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Gene therapy for recessive dystrophy epidermis bullosa using genetically corrected autologous keratinocytes

A cell and gene technology, applied in the field of gene therapy for recessive dystrophic epidermolysis bullosa using genetically corrected autologous keratinocytes, which can solve systemic toxicity and other problems

Pending Publication Date: 2022-07-05
THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIV +1
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

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Problems solved by technology

Systemic therapy of C7 can lead to systemic toxicity

Method used

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  • Gene therapy for recessive dystrophy epidermis bullosa using genetically corrected autologous keratinocytes
  • Gene therapy for recessive dystrophy epidermis bullosa using genetically corrected autologous keratinocytes
  • Gene therapy for recessive dystrophy epidermis bullosa using genetically corrected autologous keratinocytes

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Embodiment 1

[0114] Cellular reprogramming of autologous cells as a treatment for recessive dystrophic epidermolysis bullosa (RDEB).

[0115] Recessive dystrophic epidermolysis bullosa (RDEB) is a severe blistering skin disease caused by loss-of-function mutations in COL7A1, the gene encoding collagen type VII (C7). C7 is a major component of anchoring fibrils (AF), which stabilize the basement membrane zone (BMZ) of the epidermis to the dermis. C7 performs this function by using multiple domains including the amino-terminal non-collagen NC1 domain that binds BMZ ligands, the central collagen domain that assembles into a triple helix, and the NC2 domain that catalyzes the assembly of C7 into AF . Loss of these functional C7 domains in RDEB results in severe BMZ segregation. This produces extensive and painful blistering, erosion, and scarring, which in turn leads to the aggressive and often fatal form of SCC that occurs in the second and third decades. Despite advances in molecular diag...

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Abstract

Methods for cell-based delivery of collagen VII to treat epidermis bullosa and corneal erosion are provided. The present disclosure also provides compositions and pharmaceutical compositions comprising, or consisting essentially of, or further consisting of, a keratinocyte sheet or a corneal cell sheet.

Description

[0001] Division description [0002] This application is a Chinese patent application No. 201780005233.2 filed with the State Intellectual Property Office on June 27, 2018 "Gene therapy for recessive dystrophic epidermolysis bullosa using genetically corrected autologous keratinocytes ” divisional application. [0003] Federally funded research and development [0004] This invention was made with government support under Contract AR055914 awarded by the National Institutes of Health. The government has certain rights in the invention. [0005] CROSS-REFERENCE TO RELATED APPLICATIONS [0006] This application claims priority under 35 U.S.C. 119(e) to US Serial No. 62 / 274,700, filed on January 4, 2016, and US Serial No. 62 / 414,533, filed on October 28, 2016, and the patent's The contents of each are incorporated herein by reference. technical field [0007] The present disclosure generally relates to methods and compositions for treating epidermolysis bullosa (EB) and corn...

Claims

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Application Information

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IPC IPC(8): A61L27/36A61K48/00A61K38/39A61P27/02A61P17/00
CPCA61L27/3604A61L27/3695A61K48/0091A61K48/0075A61K48/0058A61K38/39A61P27/02A61P17/00A61L2430/16C12N5/0629C12N2510/00C12N2760/16043C12N5/00
Inventor 祖拉布·希普拉什维利尼贡·T·纽伦M·皮特·马林科维奇吉恩·唐阿尔弗雷德·T·莱恩保罗·A·卡瓦里
Owner THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIV
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