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APOE gene therapy

A gene therapy, gene therapy technology, applied in the direction of genetic engineering, chemical instruments and methods, single-stranded DNA virus, etc.

Pending Publication Date: 2022-07-29
CORNELL UNIVERSITY
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  • Abstract
  • Description
  • Claims
  • Application Information

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Problems solved by technology

In contrast, APOE2 attenuates these effects

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example 1

[0173] Overview

[0174]The pathogenesis of Alzheimer's disease (AD) is complex and characterized by central nervous system (CNS) accumulation of amyloid-beta (Aβ) and amyloid plaques, aberrant tau phosphorylation, tau tangles, inflammation, and neurological Meta-progressive loss, leading to progressive cognitive decline1. Genetics plays a major role in the risk of these pathogenic processes (DeTure & Dickson, 2019; Holtzman et al., 2012; Safieh et al., 2019; Fernandez et al., 2019). Early-onset autosomal dominant AD is caused by mutations in amyloid precursor protein (APP) and presenilin (PSEN) 1 and 2 (Campion et al., 1999; Carmona et al., 2018), the APP and the PSEN 1 and 2 are genes that affect APP processing, altering Aβ peptide production, leading to aggregation and plaque formation (Carmona et al., 2018; Dai et al., 2018). The major genetic factor in sporadic late-onset AD is a variant of apolipoprotein E (APOE), a lipid transporter (Tzioras et al., 2019; Wolters et...

example 2

[0211] Overview

[0212] Alzheimer's disease (AD) is a degenerative brain disease and the most common cause of dementia, currently affecting 5.8 million Americans and 50 million people worldwide. AD symptoms include a progressive decline in cognitive and functional abilities and brain pathology, including extracellular beta-amyloid plaques, intracellular tau tangles, chronic inflammation, and brain atrophy. The strongest genetic risk factor for susceptibility to late-onset AD involves polymorphisms of the apolipoprotein E (APOE) allele. APOE4 is found at high frequencies in AD patients, and homozygous inheritance is associated with a 14.5-fold increased risk of developing AD. Conversely, APOE2 reduced the risk of AD development and delayed the onset of the disease, eg, reduced the risk of developing AD by ≥50% and delayed the age of onset. Based on epidemiological data, APOE4 was associated with increased brain amyloid load and greater memory impairment in AD, whereas APOE...

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Abstract

Provided is a gene therapy vector comprising an expression cassette encoding a mammalian apolipoprotein E having a residue other than arginine at at least one of position 112, position 136, or position 158, rather than a mammalian apolipoprotein E having R112, R136, and R158 or a mammalian apolipoprotein E having C112, R136, and C158, or encoding an antibody that binds to APOE4 or disrupts the binding of APOE to heparan sulfate proteoglycan, and methods of using the same.

Description

technical field [0001] This application claims the benefit of the filing date of US Application No. 62 / 939,999, filed November 25, 2019, the disclosure of which is incorporated herein by reference. Background technique [0002] Alzheimer's disease (AD) directly affects 5 million Americans, and its prevalence and economic impact are rapidly increasing. Existing drugs have little effect on the underlying disease process, and there are currently no preventive therapies available. Inheritance of the variant APOE4 conveys a high risk of developing AD, while inheritance of the APOE2 gene is protective, reducing the risk of developing AD by approximately 50% and delaying the age of onset. APOE4 is associated with increased brain amyloid load and greater memory impairment in AD. In contrast, APOE2 attenuated these effects. A previous study showed that delivery of the adeno-associated virus (AAV) gene encoding the human APOE2 coding sequence to the CNS of a murine model expressing...

Claims

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Application Information

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IPC IPC(8): C07K14/775A61P25/28
CPCC07K14/775A61P25/28C12N15/86C12N15/113A61K48/005A61K48/0075C12N2750/14143C12N2310/141
Inventor R·G·克雷斯托S·M·凯明斯基K·斯泰尔斯D·松地
Owner CORNELL UNIVERSITY
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