WO1997041150A3 - Gene therapy for mitochondrial dna deffects using peptide nucleic acids - Google Patents

Gene therapy for mitochondrial dna deffects using peptide nucleic acids Download PDF

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Publication number
WO1997041150A3
WO1997041150A3 PCT/GB1997/001102 GB9701102W WO9741150A3 WO 1997041150 A3 WO1997041150 A3 WO 1997041150A3 GB 9701102 W GB9701102 W GB 9701102W WO 9741150 A3 WO9741150 A3 WO 9741150A3
Authority
WO
WIPO (PCT)
Prior art keywords
nucleic acids
peptide nucleic
mitochondrial dna
deffects
gene therapy
Prior art date
Application number
PCT/GB1997/001102
Other languages
French (fr)
Other versions
WO1997041150A2 (en
Inventor
Douglas Matthew Turnbull
Robert Neil Lightowlers
Robert William Taylor
Original Assignee
Univ Newcastle
Douglas Matthew Turnbull
Robert Neil Lightowlers
Robert William Taylor
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 Newcastle, Douglas Matthew Turnbull, Robert Neil Lightowlers, Robert William Taylor filed Critical Univ Newcastle
Publication of WO1997041150A2 publication Critical patent/WO1997041150A2/en
Publication of WO1997041150A3 publication Critical patent/WO1997041150A3/en

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004Oxidoreductases (1.)
    • C12N9/0053Oxidoreductases (1.) acting on a heme group of donors (1.9)
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/001Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof by chemical synthesis
    • C07K14/003Peptide-nucleic acids (PNAs)
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy

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  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Toxicology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

The invention concerns a method for selectively preventing the replication of mitochondrial DNA using complementary peptide nucleic acids; and the peptide nucleic acids for use in the method. Additionally novel targeting means are described for ensuring the targeting of the peptide nucleic acids into mitochondria.
PCT/GB1997/001102 1996-04-27 1997-04-22 Gene therapy for mitochondrial dna deffects using peptide nucleic acids WO1997041150A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GBGB9608803.4A GB9608803D0 (en) 1996-04-27 1996-04-27 Mitochondrial dna defects
GB9608803.4 1996-04-27

Publications (2)

Publication Number Publication Date
WO1997041150A2 WO1997041150A2 (en) 1997-11-06
WO1997041150A3 true WO1997041150A3 (en) 1997-12-04

Family

ID=10792795

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/GB1997/001102 WO1997041150A2 (en) 1996-04-27 1997-04-22 Gene therapy for mitochondrial dna deffects using peptide nucleic acids

Country Status (2)

Country Link
GB (1) GB9608803D0 (en)
WO (1) WO1997041150A2 (en)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6472209B1 (en) 1997-10-17 2002-10-29 Mayo Foundation For Medical Education And Research Using polyamide nucleic acid oligomers to engender a biological response
US6723560B2 (en) 1998-10-08 2004-04-20 Mayo Foundation For Medical Education And Research Using polyamide nucleic acid oligomers to engender a biological response
US6989270B1 (en) 1997-10-17 2006-01-24 Mayo Foundation For Medical Education And Research Using polyamide nucleic acid oligomers to engender a biological response
EP1574210B1 (en) 1999-02-26 2016-04-06 Novartis Vaccines and Diagnostics, Inc. Microemulsions with adsorbed macromolecules

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1992020702A1 (en) * 1991-05-24 1992-11-26 Ole Buchardt Peptide nucleic acids
WO1995034665A2 (en) * 1994-06-16 1995-12-21 Peter Seibel Chimerical peptide-nucleic acid fragment, process for producing the same and its use for appropriately introducing nucleic acids into cell organelles and cells
WO1996004000A1 (en) * 1994-08-05 1996-02-15 The Regents Of The University Of California PEPTIDE-BASED NUCLEIC ACID MIMICS (PENAMs)
EP0700928A1 (en) * 1994-08-08 1996-03-13 Bayer Ag Nucleic acid-binding oligomers for therapy and diagnostics

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1992020702A1 (en) * 1991-05-24 1992-11-26 Ole Buchardt Peptide nucleic acids
WO1995034665A2 (en) * 1994-06-16 1995-12-21 Peter Seibel Chimerical peptide-nucleic acid fragment, process for producing the same and its use for appropriately introducing nucleic acids into cell organelles and cells
WO1996004000A1 (en) * 1994-08-05 1996-02-15 The Regents Of The University Of California PEPTIDE-BASED NUCLEIC ACID MIMICS (PENAMs)
EP0700928A1 (en) * 1994-08-08 1996-03-13 Bayer Ag Nucleic acid-binding oligomers for therapy and diagnostics

Also Published As

Publication number Publication date
WO1997041150A2 (en) 1997-11-06
GB9608803D0 (en) 1996-07-03

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