INDUCTION OF EXON SKIPPING IN EUKARYOTIC CELLS
The present invention provides a method for at least in part decreasing the production of an aberrant protein in a cell, said cell comprising pre-mRNA comprising exons coding for said protein, by inducing so-called exon skipping in said cell. Exon-skipping results in mature mRNA that does not contai...
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| creator | JOHANNES THEODORUS DEN DUNNEN JUDITH CHRISTINA THEODORA VAN DEUTEKOM GARRIT-JAN BOUDEWIJN VAN OMMEN |
| description | The present invention provides a method for at least in part decreasing the production of an aberrant protein in a cell, said cell comprising pre-mRNA comprising exons coding for said protein, by inducing so-called exon skipping in said cell. Exon-skipping results in mature mRNA that does not contain the skipped exon which leads to an altered product if said exon codes for amino acids. Exon skipping is performed by providing a cell with an agent capable of specifically inhibiting an exon inclusion signal, for instance an exon recognition sequence, of said exon. Said exon inclusion signal can be interfered with by a nucleic acid comprising complementarity to a part of said exon. Said nucleic acid, which is also herewith provided, can be used for the preparation of a medicament, for instance for the treatment of an inherited disease. |
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Exon-skipping results in mature mRNA that does not contain the skipped exon which leads to an altered product if said exon codes for amino acids. Exon skipping is performed by providing a cell with an agent capable of specifically inhibiting an exon inclusion signal, for instance an exon recognition sequence, of said exon. Said exon inclusion signal can be interfered with by a nucleic acid comprising complementarity to a part of said exon. Said nucleic acid, which is also herewith provided, can be used for the preparation of a medicament, for instance for the treatment of an inherited disease.</description><language>eng ; por</language><subject>AGRICULTURE ; ANIMAL HUSBANDRY ; BEER ; BIOCHEMISTRY ; CARE OF BIRDS, FISHES, INSECTS ; CHEMISTRY ; COMPOSITIONS OR TEST PAPERS THEREFOR ; COMPOSITIONS THEREOF ; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL ORENZYMOLOGICAL PROCESSES ; CULTURE MEDIA ; ENZYMOLOGY ; FISHING ; FORESTRY ; HUMAN NECESSITIES ; HUNTING ; HYGIENE ; MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEICACIDS OR MICROORGANISMS ; MEDICAL OR VETERINARY SCIENCE ; METALLURGY ; MICROBIOLOGY ; MICROORGANISMS OR ENZYMES ; MUTATION OR GENETIC ENGINEERING ; NEW BREEDS OF ANIMALS ; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES ; PROCESSES OF PREPARING SUCH COMPOSITIONS ; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS ; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR ; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS ; SPIRITS ; TRAPPING ; VINEGAR ; WINE</subject><creationdate>2017</creationdate><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktohtml>$$Uhttps://worldwide.espacenet.com/publicationDetails/biblio?FT=D&date=20170725&DB=EPODOC&CC=PT&NR=2801618T$$EHTML$$P50$$Gepo$$Hfree_for_read</linktohtml><link.rule.ids>230,308,780,885,25563,76318</link.rule.ids><linktorsrc>$$Uhttps://worldwide.espacenet.com/publicationDetails/biblio?FT=D&date=20170725&DB=EPODOC&CC=PT&NR=2801618T$$EView_record_in_European_Patent_Office$$FView_record_in_$$GEuropean_Patent_Office$$Hfree_for_read</linktorsrc></links><search><creatorcontrib>JOHANNES THEODORUS DEN DUNNEN</creatorcontrib><creatorcontrib>JUDITH CHRISTINA THEODORA VAN DEUTEKOM</creatorcontrib><creatorcontrib>GARRIT-JAN BOUDEWIJN VAN OMMEN</creatorcontrib><title>INDUCTION OF EXON SKIPPING IN EUKARYOTIC CELLS</title><description>The present invention provides a method for at least in part decreasing the production of an aberrant protein in a cell, said cell comprising pre-mRNA comprising exons coding for said protein, by inducing so-called exon skipping in said cell. Exon-skipping results in mature mRNA that does not contain the skipped exon which leads to an altered product if said exon codes for amino acids. Exon skipping is performed by providing a cell with an agent capable of specifically inhibiting an exon inclusion signal, for instance an exon recognition sequence, of said exon. Said exon inclusion signal can be interfered with by a nucleic acid comprising complementarity to a part of said exon. Said nucleic acid, which is also herewith provided, can be used for the preparation of a medicament, for instance for the treatment of an inherited disease.