7 results about "Presenilin" patented technology

Provided herein are compositions comprising a nucleic acid encoding an amyloid-beta precursor protein and / or a nucleic acid encoding premature protein-1, cells comprising these compositions, animals comprising these compositions, methods of making these cells and animals, methods of modeling Alzheimer's disease, and methods of preparing such cells and animals. A method of assessing a treatment candidate for treating or ameliorating a symptom or phenotype of Alzheimer's disease, and a method of assessing a treatment candidate for preventing or preventing a symptom or phenotype of Alzheimer's disease.

The present disclosure provides, among other things, human codon-optimized sequences encoding presenilin 1, and methods for using the sequences in gene therapy to treat neurodegenerative diseases including, but not limited to Alzheimer's disease, frontotemporal dementia, frontotemporal lobar degeneration, Pick's disease, Lewy body dementia, memory loss, and cognitive impairment including mild cognitive impairment (MCI).

The present disclosure provides, among other things, methods for using presenilin based gene therapy to treat neurodegenerative dementia including, but not limited to Alzheimers disease, frontotemporal dementia, frontotemporal lobar degeneration, Picks disease, Lewy body dementia, memory loss, and cognitive impairment including mild cognitive impairment (MCI).

The present invention relates to the use of a novel compound for preventing, alleviating or treating Alzheimer's disease, and the novel compound exhibits an inhibitory effect on tau protein aggregation. In addition, it has been identified that a reduction in presenilin 1 is made by using the novel compounds for treatment. Therefore, the novel compound can be effectively used in the development of a therapeutic agent for Alzheimer's disease.

The invention relates to application of a sarsasapogenin structure derivative in preparation of a medicine for treating Alzheimer's disease. The structural formula of the sarsasapogenin structure derivative is shown in the specification. According to the application of the sarsasapogenin structure derivative in preparation of the medicine for treating the Alzheimer's disease, a double-transgenic Alzheimer's disease model mouse with APP protein overexpression and PS1 premature senile gene mutation can prove that the small molecule compound has a good effect of treating the Alzheimer's disease; the compound has a potential huge value for treating Alzheimer's disease, and also has important scientific and commercial application values.

Disclosed herein are products, methods, and uses for treating, ameliorating, delaying the progression of, and / or preventing a disease or disorder associated with expression of an aberrant lamin A (LMNA) gene or progerin gene. Such disease or disorder includes, but is not limited to, a laminopathy, progeroid syndrome, progeria, or aging disorder resulting from the aberrant expression of LMNA or progerin. In some instances, the progeria is Hutchinson-Gilford progeria syndrome (HGPS). In some instances, the disease or disorder associated with the expression of progerin is premature aging or natural aging including, but not limited to, atherosclerosis, alopecia, osteoporosis, cardiovascular disease, skin abnormalities, fat storage, stroke, myocardial infarction, stroke, heart failure, muscle wasting, muscle weakness, myotonia, skeletal muscle problems, abnormalities of the retina, hip weakness, abdominal muscle weakness, joint and spinal abnormalities, lower leg weakness, shoulder weakness, hearing loss, and / or tissue inflammation. More particularly, disclosed herein are RNA interference-based products, methods, and uses for inhibiting or downregulating the expression of progerin. Even more particularly, the disclosure provides guide RNA and a CRISPR endonuclease for inhibiting or downregulating the expression of progerin and methods of using said guide RNA and a CRISPR endonuclease to correct a mutation in the LMNA gene and inhibit or downregulate progerin expression in cells and / or in cells of a subject having a condition resulting from the expression of progerin including, but not limited to, HGPS or progeria, an HGPS-like condition affecting LMNA mutations that affect exon 11 splicing, or a condition resulting from the expression of progerin.

The present invention relates to use of a polynucleotide. The in vitro administration of the polynucleotide sequence set forth in SEQ ID NO: 5 to somatic cells of naturally aged subjects can significantly reduce the expression of aging markers, DNA damage markers, and presenilin in the somatic cells, demonstrating potential efficacy in preventing or delaying aging.