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Gene therapy for haploinsufficiency

A dysfunctional, haploid technology, applied in gene therapy, genetic engineering, virus/bacteriophage, etc., can solve the problems of reduced transcript stability, reduced gene transcription, and insufficient gene products to produce wild-type phenotypes

Pending Publication Date: 2019-12-24
RGT UNIV OF CALIFORNIA
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

In other diseases, genomic alterations reduce transcription of one or both copies of a gene or reduce transcript stability such that the gene product is insufficient to produce a wild-type phenotype

Method used

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  • Gene therapy for haploinsufficiency
  • Gene therapy for haploinsufficiency
  • Gene therapy for haploinsufficiency

Examples

Experimental program
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Embodiment

[0241] Rescuing obesity from haploid insufficiency

[0242] I. Introduction

[0243] More than 300 genes are known to contribute to human disease due to haploinsufficiency (1, 2), resulting in a wide range of phenotypes including cancer, neurological disorders, developmental disorders, immune disorders, metabolic disorders, infertility, renal disease, limb deformities etc. (1). Large-scale exome sequencing analysis estimated a total of 3230 human genes that may be heterozygous for loss-of-function (LoF) intolerance (3). Gene therapy holds great promise for correcting haploid insufficiency disorders by inserting one or more functionally recombinant copies of a mutated gene. Currently, 2300 gene therapy clinical trials are underway, most of which use adeno-associated virus (AAV) to deliver recombinant genes (4). AAV is a preferred method of gene delivery because of its ability to deliver DNA without integration into the genome, without causing pathogenicity and providing long...

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Abstract

Methods and compositions are provided for activating transcription in a mammalian cell.

Description

[0001] Cross References to Related Applications [0002] This application claims priority to U.S. Provisional Application No. 62 / 455,988, filed February 7, 2017, which is incorporated herein by reference for all purposes. [0003] Claims of Inventions Made Under Federally Sponsored Research and Development [0004] This invention was made with government support under grant number R01 DK090382 awarded by the National Institutes of Health. The government has certain rights in this invention. [0005] Submit a reference to a sequence listing [0006] This application includes a sequence listing in a text file named "081906-224410PC-1072775_SequenceListing.txt" generated on February 6, 2018 and contains 107 kb. The material contained in this text file is incorporated by reference in its entirety for all purposes. [0007] field of invention [0008] The present disclosure generally relates to methods and compositions for activating transcription in mammalian cells. Backgroun...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K38/00A61K38/17A61K47/64A61K48/00A61P3/04A61P25/00A61P25/08A61P25/28A61P27/02A61P13/12
CPCA61K38/17C12N9/22A01K2217/077A01K2227/105A01K2267/0362C12N2750/14143C12N15/113C12N2310/20A61P13/12A61P25/00A61P25/08A61P25/28A61P27/02A61P3/04A61P43/00A01K67/0276A61K48/0016A61K48/0066C12N15/86C12N15/102
Inventor N·阿黑度威N·玛莎鲁
Owner RGT UNIV OF CALIFORNIA
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