</description><subject>AGRICULTURE</subject><subject>ANIMAL HUSBANDRY</subject><subject>BEER</subject><subject>BIOCHEMISTRY</subject><subject>CARE OF BIRDS, FISHES, INSECTS</subject><subject>CHEMISTRY</subject><subject>COMPOSITIONS OR TEST PAPERS THEREFOR</subject><subject>COMPOSITIONS THEREOF</subject><subject>CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL ORENZYMOLOGICAL PROCESSES</subject><subject>CULTURE MEDIA</subject><subject>ENZYMOLOGY</subject><subject>FISHING</subject><subject>FORESTRY</subject><subject>HUMAN NECESSITIES</subject><subject>HUNTING</subject><subject>HYGIENE</subject><subject>MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEICACIDS OR MICROORGANISMS</subject><subject>MEDICAL OR VETERINARY SCIENCE</subject><subject>METALLURGY</subject><subject>MICROBIOLOGY</subject><subject>MICROORGANISMS OR ENZYMES</subject><subject>MUTATION OR GENETIC ENGINEERING</subject><subject>NEW BREEDS OF ANIMALS</subject><subject>PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES</subject><subject>PROCESSES OF PREPARING SUCH COMPOSITIONS</subject><subject>PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS</subject><subject>REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR</subject><subject>SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS</subject><subject>SPIRITS</subject><subject>TRAPPING</subject><subject>VINEGAR</subject><subject>WINE</subject><fulltext>true</fulltext><rsrctype>patent</rsrctype><creationdate>2017</creationdate><recordtype>patent</recordtype><sourceid>EVB</sourceid><recordid>eNrjZNDz9HMJdQ7x9PdT8HdTcI0A0sHengEBnn7uCp5-Cq6h3o5Bkf4hns4Kzq4-PsE8DKxpiTnFqbxQmptBwc01xNlDN7UgPz61uCAxOTUvtSQ-IMTIwsDQzNAiJMSYCCUAqb4l5g</recordid><startdate>20170725</startdate><enddate>20170725</enddate><creator>JOHANNES THEODORUS DEN DUNNEN</creator><creator>JUDITH CHRISTINA THEODORA VAN DEUTEKOM</creator><creator>GARRIT-JAN BOUDEWIJN VAN OMMEN</creator><scope>EVB</scope></search><sort><creationdate>20170725</creationdate><title>INDUCTION OF EXON SKIPPING IN EUKARYOTIC CELLS</title><author>JOHANNES THEODORUS DEN DUNNEN ; JUDITH CHRISTINA THEODORA VAN DEUTEKOM ; GARRIT-JAN BOUDEWIJN VAN OMMEN</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-epo_espacenet_PT2801618TT3</frbrgroupid><rsrctype>patents</rsrctype><prefilter>patents</prefilter><language>eng ; por</language><creationdate>2017</creationdate><topic>AGRICULTURE</topic><topic>ANIMAL HUSBANDRY</topic><topic>BEER</topic><topic>BIOCHEMISTRY</topic><topic>CARE OF BIRDS, FISHES, INSECTS</topic><topic>CHEMISTRY</topic><topic>COMPOSITIONS OR TEST PAPERS THEREFOR</topic><topic>COMPOSITIONS THEREOF</topic><topic>CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL ORENZYMOLOGICAL PROCESSES</topic><topic>CULTURE MEDIA</topic><topic>ENZYMOLOGY</topic><topic>FISHING</topic><topic>FORESTRY</topic><topic>HUMAN NECESSITIES</topic><topic>HUNTING</topic><topic>HYGIENE</topic><topic>MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEICACIDS OR MICROORGANISMS</topic><topic>MEDICAL OR VETERINARY SCIENCE</topic><topic>METALLURGY</topic><topic>MICROBIOLOGY</topic><topic>MICROORGANISMS OR ENZYMES</topic><topic>MUTATION OR GENETIC ENGINEERING</topic><topic>NEW BREEDS OF ANIMALS</topic><topic>PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES</topic><topic>PROCESSES OF PREPARING SUCH COMPOSITIONS</topic><topic>PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS</topic><topic>REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR</topic><topic>SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS</topic><topic>SPIRITS</topic><topic>TRAPPING</topic><topic>VINEGAR</topic><topic>WINE</topic><toplevel>online_resources</toplevel><creatorcontrib>JOHANNES THEODORUS DEN DUNNEN</creatorcontrib><creatorcontrib>JUDITH CHRISTINA THEODORA VAN DEUTEKOM</creatorcontrib><creatorcontrib>GARRIT-JAN BOUDEWIJN VAN OMMEN</creatorcontrib><collection>esp@cenet</collection></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext_linktorsrc</fulltext></delivery><addata><au>JOHANNES THEODORUS DEN DUNNEN</au><au>JUDITH CHRISTINA THEODORA VAN DEUTEKOM</au><au>GARRIT-JAN BOUDEWIJN VAN OMMEN</au><format>patent</format><genre>patent</genre><ristype>GEN</ristype><title>INDUCTION OF EXON SKIPPING IN EUKARYOTIC CELLS</title><date>2017-07-25</date><risdate>2017</risdate><abstract>The present invention provides a method for at least in part decreasing the production of an aberrant protein in a cell, said cell comprising pre-mRNA comprising exons coding for said protein, by inducing so-called exon skipping in said cell. Exon-skipping results in mature mRNA that does not contain the skipped exon which leads to an altered product if said exon codes for amino acids. Exon skipping is performed by providing a cell with an agent capable of specifically inhibiting an exon inclusion signal, for instance an exon recognition sequence, of said exon. Said exon inclusion signal can be interfered with by a nucleic acid comprising complementarity to a part of said exon. Said nucleic acid, which is also herewith provided, can be used for the preparation of a medicament, for instance for the treatment of an inherited disease.</abstract><oa>free_for_read</oa></addata></record> |
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| subjects | AGRICULTURE ANIMAL HUSBANDRY BEER BIOCHEMISTRY CARE OF BIRDS, FISHES, INSECTS CHEMISTRY COMPOSITIONS OR TEST PAPERS THEREFOR COMPOSITIONS THEREOF CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL ORENZYMOLOGICAL PROCESSES CULTURE MEDIA ENZYMOLOGY FISHING FORESTRY HUMAN NECESSITIES HUNTING HYGIENE MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEICACIDS OR MICROORGANISMS MEDICAL OR VETERINARY SCIENCE METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING NEW BREEDS OF ANIMALS PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES PROCESSES OF PREPARING SUCH COMPOSITIONS PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS SPIRITS TRAPPING VINEGAR WINE |
| title | INDUCTION OF EXON SKIPPING IN EUKARYOTIC CELLS |
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