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1. (WO1997018319) JOINT EXPRESSION OF GTP CYCLOHYDROLASE AND TYROSINE HYDROXYLASE
Latest bibliographic data on file with the International Bureau

Pub. No.: WO/1997/018319 International Application No.: PCT/US1996/017696
Publication Date: 22.05.1997 International Filing Date: 01.11.1996
Chapter 2 Demand Filed: 06.06.1997
IPC:
A61K 38/00 (2006.01) ,A61K 48/00 (2006.01) ,C12N 9/02 (2006.01) ,C12N 9/78 (2006.01)
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38
Medicinal preparations containing peptides
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
9
Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
02
Oxidoreductases (1.), e.g. luciferase
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
9
Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
14
Hydrolases (3.)
78
acting on carbon to nitrogen bonds other than peptide bonds (3.5)
Applicants:
SOMATIX THERAPY CORPORATION [US/US]; Suite 100 950 Marina Village Parkway Alameda, CA 94501, US
Inventors:
LEFF, Stuart, E.; US
MANDEL, Ron; US
Agent:
HALLUIN, Albert, P. ; Pennie & Edmonds 1155 Avenue of the Americas New York, NY 10036, US
Priority Data:
557,53014.11.1995US
Title (EN) JOINT EXPRESSION OF GTP CYCLOHYDROLASE AND TYROSINE HYDROXYLASE
(FR) EXPRESSION CONJOINTE DE LA GTP-CYCLOHYDROLASE ET DE LA TYROSINE-HYDROXYLASE
Abstract:
(EN) The invention provides novel genetically modified host cells, vectors, and methods for the treatment of Parkinson's disease and other related disease conditions in which the dopamine production is deficient. The invention relates to the surprising discovery that coexpression of tyrosine hydroxylase and GTP cyclohydrolase enables host cells to produce elevated dopamine and L-DOPA without adding tetrahydrobiopterin and that the coexpression of GTP cyclohydrolase stabilizes tyrosine hydroxylase. One embodiment of the invention is genetically modified host cells containing heterologous polynucleotide sequences encoding and capable of expressing GTP cyclohydrolase and tyrosine hydroxylase. Other embodiments of the invention include genetic construction that contain and can simultaneously express polynucleotide sequences encoding a GTP cyclohydrolase and a tyrosine hydroxylase. The invention also provides methods of producing L-DOPA or dopamine using the host cells of the invention. The invention also provides methods of treating patients of Parkinson's disease or other diseases related to a dopamine production deficiency using the vectors and methods of the invention.
(FR) L'invention concerne de nouvelles cellules hôtes génétiquement modifiées des vecteurs et des méthodes de traitement de la maladie de Parkinson et d'autres pathologies apparentées dans lesquelles la production de dopamine est insuffisante. L'invention concerne la découverte surprenante suivante: la coexpression de la tyrosine-hydroxylase et de la GTP-hydrolase qui permet aux cellules hôtes de produire des quantités élevées de dopamine et de L-DOPA sans adjonction de tétrahydrobioptérine, et la coexpression de la GTP-cyclohydrolase qui stabilise la tyrosine-hydroxylase. Un des modes de réalisation est représenté par des cellules hôtes modifiées génétiquement contenant des séquences de polynucléotides hétérologues codant pour la GTP-cyclohydrolase et la tyrosine-hydroxylase et capables de les exprimer. D'autres modes de réalisation comprennent une construction génétique qui contient et peut simultanément exprimer des séquences de polynucléotides codant une GTP-hydrolase et une tyrosine-hydroxylase. L'invention concerne aussi des procédés de production de L-dopa ou de dopamine à l'aide des cellules hôtes de l'invention. L'invention concerne également des méthodes de traitement de patients atteints de maladie de Parkinson ou d'autres pathologies apparentées avec une insuffisance de la production de dopamine à l'aide des vecteurs et des méthodes de l'invention.
Designated States: AU, CA, JP
European Patent Office (AT, BE, CH, DE, DK, ES, FI, FR, GB, GR, IE, IT, LU, MC, NL, PT, SE)
Publication Language: English (EN)
Filing Language: English (EN)
Also published as:
AU1997010737