Chemistry: molecular biology and microbiology – Treatment of micro-organisms or enzymes with electrical or... – Modification of viruses
Patent
1997-09-12
1999-02-09
Ketter, James
Chemistry: molecular biology and microbiology
Treatment of micro-organisms or enzymes with electrical or...
Modification of viruses
4353201, C12N 1564
Patent
active
058693068
DESCRIPTION:
BRIEF SUMMARY
TECHNICAL FIELD
This invention relates to a process for producing a freeze-dried preparation of a virus vector for gene therapy which is excellent in safety and storage stability and a gene transfer preparation obtained by this process.
BACKGROUND ART
Owing to the rapid progress in genetic engineering, there have been developed various molecular biological processes. With these developments, techniques for analyzing genetic information and gene functions have been remarkably advanced. As a result, a number of attempts have been made to feed back the results thus achieved into actual clinical treatments. One of the most remarkable advances has been achieved in the field of gene therapy. That is to say, there have been successfully identified and decoded genes causative of various hereditary diseases. On the other hand, techniques have been established for physically or chemically transferring these genes into cells. Accordingly, gene therapy has already completed the stage of fundamental experiments and thus reached the stage of clinical application.
Since the first clinical test on gene therapy was performed in 1989 in the United States, gene therapy has been already applied to clinical tests in Italy, the Netherlands, France, England and China. In the United States, in particular, the Recombinant DNA Committee (RAC) of NIH has approved 54 gene therapy protocols by July 1994 and, therefore, attempts have been made to apply gene therapy to the treatment of hereditary diseases such as congenial immunological deficiency (adenosine deaminase deficiency), familial hypercholesterolemia and cystic fibrosis and various types of cancer such as malignant melanoma and glioma. Moreover, a number of fundamental studies on the gene therapy for AIDS have been made in recent years.
Gene therapy is classified into germline cell gene therapy and somatic cell gene therapy depending on the type of the target cells to which genes are to be transferred. From another point of view, it is classified into augmentation gene therapy wherein a new (normal) gene is added while leaving the abnormal (causative) gene as such and replacement gene therapy wherein the abnormal gene is replaced by the normal one. At the present stage, the augmentation gene therapy on somatic cells is exclusively effected due to ethical and technical restrictions. A gene therapy process performed today comprises taking out the target cells from the body and, after the completion of the gene transfer, putting the cells back into the body again through self-transplantation (i.e., ex vivo gene therapy). Further, it is now under consideration to administer genes directly to patients in future (i.e., in vivo gene therapy).
One of the large problems in the clinical application of gene therapy is how to safely and efficiently introduce a foreign gene into the target cells. Although it was tried to employ physical procedures such as microinjection early in the 1980's , only a poor transfer efficiency could be established and genes could not be transferred in a stable state thereby. Furthermore, the limited techniques for cell incubation on a mass scale in those days made it impossible to put such attempts into practical use. Subsequently, there were developed recombinant viruses (virus vectors) for efficiently transferring foreign genes into target cells, which made it possible for the first time to apply the gene therapy to clinical purposes.
There are several types of virus vectors as will be described hereinbelow. The virus vectors most frequently employed in the gene therapy today are retrovirus vectors originating in moloney murine leukemia virus (MoMLV). That is to say, genes are transferred by taking advantage in the propagation manner of this virus. A retrovirus is an RNA virus having an envelope which invades into cells through the bond of the envelope protein to the receptor in the host cell side. After the invasion, the single-stranded virus RNA is converted into a double-stranded DNA via a reverse transcriptase and thus integrated into the genomic DNA
REFERENCES:
patent: 5292653 (1994-03-01), Kit et al.
patent: 5681746 (1997-10-01), Bedner et al.
European Search Report dated 15 May 1998.
Venkatesh et al., "Selective induction of toxicity to human cells expressing human immunodeficiency virus type 1 Tat by a conditionally cytotoxic adenovirus vector," Proc. Natl. Acad. Sci. USA 87:8746-8750 (1990).
Poznansky et al., "Gene Transfer into Human Lymphocytes by a Defective Human Immunodeficiency Virus Type 1 Vector," Journal of Virology 65:532-536 (1991).
Brady et al., "Specific ablation of human immunodeficiency virus Tat-expressing cells by conditionally toxic retroviruses," Proc. Natl. Acad. Sci. USA 91:365-369 (1994).
Biasolo et al., "Gene therapy of AIDS: inhibition of HIV by retroviral vectors," ECB6: Proceedings of the 6th European Congress on Biotechnology: 685-688, 1994, Elsevier Science B.V.
Caruso et al., "HIV-triggered killing of booby trapped cells prevents viral spread in an HIV-infected cell population," Bone Marrow Transplantation 9(Suppl. 1):187-188, 1992.
"Improved Methods of Retroviral Vector Transduction and Production for Gene Therapy", Kotani et al. Human Gene Therapy, vol. 5, 1994, pp. 19-28.
English language translation and copy of "Experimental Techniques in Virology", revised second edition, General Principles, Edited by the Research Fellow Associtation, National Institute of Health, Japan, and published by Maruzen Co., Ltd.
Iijima Osamu
Kuma Hidekazu
Suzuki Yosuke
Hisamitsu Pharmaceutical Co. Inc.
Ketter James
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