6 results about "Amyloid Protein Precursor" patented technology

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).

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.

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 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.