JP2020517720A5 - - Google Patents
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- JP2020517720A5 JP2020517720A5 JP2019558574A JP2019558574A JP2020517720A5 JP 2020517720 A5 JP2020517720 A5 JP 2020517720A5 JP 2019558574 A JP2019558574 A JP 2019558574A JP 2019558574 A JP2019558574 A JP 2019558574A JP 2020517720 A5 JP2020517720 A5 JP 2020517720A5
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Claims (20)
- (i)脆弱なCD34+造血幹細胞(HSC)集団及び/又はCD34+ Lo HSC集団を有する対象から取得したCD34+細胞を含む生体試料から、CD3+、CD14+、CD16+及びCD19+細胞のみを除去する工程;
(ii)前記生体試料内に残ったCD34+細胞を、治療用遺伝子を含むウイルスベクターを前記残ったCD34+細胞のゲノムに挿入することによって遺伝子改変する工程;並びに
(iii)前記遺伝子改変されたCD34+細胞を、前記対象への投与のために製剤化する工程を含む、方法。 - 前記対象がファンコーニ貧血を有するヒト患者である、請求項1に記載の方法。
- 治療用遺伝子が、FancA、FancB、FancC、FancD1、FancD2、FancE、FancF、FancG、FancI、FancJ、FancL、FancM、FancN、FancO、FancP、FancQ、FancR、FancS、FancT、FancU、FancV又はFancWを含む、請求項1に記載の方法。
- 前記除去する工程が、CD3に結合する結合タンパク質、CD14に結合する結合タンパク質、CD16に結合する結合タンパク質及びCD19に結合する結合タンパク質からなる結合タンパク質と、前記生体試料を接触させる工程を含み、結合タンパク質が、磁気ビーズ、フルオロフォア及び/又は親和性タグに結合されている、請求項1に記載の方法。
- 前記除去する工程が、磁気分離、蛍光活性化細胞選別(FACs)、ナノ選別、アフィニティークロマトグラフィー、パニング及び/又は選択的凝集を実施する工程を含む、請求項1に記載の方法。
- 前記ウイルスベクターが、レンチウイルスベクターである、請求項1に記載の方法。
- 前記レンチウイルスベクターが、配列番号56で表される配列を有する、ヒトホスホグリセリン酸キナーゼ(PGK)プロモータを含む、請求項6に記載の方法。
- 前記生体試料が、骨髄試料又は動員末梢血試料である、請求項1に記載の方法。
- 治療的又は実験的目的のために、アクセサリー細胞を含むCD34+細胞集団を形成する工程を含む方法であって、
該方法は、脆弱なCD34+細胞及び/又はCD34+ Lo 細胞集団を含む骨髄試料又は動員末梢血試料から、赤血球を除去する工程;及びその後、
(i)CD45RA+細胞のみ、
(ii)CD3+、CD14+及びCD19+細胞のみ、又は
(iii)CD3+、CD14+、CD16+及び及びCD19+細胞のみ
を試料から除去する工程;
を含み、
これにより、治療的又は実験的目的のために、アクセサリー細胞を含むCD34+細胞集団を形成する方法。 - アクセサリー細胞が間葉系幹細胞である、請求項9に記載の方法。
- CD34+細胞集団内のCD34+細胞が、CD34+、CD45RA−及びCD90+である、請求項9に記載の方法。
- CD34+細胞集団内のCD34+細胞を遺伝子改変する工程をさらに含む、請求項9に記載の方法。
- 前記遺伝子改変する工程が、レンチウイルスベクターをCD34+細胞に形質導入する工程を含む、請求項12に記載の方法。
- 前記レンチウイルスベクターが、配列番号31、32、33、34、35、41、42、43、46、47、48、49又は50で表される配列を含む、請求項13に記載の方法。
- 前記レンチウイルスベクターが、配列番号36、37、38、39、40、44、45、51、52、53、54又は55で表される配列を有するタンパク質をコードする配列を含む、請求項13に記載の方法。
- 前記レンチウイルスベクターが、治療用遺伝子を含む、請求項13に記載の方法。
