WO1998049350A8 - In vivo use of recombinagenic oligonucleobases to correct genetic lesions in hepatocytes - Google Patents

In vivo use of recombinagenic oligonucleobases to correct genetic lesions in hepatocytes

Info

Publication number
WO1998049350A8
WO1998049350A8 PCT/US1998/008834 US9808834W WO9849350A8 WO 1998049350 A8 WO1998049350 A8 WO 1998049350A8 US 9808834 W US9808834 W US 9808834W WO 9849350 A8 WO9849350 A8 WO 9849350A8
Authority
WO
WIPO (PCT)
Prior art keywords
hepatocytes
genetic changes
vivo use
phenotype
genetic lesions
Prior art date
Application number
PCT/US1998/008834
Other languages
French (fr)
Other versions
WO1998049350B1 (en
WO1998049350A1 (en
Inventor
Clifford J Steer
Betsy T Kren
Paramita T Bandyopadhyay
Original Assignee
Univ Minnesota
Clifford J Steer
Betsy T Kren
Paramita T Bandyopadhyay
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Univ Minnesota, Clifford J Steer, Betsy T Kren, Paramita T Bandyopadhyay filed Critical Univ Minnesota
Priority to CA002288209A priority Critical patent/CA2288209A1/en
Priority to JP54742998A priority patent/JP2002511851A/en
Priority to EP98920930A priority patent/EP0979311A1/en
Priority to NZ500694A priority patent/NZ500694A/en
Priority to AU73654/98A priority patent/AU749410B2/en
Priority to US09/108,006 priority patent/US6524613B1/en
Publication of WO1998049350A1 publication Critical patent/WO1998049350A1/en
Publication of WO1998049350B1 publication Critical patent/WO1998049350B1/en
Publication of WO1998049350A8 publication Critical patent/WO1998049350A8/en

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/107—Emulsions ; Emulsion preconcentrates; Micelles
    • A61K9/1075—Microemulsions or submicron emulsions; Preconcentrates or solids thereof; Micelles, e.g. made of phospholipids or block copolymers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents

Abstract

The present invention concerns compositions and methods for the introduction of specific genetic changes in endogenous genes of the cells of an animal. The genetic changes are effected by oligonucleotides or oligonucleotide derivatives and analogs, which are generally less than about 100 nucleotides in length. The invention provides for macromolecular carriers, optionally incorporating ligands for clathrin coated pit receptors. In one embodiment the ligand is a lactose or galactose and the genetic changes are made in hepatocytes. By means of the invention up to 40 % of the copiies of a target gene have been changed in vitro. Repair of mutant genes having a Crigler-Najjar like phenotype and Hemophilia B phenotype were observed.
PCT/US1998/008834 1997-04-30 1998-04-30 In vivo use of recombinagenic oligonucleobases to correct genetic lesions in hepatocytes WO1998049350A1 (en)

Priority Applications (6)

Application Number Priority Date Filing Date Title
CA002288209A CA2288209A1 (en) 1997-04-30 1998-04-30 In vivo use of recombinagenic oligonucleobases to correct genetic lesions in hepatocytes
JP54742998A JP2002511851A (en) 1997-04-30 1998-04-30 Use of recombinant oligonucleobases in vivo to correct hepatocyte genetic lesions in vivo
EP98920930A EP0979311A1 (en) 1997-04-30 1998-04-30 $i(IN VIVO) USE OF RECOMBINAGENIC OLIGONUCLEOBASES TO CORRECT GENETIC LESIONS IN HEPATOCYTES
NZ500694A NZ500694A (en) 1997-04-30 1998-04-30 Use of chimeric mutational vector (CMV), a macromolecule and a ligand for cell surface receptor adhesion molecule (clatherin) in gene therapy; the CMV vector derived from REC2 homologous pairing with Ustilago maydis
AU73654/98A AU749410B2 (en) 1997-04-30 1998-04-30 In vivo use of recombinagenic oligonucleobases to correct genetic lesions in hepatocytes
US09/108,006 US6524613B1 (en) 1997-04-30 1998-06-30 Hepatocellular chimeraplasty

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US4528897P 1997-04-30 1997-04-30
US60/045,288 1997-04-30
US5483797P 1997-08-05 1997-08-05
US60/054,837 1997-08-05
US6499697P 1997-11-10 1997-11-10
US60/064,996 1997-11-10

Related Child Applications (1)

Application Number Title Priority Date Filing Date
US09/108,006 Continuation-In-Part US6524613B1 (en) 1997-04-30 1998-06-30 Hepatocellular chimeraplasty

Publications (3)

Publication Number Publication Date
WO1998049350A1 WO1998049350A1 (en) 1998-11-05
WO1998049350B1 WO1998049350B1 (en) 1999-01-07
WO1998049350A8 true WO1998049350A8 (en) 2000-05-25

Family

ID=27366660

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US1998/008834 WO1998049350A1 (en) 1997-04-30 1998-04-30 In vivo use of recombinagenic oligonucleobases to correct genetic lesions in hepatocytes

Country Status (8)

Country Link
EP (1) EP0979311A1 (en)
JP (1) JP2002511851A (en)
KR (1) KR20010020375A (en)
CN (1) CN1268186A (en)
AU (1) AU749410B2 (en)
CA (1) CA2288209A1 (en)
NZ (1) NZ500694A (en)
WO (1) WO1998049350A1 (en)

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Also Published As

Publication number Publication date
AU749410B2 (en) 2002-06-27
CA2288209A1 (en) 1998-11-05
JP2002511851A (en) 2002-04-16
NZ500694A (en) 2001-09-28
CN1268186A (en) 2000-09-27
KR20010020375A (en) 2001-03-15
EP0979311A1 (en) 2000-02-16
WO1998049350A1 (en) 1998-11-05
AU7365498A (en) 1998-11-24

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