{"search_session":{},"preferences":{"l":"en","queryLanguage":"en"},"patentId":"074-747-667-078-413","frontPageModel":{"patentViewModel":{"ref":{"entityRefType":"PATENT","entityRefId":"074-747-667-078-413"},"entityMetadata":{"linkedIds":{"empty":true},"tags":[],"collections":[{"id":10716,"type":"PATENT","title":"Pierre-and-Marie-Curie University Patent Portfolio","description":"","access":"OPEN_ACCESS","displayAvatar":true,"attested":false,"itemCount":6628,"tags":[],"user":{"id":91044780,"username":"Cambialens","firstName":"","lastName":"","created":"2015-05-04T00:55:26.000Z","displayName":"Cambialens","preferences":"{\"usage\":\"public\",\"beta\":false}","accountType":"PERSONAL","isOauthOnly":false},"notes":[{"id":8229,"type":"COLLECTION","user":{"id":91044780,"username":"Cambialens","firstName":"","lastName":"","created":"2015-05-04T00:55:26.000Z","displayName":"Cambialens","preferences":"{\"usage\":\"public\",\"beta\":false}","accountType":"PERSONAL","isOauthOnly":false},"text":"
Search Applicants and Owners separately:\"Uni* Paris vi\";\"Uni* Paris 6\";\"Pierre Marie Curie Uni*\";\"Pie* Mar* Cur* Uni*\".
Select more for logical variants. Add to collection. Select all patents in the collection and expand by simple families. Add to collection. Total patents: 3587
Search Applicants and Owners separately:\"Uni* Paris vi\";\"Uni* Paris 6\";\"Pierre Marie Curie Uni*\";\"Pie* Mar* Cur* Uni*\".
Select more for logical variants. Add to collection. Select all patents in the collection and expand by simple families. Add to collection. Total patents: 3587
a) two target binding domains AS and AS′ that target the binding of the nucleic acid molecule to a target pre-mRNA, wherein said two target binding domains AS and AS′ are located respectively at the 5′-end and at the 3′-end of the nucleic acid molecule,\n
b) a 3′ splice region comprising a branch point, a polypyrimidine tract and a 3′ splice acceptor site,\n
c) a 5′ splice region comprising a 5′ splice donor site,\n
d) a spacer sequence that separates said 3′ splice region from said 5′-end target binding domain AS,\n
e) a spacer sequence that separates said 5′ splice region from said 3′-end target binding domain AS′, said spacer comprising a downstream intronic splice enhancer (DISE) and\n
f) a nucleotide sequence to be trans-spliced to said target pre-mRNA, wherein said nucleotide sequence encodes at least a part of a polypeptide, and is located between said 3′ splice region and said 5′ splice region of said nucleic acid."],"number":1,"annotation":false,"title":false,"claim":true},{"lines":["The nucleic acid molecule according to claim 1, wherein said two target binding domains AS and AS′ target binding of said nucleic acid molecule to the pre-mRNA of the dystrophin (DMD) gene, and wherein the nucleotide sequence to be trans-spliced encodes at least a part of the dystrophin polypeptide."],"number":2,"annotation":false,"title":false,"claim":true},{"lines":["The nucleic acid molecule according to claim 2, wherein said nucleotide sequence to be trans-spliced comprises at least one exon of the DMD gene."],"number":3,"annotation":false,"title":false,"claim":true},{"lines":["The nucleic acid molecule according to claim 2, wherein said nucleotide sequence to be trans-spliced comprises at least the sequence of exon 23 of the DMD gene, or the sequence of exon 70 of the DMD gene."],"number":4,"annotation":false,"title":false,"claim":true},{"lines":["The nucleic acid molecule according to claim 4, wherein said nucleotide sequence to be trans-spliced comprises the nucleotide sequence of exon 23 of the human DMD gene as set forth in SEQ ID NO 60."],"number":5,"annotation":false,"title":false,"claim":true},{"lines":["The nucleic acid molecule according to claim 1, wherein each of said target binding domains AS and AS′ comprises between 100 and 200 nucleotides."],"number":6,"annotation":false,"title":false,"claim":true},{"lines":["The nucleic