Aav gene therapy for treating nephrotic syndrome

A nephrotic syndrome, gene therapy technology, applied in the field of treatment of monogenic forms of nephrotic syndrome, can solve the problem of not exploring AAV serotypes

Pending Publication Date: 2021-10-22
UNIV OF BRISTOL
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

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Problems solved by technology

The study also did not explore suitable AAV serotypes for transduction of human kidney cells

Method used

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  • Aav gene therapy for treating nephrotic syndrome
  • Aav gene therapy for treating nephrotic syndrome
  • Aav gene therapy for treating nephrotic syndrome

Examples

Experimental program
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Embodiment Construction

[0052] method

[0053] Vector production

[0054] We use human ( Figure 6) and mouse (sequence not shown) podocin cDNA (Origene, Herford, Germany) and human VDR and Smad7 cDNA, pAV.hNPHS1.mpodHA.WPRE was prepared from the CMV eGFP L22Y pUC-AV2 construct (a kind gift of Amit Nathwani) .bGH, pAV.mNPHS1.mpodHA.WPRE.bGH and pAV.hNPHS1.hpodHA.WPRE.bGH ( Figure 1A ) pAV.mNPHS1.hHAVDR.WPRE.bGH and pAV.mNPHS1.hHASmad7.WPRE.bGH. Human embryonic kidney 293T cells were transfected with capsid plasmids (pAAV9 from Penn Vector Core, pAAV LK03 was a kind gift from Mark Kay), helper plasmids with adenovirus genes, and transgenic plasmids using polyethyleneimine. Cells and supernatants were harvested 72 hours after transfection. Cells were subjected to 5 freeze-thaw cycles, while supernatants were subjected to PEG precipitation (8% PEG 0.5N NaCl). These were combined and incubated with 0.25% sodium deoxycholate and 70 units / ml Benzonase at 37°C for 30 minutes. Vector was purified by io...

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Abstract

The present invention provides an adeno-associated virus (AAV) vector gene therapy for use in treating a monogenic form of nephrotic syndrome, wherein the AAV vector comprises a NS-associated transgene and minimal nephrin promoter NPHS1 or podocin promoter NPHS2.

Description

[0001] field of invention [0002] The present invention relates to gene therapy for the treatment of monogenic forms of nephrotic syndrome. [0003] Background of the invention [0004] Nephrotic syndrome (NS) is a chronic kidney disease characterized by marked proteinuria, hypoalbuminemia, edema, and hyperlipidemia, and is the most common primary glomerular disease in children, occurring in It affects 2 / 100,000 children under 16 in Europe and the United States. NS is associated with different ages of onset, from less than 3 months of age at diagnosis to early adulthood, and into distinct patient populations based on their sensitivity to corticosteroids: approximately 80% of children with NS are classified as having corticosteroids Sensitive nephrotic syndrome (SSNS) and can be successfully treated with corticosteroid therapy. A subset of patients originally classified as SSNS relapses and requires further steroid therapy, and an additional 10-15% of NS patients do not achie...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K48/00C12N15/861
CPCC12N2750/14143C12N2830/008C12N2830/48A01K2217/075A01K2217/206A01K2267/035A01K2227/105A61K48/005A61K48/0058C12N15/86C12N2830/50
Inventor M.A.萨利姆-乌丁G.I.韦尔什
Owner UNIV OF BRISTOL
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