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Artificial transcription factors for the treatment of diseases caused by opa1 haploinsufficiency

An artificial transcription factor, VP16 technology, applied in the field of artificial transcription factors, can solve the problem that there is no feasible treatment for this disease

Inactive Publication Date: 2016-02-24
ALIOPHTHA
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

Until now, there has been no viable treatment for this disease

Method used

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  • Artificial transcription factors for the treatment of diseases caused by opa1 haploinsufficiency
  • Artificial transcription factors for the treatment of diseases caused by opa1 haploinsufficiency
  • Artificial transcription factors for the treatment of diseases caused by opa1 haploinsufficiency

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Experimental program
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Embodiment

[0067] Cloning of DNA plasmids

[0068] For all cloning steps, restriction endonucleases and T4 DNA ligase were purchased from New England Biolabs. Shrimp alkaline phosphatase (SAP) was from Promega. High-fidelity PlatinumPfx DNA polymerase (Invitrogen) was used in all standard PCR reactions. DNA fragments and plasmids were isolated using NucleoSpinGel and PCRClean-up kits, NucleoSpinPlasmid kit or NucleoBondXtraMidiPlus kit (Macherey-Nagel) according to manufacturer's instructions. Oligonucleotides were purchased from Sigma-Aldrich. All relevant DNA sequences of newly generated plasmids were verified by sequencing (Microsynth).

[0069] Cloning of Hexameric Zinc Finger Protein Library for Yeast One-Hybridization

[0070] A hexameric zinc finger protein library containing zinc finger (ZF) modules binding GNN and / or CNN and / or ANN was cloned following Gonzalez B. et al., 2010, NatProtoc 5, 791-810 with the following modifications. DNA sequences encoding GNN, CNN and AN...

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Abstract

The invention relates to an artificial transcription factor comprising a polydactyl zinc finger protein targeting specifically the OPA1 promoter fused to an activatory protein domain, and a nuclear localization sequence. Artificial transcription factors directed against the OPA1 promoter are useful for the treatment of diseases associated with OPA1 haploinsufficiency, such as autosomal dominant optic atrophy, syndromic autosomal dominant optic atrophy plus and normal tension glaucoma.

Description

field of invention [0001] The present invention relates to artificial transcription factors comprising a polydactyly zinc finger protein specifically targeting the OPA1 gene promoter and a nuclear localization sequence fused to an activation domain, and to their role in the treatment of OPA1 mutations resulting in haploinsufficiency Use in diseases such as autosomal dominant optic atrophy (ADOA) or syndromic ADOA+ (syndromic ADOAplus). Background technique [0002] Artificial transcription factors (AFTs) have been proposed to be useful tools for modulating gene expression (Sera T., 2009, AdvDrugDelivRev61, 513-526). Many naturally occurring transcription factors, which affect gene expression by repressing or activating gene transcription, have complex specific domains that recognize a certain DNA sequence. This makes them unattractive targets for manipulation if the skilled person intends to modify their specificity and target gene(s). However, a certain class of transcrip...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): C07K14/47
CPCC07K14/4702C07K2319/09C07K2319/10C07K2319/71C07K2319/81A61K47/60A61K38/17A61P25/00A61P27/02A61P27/06A61P43/00C07K14/435
Inventor 艾伯特·诺伊特茨纳约瑟夫·弗拉默尔爱丽丝·赫胥黎
Owner ALIOPHTHA
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