonucleic acid sequence selected from a group. A method of imaging amyloid precursor protein (APP) rG4 in a cell comprising transfecting the cell with a messenger RNA of APP, permeating the cell, contacting the permeated cell with a cyanine3 (Cy3) labeled APP nucleic acid probe and the L-RNA aptamer-ASO conjugate, and subjecting the product to fluorescence microscopy analysis to produce an image of the APP rG4 in the cell.

Described herein are methods for identifying and measuring one or more non-classical variant(s) of amyloid precursor protein (APP) gene. Provided herein are methods for diagnosing and treating an individual having or suspected of having Alzheimer's disease following identification of an expression profile or an activity profile of the one or more non-classical variant(s).

The present invention provides siRNA, peptide oligonucleotide drugs, and their applications for suppressing the expression of the amyloid precursor protein (APP) gene in human cells. The siRNA exhibits potent activity in inhibiting APP expression. Through appropriate modifications, its ability to silence the target is enhanced while reducing off-target activity. The described siRNA and its conjugates hold promise for clinical application in the prevention and treatment of diseases associated with the APP target, including cerebral amyloid angiopathy (CAA), early-onset familial Alzheimer's disease (EOFAD), or Alzheimer's disease (AD).

In one aspect, methods of diagnosing a subject as having Alzheimer's disease and prognosing a subject as being at risk of progressing to Alzheimer's disease are provided. In some embodiments, the method comprises determining one or more of the level of expression of rhotekin 2 (RTKN2), the level of expression of microtubule-associated Ser / Thr kinase 4 (MAST4), the level of binding of forkhead box O1 (FOXO1) to the RTKN2 promoter, and the level of binding of amyloid precursor protein (APP) to the MAST4 promoter in a sample from the subject.

This disclosure pertains to the field of biomedicine, specifically relating to double-stranded oligonucleotides targeting the APP gene and their applications. Specifically, it provides double-stranded oligonucleotide agents or their salts, conjugates, or compositions for inhibiting amyloid precursor protein (APP) expression, wherein the double-stranded oligonucleotide agent comprises a sense strand and an antisense strand forming a double-stranded region; wherein the antisense strand sequence comprises at least 15 consecutive nucleotides of any of the sequences shown in SEQ ID NO:1-154 with a difference of no more than 3 nucleotides, and / or the sense strand sequence comprises at least 15 consecutive nucleotides of any of the sequences shown in SEQ ID NO:155-308 with a difference of no more than 3 nucleotides. The double-stranded oligonucleotide agent or its salt for inhibiting APP expression disclosed in this application can significantly inhibit APP expression and can be used for the prevention and / or treatment of diseases or conditions mediated by the APP gene and / or associated with protein amyloidosis.

An RNAi agent targeting an amyloid precursor protein, such as a double-stranded small interfering RNA (siRNA) agent. A method for inhibiting the expression of the APP gene by using the RNAi agent and a method for preventing and treating APP-related diseases, such as cerebral amyloid angiopathy (CAA) or Alzheimer's disease (AD), including early-onset familial Alzheimer's disease (EOFAD). The siRNA significantly inhibits the expression level of the APP gene and has a long-lasting drug effect.

The present invention provides a medicine for treating a neurodegenerative disease, which employs LEFTY2 (Left-Right Determination Factor 2) generated by co-culturing mesenchymal stem cells of mammals with nerve cells having mutations in the APP (Amyloid precursor protein) gene, or a specific protein. The LEFTY2 has an effect of inhibiting Beta amyloid and a hyperphosphorylated neuronal microtubule-associated protein (Tau protein), without affecting the development of nerve cells and having the ability to promote the growth of the nerve cells; and the two types of proteins have a crucial impact on the neurodegenerative diseases.

The present application relates to a peptide having inhibitory activity against amyloid precursor protein and uses thereof, and provides a peptide consisting of any one of the amino acid sequences set forth in SEQ ID NOs: 1 to 8 and SEQ ID NOs: 20 to 23, and a pharmaceutical composition for preventing or treating degenerative neurological diseases comprising the peptide as an active ingredient.

Described herein are CRISPR / Cas9 constructs designed for the C-terminal truncation of human amyloid precursor protein (APP) as well as methods of making and using such a construct.

The invention relates to the field of human genetics, more specifically to treatments for a disease or condition associated with an abnormal processing of the Amyloid Precursor Protein (APP), preferably familiar Alzheimer disease (FAD). The invention in particular relates to antisense oligonucleotides (AON's) that can be used for treating such diseases or conditions.

There are disclosed therapies and preventions of prion protein complex infections. The transcription of the amyloid precursor protein gene and PrP gene and the RNA transcript are the rate-limiting steps and are most susceptible for blockage and control of the process of amyloid protein formation and PrPsc formation. Thus, therapies and prevention regimes for prion protein complex infections interrupt this process at the level of DNA transcription to RNA, RNA transport to the mitochondrion for protein synthesis and deposition in the cerebral cortex neurons.

The present invention relates to the use of mitoxantrone hydrochloride as a therapeutic drug for the treatment of neurodegenerative disorder in a subject, wherein the mitoxantrone hydrochloride is an inhibitor of β-amyloid precursor protein (APP) and leucine-rich repeat kinase 2 (LRRK2), capable of inhibiting APP and LRRK2 protein levels in the subject.

The invention belongs to the technical field of biological medicine, and relates to a method for preparing PAPA / siRNA nanoparticles by adopting a delivery carrier PAH-AM-PEG-ApoE (159-167) 2 (PAPA) modified by apolipoprotein E (ApoE) peptide through an electrostatic binding self-assembly method by designing and screening a siRNA sequence aiming at beta-site amyloid precursor protein cutting enzyme 1 (BACE1) and glycogen synthase kinase-3beta (GSK3beta) and adopting a delivery carrier PAH-AM-PEG-ApoE (159-167) 2 (PAPA) modified by ApoE peptide. The siRNA nano delivery system constructed by the invention aims to explore a new way for AD treatment.

There are disclosed therapies and preventions of prion protein complex infections. The transcription of the amyloid precursor protein gene and PrP gene and the RNA transcript are the rate-limiting steps and are most susceptible for blockage and control of the process of amyloid protein formation and PrPsc formation. Thus, therapies and prevention regimes for prion protein complex infections interrupt this process at the level of DNA transcription to RNA, RNA transport to the mitochondrion for protein synthesis and deposition in the cerebral cortex neurons.

Methods of treating and preventing prion protein complex infection are disclosed. Transcription of an amyloid protein precursor protein gene and a PrP gene and RNA transcription are speed limiting steps, and the process of amyloid protein formation and PrPsc formation is most easily blocked and controlled. Thus, the regimen for the treatment and prevention of prion protein complex infection interrupts this process at the level of DNA transcription to RNA, RNA transport to mitochondria for protein synthesis and deposition at cerebral cortical neurons.

The present disclosure provides wild-derived mouse models that comprise a nucleic acid encoding a human amyloid precursor protein (APP), a nucleic acid encoding a mutated human presenilin (1) protein (PSEN1), and in some embodiments, a human apolipoprotein E (APOE), or human amyloid beta and human tau. These mouse models are useful, for example, for Alzheimer's disease studies.

The present invention provides compositions and methods useful for reducing amyloid precursor (APP) gene expression and treating APP-related diseases and disorders. The present invention provides an APPdsRNA agent, an APP antisense polynucleotide agent, a composition comprising an APPdsRNA agent, and a composition comprising an APP antisense polynucleotide agent that can be used to reduce the expression of APP in a cell and a subject.