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1. WO1996004397 - GENE THERAPY USING HOMOLOGOUS RECOMBINATION

Publication Number WO/1996/004397
Publication Date 15.02.1996
International Application No. PCT/SE1994/001038
International Filing Date 03.11.1994
Chapter 2 Demand Filed 05.03.1996
IPC
A61K 38/17 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
17from animals; from humans
A61K 38/37 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
17from animals; from humans
36Blood coagulation or fibrinolysis factors
37Factors VIII
A61K 48/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C07K 14/755 2006.01
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
745Blood coagulation or fibrinolysis factors
755Factors VIII
CPC
A61K 38/1709
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
17from animals; from humans
1703from vertebrates
1709from mammals
A61K 38/37
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
17from animals; from humans
36Blood coagulation or fibrinolysis factors
37Factors VIII
A61K 48/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C07K 14/755
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
745Blood coagulation or fibrinolysis factors
755Factors VIII ; , e.g. factor VIII C (AHF), factor VIII Ag (VWF)
Applicants
  • ANVRET, Maria [SE/SE]; SE
  • BLOMBÄCK, Margareta [SE/SE]; SE
  • ZHANG, Zhiping [CN/SE]; SE
Inventors
  • ANVRET, Maria; SE
  • BLOMBÄCK, Margareta; SE
  • ZHANG, Zhiping; SE
Agents
  • ONN, Thorsten ; AB Stockholms Patentbyrå, Zacco & Bruhn P.O. Box 23101 S-104 35 Stockholm, SE
Priority Data
9402642-405.08.1994SE
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) GENE THERAPY USING HOMOLOGOUS RECOMBINATION
(FR) THERAPIE GENIQUE A RECOMBINAISON HOMOLOGUE
Abstract
(EN)
The present invention is related to a method to correct the mutation(s) behind a genetic disease in the genome of somatic cells derived from an individual afflicted with this disease by contacting said cells with the corresponding non-mutant DNA-fragment to allow it to undergo homologous recombination with and, thus, replace a DNA sequence of the somatic cell genome, wherein said mutation(s) is(are) located. The cells obtained according to the invention can be administered to an individual as a treatment of the said disease. The present invention is also related to a DNA-liposome suspension comprising the said non-mutant DNA-fragment. The said suspension can be used as a DNA-vehicle in the above process. In addition, it can be administered to an individual to obtain correction of mutation(s) by $i(in vivo) integration of the said DNA into a mutated gene by homologous recombination.
(FR)
Méthode de correction de mutation(s) à l'origine d'une maladie génétique dans le génome de cellules somatiques issues d'un individu atteint de cette maladie qui consiste à mettre lesdites cellules en contact avec le fragment d'ADN non-mutant correspondant de sorte qu'il subisse une recombinaison homologue et remplace, ainsi, une séquence d'ADN du génome des cellules somatiques, dans lequel cette ou ces mutation(s) est ou sont situé(es). Les cellules obtenues par ce procédé peuvent être administrées à un individu à titre de traitement de la maladie. L'invention porte également sur une suspension liposome-ADN comprenant ledit fragment d'ADN non-mutant. Cette suspension peut être utilisée comme véhicule-ADN dans le procédé précité. En outre, elle peut être administrée à un individu pour corriger la ou les mutation(s) par intégration $i(in vivo) de ladite ADN en un gène de mutation par recombinaison homologue.
Also published as
Latest bibliographic data on file with the International Bureau