SynPIII, promoter for specific expression of genes in retinal pigment epithelium

A retinal pigment and gene technology, applied in gene therapy, using vectors to introduce foreign genetic material, virus/bacteriophage, etc., can solve the problem of cell promoters not being functionally characterized

Pending Publication Date: 2020-08-14
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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  • SynPIII, promoter for specific expression of genes in retinal pigment epithelium
  • SynPIII, promoter for specific expression of genes in retinal pigment epithelium
  • SynPIII, promoter for specific expression of genes in retinal pigment epithelium

Examples

Experimental program
Comparison scheme
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preparation example Construction

[0098] Preparations for oral administration may be suitably formulated so as to provide controlled release of the active compound.

[0099] 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.

[0100] 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.

[0101] The compounds can also be formulated for topical application, such as creams or lotions.

[0102] In addition to the formulations previously described, the compounds may also be formulated as a depot formulation. Such long-acti...

example

[0111] vector construct

[0112] Order of phylogenetically conserved DNA elements identified by nucleotide sequences preceding the transcription start sites of Pde6h, Pde6c, Cngb3, Fabp7, Gngt2, Ppm1j, Gckr, Igj, Arhgdib, Clca3, Lcn2 genes Assembly resulted in the synthetic promoter used in this study (1317 bp; SEQ ID NO: 1). The ChR2-GFP coding sequence was inserted immediately after this promoter and optimized Kozak sequence (GCCACC), followed by the woodchuck hepatitis virus post-transcriptional regulatory element (WPRE) and the SV40 polyadenylation site. Retinal neurons in non-human primates were targeted using AAV serotype BP2 at a titer of 4.27E+13 GC / mL.

[0113] Viral transfection and tissue preparation

[0114] AAV administration was performed in collaboration with ophthalmologists and third-party contractors in Kunming, China. Rhesus monkeys were anesthetized with ketamine and sodium phenobarbital. Two puncture tunnels were created using a 25-gauge needle, one in...

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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 1000 bp having at least 80% identity to the sequence of SEQ ID NO:1, wherein the isolated nucleic acid molecule specifically leads to the expression in cells of the retinal pigment epithelium of a gene when operatively linkedto a nucleic acid sequence coding for the gene.

Description

technical field [0001] The present invention relates to a nucleic acid sequence that enables specific expression of a gene in retinal pigment epithelial 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 features. They determine, for example, ...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): C12N15/85
CPCA61K48/0058C12N2830/008C12N2750/14143C12N15/86A61K48/0075
Inventor J·于特纳J·科洛尔B·罗斯卡
Owner FRIEDRICH MIESCHER INST FOR BIOMEDICAL RES
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