C12N2830/50- Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g.C12N2830/00- Vector systems having a special element relevant for transcription.C12N2750/14171- Demonstrated in vivo effect.C12N2750/14143- Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector.
C12N2750/14141- Use of virus, viral particle or viral elements as a vector. C12N2750/00- MICROORGANISMS OR ENZYMES COMPOSITIONS THEREOF PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS MUTATION OR GENETIC ENGINEERING CULTURE MEDIA ssDNA viruses. branched-chain-alpha-ketoacid dehydrogenase C12Y102/04- Oxidoreductases acting on the aldehyde or oxo group of donors (1.2) with a disulfide as acceptor (1.2.4). C12Y102/00- Oxidoreductases acting on the aldehyde or oxo group of donors (1.2). C12N15/85- Vectors or expression systems specially adapted for eukaryotic hosts for animal cells. C12N15/79- Vectors or expression systems specially adapted for eukaryotic hosts. C12N15/63- Introduction of foreign genetic material using vectors Vectors Use of hosts therefor Regulation of expression. plasmids, or their isolation, preparation or purification Use of hosts therefor C12N15/00- Mutation or genetic engineering DNA or RNA concerning genetic engineering, vectors, e.g. A61P3/00- Drugs for disorders of the metabolism. A61P13/00- Drugs for disorders of the urinary system. A61P- SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS. A61K38/43- Enzymes Proenzymes Derivatives thereof. A61K38/16- Peptides having more than 20 amino acids Gastrins Somatostatins Melanotropins Derivatives thereof. A61K38/00- Medicinal preparations containing peptides. A61K- PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES. A61- MEDICAL OR VETERINARY SCIENCE HYGIENE. C12N9/0008- Oxidoreductases (1.) acting on the aldehyde or oxo group of donors (1.2). C12N9/00- Enzymes Proenzymes Compositions thereof Processes for preparing, activating, inhibiting, separating or purifying enzymes. C12N- MICROORGANISMS OR ENZYMES COMPOSITIONS THEREOF PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS MUTATION OR GENETIC ENGINEERING CULTURE MEDIA. C12- BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION OR GENETIC ENGINEERING. 
Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.) Filing date Publication date Priority claimed from EP20305201.4 external-priority Application filed by INSERM (Institut National de la Santé et de la Recherche Médicale), Assistance Publique-Hôpitaux De Paris (Aphp), Fondation Imagine, Université de Paris filed Critical INSERM (Institut National de la Santé et de la Recherche Médicale) Priority to EP21707274.3A priority Critical patent/EP4110373A1/en Priority to JP2022551246A priority patent/JP2023515820A/en Priority to US17/904,124 priority patent/US20230136699A1/en Publication of WO2021170784A1 publication Critical patent/WO2021170784A1/en Links Inventor Manuel SCHIFF Marina Cavazzana Pascale DE LONLAY-DEBENEY Marcelo SIMON SOLA Clément PONTOIZEAU Chris OTTOLENGHI Original Assignee INSERM (Institut National de la Santé et de la Recherche Médicale) Assistance Publique-Hôpitaux De Paris (Aphp) Fondation Imagine Université de Paris Priority date (The priority date is an assumption and is not a legal conclusion. Google Patents WO2021170784A1 - Gene therapy for maple syrup urine disease WO2021170784A1 - Gene therapy for maple syrup urine disease
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