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Gene therapeutics

A gene therapy and functional technology, applied in gene therapy, biochemical equipment and methods, double-stranded DNA virus, etc., can solve problems such as failure to achieve guiding effect, low efficiency, and damage to infection function combined with function

Inactive Publication Date: 2002-04-17
TAKARA HOLDINGS
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

The biggest technical problem with gene therapy is the very low efficiency of transferring therapeutic genes into target cells, especially into hematopoietic stem cells
However, in many cases the desired targeting is not achieved due to the disruption of either or both of the envelope-intrinsic infection function and the ligand-intrinsic binding function by expression as a fusion

Method used

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  • Gene therapeutics

Examples

Experimental program
Comparison scheme
Effect test

Embodiment 1

[0078] (1) The vector PGK-hADA ("Natural Medicine" 2: 876-882 (1996))-containing human ADA produced by EPHA-5-producer cells ("Natural Medicine" 2: 876-882 (1996)) The PGK vector of the gene (hADA), and the injection preparation of RetroNectin as described in Example 2 below were injected into the tail vein of C3H / HeJ mice (8 weeks old, purchased from Japan SLC). Transduction of hematopoietic stem cells was analyzed by examining the expression of transduced human ADA cDNA in mice bearing the transgene as described in Nature Medicine 2:876-882 (1996). Specifically, the presence of human ADA protein in peripheral blood cells derived from mice was confirmed by ADA isozyme analysis using cellulose acetate electrophoresis for detection. Exams are performed at the beginning of the fourth month after transplantation and repeated monthly.

[0079] (2) For mice administered with RetroNectin and the vector PGK-hADA, expression of human ADA cDNA was confirmed by analysis of transduced b...

Embodiment 2

[0081] RetroNectin (CH-296, Takara Shuzo) was dissolved in water for injection at a concentration of 2 mg / ml. This solution was equilibrated with saline to prepare a formulation for injection.

Embodiment 3

[0082] Example 3: Gene transfer using HL-60 cells as vectors

[0083] Polypeptide CH-271 was prepared as described below. Briefly, Escherichia coli HB101 / pCH101 (FERM BP-2799) was cultured according to the method described in US Patent No. 5,198,423. CH-271 was obtained from the culture.

[0084] Human leukemia HL-60 cells (purchased from Dainippon Pharmaceutical) were used at 2×10 6 Cells / ml were suspended in D-EME medium (Bio Whittaker) containing 10% fetal calf serum (FCS, Bio Whittaker). RetroNectin TM (Takara Shuzo) or CH-271 was added to 1 ml of the cell suspension at a final concentration of 100 μg / ml. A control group to which no such functional substance was added was provided.

[0085] 100 μl containing 6.23 × 10 6 cfu / ml A solution of an environmental retroviral vector with an enhanced green fluorescent protein (EGFP) gene (pLELN (Clontech), prepared using GP+E-86 cells (ATCC CRL-9642)) was added to the cells. The mixture was incubated at 37°C for 30 minutes i...

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Abstract

Gene therapeutics to be used in treating diseases showing sensitivity to gene therapy, characterized by containing as the active ingredient an efficacious amount of a functional substance which has a function of having an affinity for a virus containing a gene usable in the gene therapy and another function of having an affinity specific for a target cell with a need for the gene transfer, or an efficacious amount of a functional substance which has an affinity for the above virus and an efficacious amount of another functional substance which has an affinity specific for the above cell.

Description

technical field [0001] The present invention relates to compositions and methods for missile gene therapy, which can be used to treat diseases requiring gene therapy and for the selective transfer of genes into target cells in vivo. Background technique [0002] About 3,000 cases of gene therapy have been performed so far in the world. The biggest technical problem with gene therapy is the very low efficiency of transferring therapeutic genes to target cells, especially hematopoietic stem cells. Recently, gene transfer efficiency has been remarkably improved by using a recombinant protein of the fibronectin fragment CH-296 (Takara Shuzo; RetroNectin), thereby making gene therapy practical (Natural Medicine 2: 876-882 (1996)) . Recombinant RetroNectin molecules can bind retroviruses with incorporated therapeutic genes and target cells so that they become adjacent to each other, thereby greatly improving gene transfer efficiency. The most difficult is said to be transferrin...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K48/00C12N15/86
CPCC12N2710/10043A61K48/00C12N2810/40C12N15/86C12N2740/13043A61K39/395
Inventor 加藤郁之进浅田起代蔵上野充博桥野仁一吉冈广文田中启二
Owner TAKARA HOLDINGS
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