acid molecule according to claim 6, wherein each of said target binding domains AS and AS′ comprises 150 nucleotides."],"number":7,"annotation":false,"title":false,"claim":true},{"lines":["The nucleic acid molecule according to claim 1, wherein said 5′-end target binding domain AS targets the binding of the nucleic acid to the intron 22 of the pre-mRNA of a DMD gene."],"number":8,"annotation":false,"title":false,"claim":true},{"lines":["The nucleic acid molecule according to claim 1, wherein said 3′-end target binding domain AS′ targets the binding of the nucleic acid to the intron 23 of the pre-mRNA of a DMD gene."],"number":9,"annotation":false,"title":false,"claim":true},{"lines":["The nucleic acid molecule according to claim 1, wherein said 5′-end target binding domain AS comprises at least 20 successive nucleotides of at least one nucleotide sequence chosen among: SEQ ID NO 13 and SEQ ID NO 14."],"number":10,"annotation":false,"title":false,"claim":true},{"lines":["The nucleic acid molecule according to claim 1, wherein said 3′-end target binding domain AS′ targets the binding of the nucleic acid to a nucleotide sequence located in said 5′-half of the nucleotide sequence of intron 23."],"number":11,"annotation":false,"title":false,"claim":true},{"lines":["The nucleic acid molecule according to claim 11, wherein said 3′-end target binding domain AS′ comprises at least 20 successive nucleotides of at least one nucleotide sequence chosen among: SEQ ID NO 16, SEQ ID NO 19, and SEQ ID NO 20."],"number":12,"annotation":false,"title":false,"claim":true},{"lines":["The nucleic acid molecule according to claim 1, wherein said 3′-end target binding domain AS′ comprises at least 20 successive nucleotides of the nucleotide sequence as set forth in SEQ ID NO 21."],"number":13,"annotation":false,"title":false,"claim":true},{"lines":["The nucleic acid molecule according to claim 1, wherein each of the two spacers comprises from 10 to 100 nucleotides."],"number":14,"annotation":false,"title":false,"claim":true},{"lines":["The nucleic acid molecule according to claim 14, wherein each of the two spacers comprises from 30 to 50 nucleotides."],"number":15,"annotation":false,"title":false,"claim":true},{"lines":["The nucleic acid molecule according to claim 1, wherein said branch point is a conserved yeast branch point sequence."],"number":16,"annotation":false,"title":false,"claim":true},{"lines":["The nucleic acid molecule according to claim 16, wherein said branch point has the nucleic acid sequence of SEQ ID NO: 25."],"number":17,"annotation":false,"title":false,"claim":true},{"lines":["A recombinant vector comprising said nucleic acid of claim 1."],"number":18,"annotation":false,"title":false,"claim":true},{"lines":["The vector according to claim 18, wherein said vector is an eukaryotic expression vector."],"number":19,"annotation":false,"title":false,"claim":true},{"lines":["A cell comprising said nucleic acid molecule according to claim 1."],"number":20,"annotation":false,"title":false,"claim":true},{"lines":["The cell according to claim 18, wherein said cell is an eukaryotic cell."],"number":21,"annotation":false,"title":false,"claim":true},{"lines":["The nucleic acid molecule according to claim 1, wherein said spacer separating said 5′ splice donor site and said 3′-end target binding domain AS′ comprises the DISE sequence from the rat FGFR2 gene having the nucleic acid sequence of SEQ ID NO: 27."],"number":22,"annotation":false,"title":false,"claim":true}]}},"filters":{"npl":[],"notNpl":[],"applicant":[],"notApplicant":[],"inventor":[],"notInventor":[],"owner":[],"notOwner":[],"tags":[],"dates":[],"types":[],"notTypes":[],"j":[],"notJ":[],"fj":[],"notFj":[],"classIpcr":[],"notClassIpcr":[],"classNat":[],"notClassNat":[],"classCpc":[],"notClassCpc":[],"so":[],"notSo":[],"sat":[]},"sequenceFilters":{"s":"SEQIDNO","d":"ASCENDING","p":0,"n":10,"sp":[],"si":[],"len":[],"t":[],"loc":[]}}