HUE027124T2 - Eljárások és eszközök a 45-ös exon átugrására, Duchenne-izomdisztrófiában érintett pre-mRNS-ben - Google Patents

Eljárások és eszközök a 45-ös exon átugrására, Duchenne-izomdisztrófiában érintett pre-mRNS-ben Download PDF

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HUE027124T2
HUE027124T2 HUE13160338A HUE13160338A HUE027124T2 HU E027124 T2 HUE027124 T2 HU E027124T2 HU E13160338 A HUE13160338 A HU E13160338A HU E13160338 A HUE13160338 A HU E13160338A HU E027124 T2 HUE027124 T2 HU E027124T2
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leu
glu
oligonucleotide
gin
lys
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Kimpe Josephus Johannes De
Gerardus Johannes Platenburg
Deutekom Judith Christina Theodora Van
Annemieke Aartsma-Rus
Ommen Garrit-Jan Boudewijn Van
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Biomarin Tech Bv
Academisch Ziekenhuis Leiden
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Claims (11)

  1. SsabadaUm sgá»y ρ«»5 o k L Antiszmsz dlipitbkfeodd, amely képes a d&amp;Shpßs pe-Ä:Ni 4§sös mm$m beW a. következő ÂÂà-sxekvaaoiâ Ϊ#!1Ι '21 mikfeotid hosszúságii szatoiÄi^t^Äfe 5'- C~€MtiAG{X}€AUliGGG€A<röü^^ ACAUiXtAAlXjeAACUGGGGAAGAAAUAAUUCAGCAAUC -3! (SBQ ID NO 2) és ahoi az oligaeukieoikt öosszs legalább 21 mäkleotiii é&amp; ahol az amiszeRsz cdigosiukkotki szekvőtóáia oem.a kővetkező szekvenciák valamelyikéből áll: S'-AASOTmiCCOCímcCCAATOCCA-^ s^cinACAA<:AGirTGCCocrccccAAAh S’áiixá3€A3^^ 5!Aie?GAA1 "ΓΑ nTCIlTXlCCAGlXGG- 3* vagy M^TATrrCrrOOCOAGTTÖéAÍlGAAk
  2. 2. Az i. Igénypon! szerinti aikiszeosz oHgonukieotid, ahol az oligonukleooő hossza 21-50 mskleotid.
  3. 3. Az 1. vagy 2. ikrtáteazza a kÓvcAezök közöl kivilaszioiklkrsioiyik bäzisszelveöÄ Vágy abból áll: 5’ AUUCA AllGlíyOÖOAGAAGAÓÜOOÖC 3! (SÜ| p»4|; 5! CCAÖtiUGO AOOCAAüGOeeilGACAA 3’ tbEQlONG:: 5f CAGüi ^ G C A üli C A AG G ü U€ ü GAC 3’ ÍSBQ ÍD NO: 6} és :V GAIJUGCUGAAUUAUOUCUUCC 3‘ (SEQ ÍD NO: m
  4. 4. Az 1-3, igéoypimiök bármelyike szériáit amiszensz eíigömrRlesnkb ahol isgáMbb egy a sukleolMok közűi nukNodd-amdög.
  5. 5,. Az 1-4, igénypöHitAlikptpelyike szeriöb antisztmsz öíigsfíöfeDóiid;, amely tartalmaz egy vagy több RNS-nokleotidoí vagy DNX-mAíeoudot A. A 4. igéeypon· v'.enntt antíszeosz oUgomiklec-tid, alibi a raAleotiö-analég tóeghaíárózásk a kővetkező: módosított bázis! ős.'vagy módosított ajkorosoportot ésNagy módosított· iiitetTiUkleozídAÁtesí ImtáJsoazé pökleoíid, I, A 6, igénypont szériád antiszéttsz bitgttipÄotiö, abol: &amp; pokieoiid-aaáfög: tartalmaz módosított bázist és/vsgy Bst««mfcleo?Jó-fcőtó8k t, A 4-?, igénypontok toaeíyto szedné ^pttszettaz: ollgoa«k!eonds ahol a .®É!e»ifc!~anaÍ6g taristok ggy vagy több, a 2X 3’- észvagy 5'-pozícióba« egyszeressn vagy kétszeresen szobszÉwált enkorcsöpörtöt tartalmaz.
  6. 9. Az i-8. igénypontok bármelyike szerinti amlszensz: ôlgonakleotid, ante ív tartalmaz Z’-O-sztsbszi sutáit foszfortíoát ardiszeosz oíigoímkieobdöp f ii, A 9. igátypont szeritől amiszensz oligortskleoíki, amely iaríáímaz 2?*O^!Ílí*!fwforiÍ0Éi||jNSKÍ> II, Az 1-10. igénypontok Mntmíyíke szerinti aptiszeasz: oltgpöekieotisi amely îartateaz dbszioriisGî sntiszeosz NigonuRleotldot, aboi a cöfcmzsopöriok mindegyike 3UO-mgîil-szobástkulik és tarialtnäzza a kővetkező bázisszekveouiák bármelyikét; 5’ AUUCAAüGliGCUGACAACAGUUUGC 3<|SEQ 1D NíM), 5! CCAÔUUGCAUUCAAUGÜUCUGACÂÂ' 3*''CSEQ;1D Ä5), 5' CAGUÜGCAÜUCAAUGUÜCUGAC 3' (SIR): 3D NO: 6) és N GAUUtsOJGAAliyAUUUCUUCC 3' (SHQ ID NO: 8).
