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Synp78 (proa27), a promoter for the specific expression of genes in retinal ganglion cells

A retinal ganglion and promoter technology, used in gene therapy, genetic engineering, plant genetic improvement, etc., can solve the problem that cell promoters are not functionally characterized.

Pending Publication Date: 2021-06-08
FRIEDRICH MIESCHER INST FOR BIOMEDICAL RES
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

Because the expression of eukaryotic genes is controlled by a complex mechanism of cis- and trans-acting regulatory elements, most cellular promoters have not been extensively functionally characterized

Method used

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  • Synp78 (proa27), a promoter for the specific expression of genes in retinal ganglion cells
  • Synp78 (proa27), a promoter for the specific expression of genes in retinal ganglion cells
  • Synp78 (proa27), a promoter for the specific expression of genes in retinal ganglion cells

Examples

Experimental program
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preparation example Construction

[0097] Preparations for oral administration may be suitably formulated so as to provide controlled release of the active compound.

[0098] The compounds may be formulated for parenteral administration by injection, eg, by bolus injection or continuous infusion. Formulations for injection may be presented in unit dosage form, eg, in ampoules or in multi-dose containers, with an added preservative.

[0099] The compositions may take such forms as suspensions, solutions or emulsions in oily or aqueous vehicles, and may contain formulatory agents such as suspending, stabilizing and / or dispersing agents. Alternatively, the active ingredient may be in powder form for constitution with a suitable vehicle, eg sterile pyrogen-free water, before use.

[0100] The compounds can also be formulated for topical application, such as creams or lotions.

[0101] In addition to the formulations previously described, the compounds may also be formulated as a depot formulation. Such long-acti...

example

[0110] vector construct

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Abstract

The present invention provides an isolated nucleic acid molecule comprising, or consisting of, the nucleic acid sequence of SEQ ID NO:1 or a nucleic acid sequence of at least 1800 bp having at least 80% identity to said sequence of SEQ ID NO:1, wherein said isolated nucleic acid molecule specifically leads to the expression in retinal ganglion cells of a gene when operatively linked to a nucleic acid sequence coding for said gene.

Description

technical field [0001] The present invention relates to a nucleic acid sequence and related applications. The nucleic acid sequence enables specific expression of genes in retinal ganglion cells. Background technique [0002] For expression purposes, recombinant genes are typically transfected into target cells, cell populations or tissues as cDNA constructs in the context of an active expression cassette to allow transcription of the heterologous gene. DNA constructs are recognized by the cellular transcriptional machinery in processes involving the activity of a number of trans-acting transcription factors (TFs) on cis-regulatory elements, including enhancers, silencers, insulators, and promoters (collectively referred to herein as as "promoter"). [0003] Gene promoters are involved in regulation at all these levels, acting as determinants in gene transcription by integrating the influence of DNA sequence, transcription factor binding characteristics, and epigenetic feat...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K48/00C12N15/861
CPCA61K48/0058A61K48/0075C12N15/86C12N2750/14143C12N2830/008A61K48/00C07K14/47
Inventor J·于特纳J·科洛尔B·罗斯卡
Owner FRIEDRICH MIESCHER INST FOR BIOMEDICAL RES