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1. WO2008045548 - CODON OPTIMIZED CFTR

Publication Number WO/2008/045548
Publication Date 17.04.2008
International Application No. PCT/US2007/021862
International Filing Date 12.10.2007
IPC
C07K 14/47 2006.01
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
46from vertebrates
47from mammals
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
CPC
A61K 48/005
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
005characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
A61P 11/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
11Drugs for disorders of the respiratory system
C07K 14/4712
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
46from vertebrates
47from mammals
4701not used
4712Cystic fibrosis
C12N 2830/50
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
2830Vector systems having a special element relevant for transcription
50regulating RNA stability, not being an intron, e.g. poly A signal
C12N 2840/44
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
2840Vectors comprising a special translation-regulating system
44being a specific part of the splice mechanism, e.g. donor, acceptor
Applicants
  • COPERNICUS THERAPEUTICS INC. [US]/[US] (AllExceptUS)
  • COOPER, Mark, J. [US]/[US] (UsOnly)
  • PADEGIMAS, Linas [US]/[US] (UsOnly)
Inventors
  • COOPER, Mark, J.
  • PADEGIMAS, Linas
Agents
  • KAGAN, Sarah, A.
Priority Data
60/851,05512.10.2006US
60/885,82719.01.2007US
60/907,85219.04.2007US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) CODON OPTIMIZED CFTR
(FR) CFTR À OPTIMISATION PAR CODON
Abstract
(EN)
A synthetic hCFTR DNA sequence has been developed that produces remarkably high levels of hCFTR mRNA and protein in dosed murine lungs and human cells in culture compared to the natural hCFTR cDNA. This synthetic DNA addresses.problems inherent in some natural cDNAs, such as premature transcriptional truncation sites introduced during cDNA synthesis. Introns are initially present in mRNA until the mRNA is processed. cDNA made from processed mRNA is devoid of introns. Thus DNA sequences (exon junctions) are present in a cDNA molecule which are not present in cells in nature. These exon junctions may affect transcription. Methods for improving expression of CFTR are based on sequence changes in cDNA molecules. The improvement methods may be applied to other cDNA molecules which are refractory to in vivo expression efforts. Compositions embodying the sequence changes increase the production of both transgenic mRNA and protein from cDNA molecules.
(FR)
On a développé une séquence d'ADN hCFTR synthétique qui produit des niveaux remarquablement élevés de d'ARNm hCFTR et de protéine dans des cellules humaines et pulmonaires murines en culture par comparaison avec l'ADNc hCFTR naturel. Cet ADN synthétique permet de régler des problèmes inhérents à certains ADNc naturels, comme l'introduction de sites de troncature transcriptionnelle prématurée durant la synthèse de l'ADNc. Des introns sont présents initialement dans l'ARNm jusqu'au traitement de l'ARNm. L'ADNc issu d'ARNm traitée est dépourvu d'introns. Ainsi, des séquences d'ADN (jonctions d'exons) sont présentes dans une molécule d'ADNc, qui ne sont pas présentes dans les cellules à l'état naturel. Ces jonctions d'exons peuvent affecter la transcription. Les procédés d'amélioration de l'expression de CFTR reposent sur des changements de séquences dans les molécules d'ADNc. Il est possible d'appliquer des procédés d'amélioration à d'autres molécules d'ADNc qui sont réfractaires aux efforts d'expression in vivo. Des compositions incorporant les changements de séquences et augmentent la production d'ARNm transgénique et de protéine à partir des molécules d'ADNc.
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