  7. 12. Anószensx oligotiukieoiid., amely fezibít ioáí aatisxeasz oligimukleobd., aböl a eukorcsoponok 2!-Ö-meti l-szubsztimadak, és Mzlsszekveteála: ^ következő Mzisszekveimiák Mrn?slf íte: ^AUOCAAUSyUCliOASAASAOiffiPeC 3'pEQ ÏD UÖ-A'k siccÂGmœMÂmhÂVGmcmmÂÂ. y peq mm: s>, $ eAöu'uocAmcAA uguucuo« r (sbq m m: 6> és 5! GAÜUGCUGAAÜUAUUUCUUCC 3‘ f$EQ 10 NO: 8). 1:3. A 7-9. igä®fäm(®k?bämetyike szmöi mûsmm ôU^màMotià,Æ&amp;t» mMmimi imemukieozid kötés » kővetkezőkből álló csoportból vari kiválasztva: mortöíiaogemc., karbamátgerínc, szrloxangerine. szül ftdger Inc, szalíoxidgerinc.. ssalfortgerme, formacetilgeme, {iofonoacatii-görusc. sBCitlénlbrmacöni'-gerisó. óksasaikgériao^ alMat tartalmáéi gér»,. szíAÉmátgemíe, szölkmátgcr». sAÄöäiaidgcrme. metiMóiimsáS* geAfic, mctijéRhítteiaö-geritio ós aiaÜgeAsö.
  8. 14. Az í-MS. ígéöypöstök MiaaÉvlk« vàgÿ s 13. igépypom: szériái «atissxmsz »bgoaukieoild, amely tartalmaz feszftirá0msöát ariorlölí8ö-Aligöí8öFt {PMÖJ, peptíd-oekieinsav&amp;t (FN A) és/vagy zárt β#Μΐ: (LN A).
  9. 15. Vírosaiapú vektor, amely tartalmaz egy, az !.. 2, vagy 1 igélypoitihart maghatározott anbszöosx ölsgea«kleö5i(íó>p®ssziágái vezérlő expresszié:* kazettát lő. Gyógyászig! készítmény, ataely tartalmaz az 1-14. igénypontok bármelyikében meglatátözotí antisáessz ollgtmukleoíidot és/vagy a |;#> sgÍsypoíÁ4»M vektort és ^péS5»t8ag.æl-^iftÉS.*^Â
  10. 17, A ló, IpRypötii ssstrírttí gyógyiszgikészgpiéíÍy, amely olyast M -*ar- tahnaz, amely képes egy páeieas Í8^éÉa;;|iit«ö^Íf#áí » r-es, 44-es, 46 -as, 5 S -es, 63¾ 59¾ vagy ö7-es (amlgi azt a 2, táblázat bmm&amp;4i}. mm árugrását indokaim vágy éllsegdent, vagy tanain» olya« vlrusalapó vektortis, amely tartalmaz egyi: az ardisrnnsz öigtarukleoóá: o^ressdégál vezérli: sxpressziós kazettát, Api a késxhmenv képes legalább két célzott esőn kizárására a dtsztetfo pre-mlbNS-höl, előnyösén; ahol g késsapssésty képes a pce-m&amp;NS-ben a két célzói! extmköAM teljes: régió teázására. IS. Az 1-14. igénypontok Mmmlyikéhan meghatározett wÉsmm. oligernÄmid, á ΙΑ. ígénygösí szériát! vektor vagy a. ló. Igénypont szerinti gyógyászati: kíteltmény, a 45-ös exen ámgrásáíi^í mdnMtásara vagy elősegitésére, BMÚA>m vagy :8M:D~ösm szenvedd páeiíms kezelésében történd alkalmazásra,
    13, A ifi igénypont szerintj késxftsbépy á diszímfso pfe-mí&amp;NS-ööl, legalább két célzott mm átogfósáraj eíöpyöiseö: á.pre-nrlÍlNS-bso: a két célzott exms k&amp;íóöi teljes régió stpgrására, DMG-feen vagy BMD- ben iszébvétiö: kStátoasé®^..
  11. 20. In vssro eljárás á disz-rolm prcmRMS 45-ös exonja árugrásának indukáláxára vagy elősegítésére a páciens Izolált séfjében:, ábó! áz eljárás magában foglalja &amp; sejt ellátását 1-14. Igénypontok báromlyikeben »ghatárözbtí aplisZétisz ötigötíákleoiidtlak &amp; 15. igénypont: szeAntii vektorral vagy a 16, igénypont szermti
HUE13160338A 2007-10-26 2009-01-13 Eljárások és eszközök a 45-ös exon átugrására, Duchenne-izomdisztrófiában érintett pre-mRNS-ben HUE027124T2 (hu)

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