- 前記治療用遺伝子が、FancA、FancB、FancC、FancD1、FancD2、FancE、FancF、FancG、FancI、FancJ、FancL、FancM、FancN、FancO、FancP、FancQ、FancR、FancS、FancT、FancU、FancV、FancW、γC、JAK3、IL7RA、RAG1、RAG2、DCLRE1C、PRKDC、LIG4、NHEJ1、CD3D、CD3E、CD3Z、CD3G、PTPRC、ZAP70、LCK、AK2、ADA、PNP、WHN、CHD7、ORAI1、STIM1、CORO1A、CIITA、RFXANK、RFX5、RFXAP、RMRP、DKC1、TERT、TINF2、DCLRE1B、SLC46A1、F8、F9、IDUA、イズロニダーゼ、IDS、GNS、HGSNAT、SGSH、NAGLU、GUSB、GALNS、GLB1、ARSB、HYAL1、101F6、123F2、53BP2、abl、ABLI、ADP、aFGF、APC、ApoAI、ApoAIV、ApoE、ATM、BAI−1、BDNF、Beta*(BLU)、bFGF、BLC1、BLC6、BRCA1、BRCA2、CBFA1、CBL、C−CAM、CFTR、CNTF、COX−1、CSFIR、CTS−1、シトシンデアミナーゼ、DBCCR−1、DCC、Dp、DPC−4、E1A、E2F、EBRB2、erb、ERBA、ERBB、ETS1、ETS2、ETV6、Fab、FCC、FGF、FGR、FHIT、fms、FOX、FUS 1、FUS1、FYN、G−CSF、GDAIF、Gene 21、Gene 26、GM−CSF、GMF、gsp、HCR、HIC−1、HRAS、hst、IGF、IL−1、IL−2、IL−3、IL−4、IL−5、IL−6、IL−7、IL−8、IL−9、IL−10、IL−11 IL−12、ING1、インターフェロンα、インターフェロンβ、インターフェロンγ、IRF−1、JUN、KRAS、LCK、LUCA−1、LUCA−2、LYN、MADH4、MADR2、MCC、mda7、MDM2、MEN−I、MEN−II、MLL、MMAC1、MYB、MYC、MYCL1、MYCN、neu、NF−1、NF−2、NGF、NOEY1、NOEY2、NRAS、NT3、NT5、OVCA1、p16、p21、p27、p53、p57、p73、p300、PGS、PIM1、PL6、PML、PTEN、raf、Rap1A、ras、Rb、RB1、RET、rks−3、ScFv、scFV ras、SEM A3、SRC、TAL1、TCL3、TFPI、トロンボスポンジン、チミジンキナーゼ、TNF、TP53、trk、T−VEC、VEGF、VHL、WT1、WT−1、YES、zac1、α2β1、αvβ3、αvβ5、αvβ63、BOB/GPR15、Bonzo/STRL−33/TYMSTR、CCR2、CCR3、CCR5、CCR8、CD4、CD46、CD55、CXCR4、アミノペプチダーゼ−N、HHV−7、ICAM、ICAM−1、PRR2/HveB、HveA、α−ジストログリカン、LDLR/α2MR/LRP、PVR、PRR1/HveC、ラミニン受容体、可溶性CD40、CTLA、Fas L、グロビンファミリー遺伝子、WAS、phox、ジスロトフィン、ピルビン酸キナーゼ、CLN3、ABCD1、アリールスルファターゼA、SFTPB、SFTPC、NLX2.1、ABCA3、GATA1、リボソームタンパク質遺伝子、TERT、TERC、DKC1、TINF2、CFTR、LRRK2、PARK2、PARK7、PINK1、SNCA、PSEN1、PSEN2、APP、SOD1、TDP43、FUS、ユビキリン2、C9ORF72及び/又はCD4、CD5、CD7、CD52、IL−1、IL−2、IL−6に対する抗体;TNF;P53、PTPN22、DRB1*1501/DQB1*0602若しくは自己反応性T細胞に特異的に存在するTCR;IL−4;IL−10;IL−12;IL−13;IL−1Ra、sIL−1RI、sIL−1RII;sTNFRI;及び/又はsTNFRIIを含む、請求項16に記載の方法。
- 硫酸プロタミン、幹細胞因子、トロンボポエチン、Flt−3リガンド及びN−アセチルシステインを補填した培地におけるCD34+細胞集団内のCD34+細胞を培養する工程をさらに含む、請求項9に記載の方法。
- 請求項9に記載の方法により形成されたCD34+細胞集団を含む、治療用細胞製剤。
- CD34+細胞集団内のCD34+細胞が遺伝子改変されている、請求項19に記載された治療用細胞製剤。
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US62/503,801 | 2017-05-09 | ||
PCT/US2018/029983 WO2018201065A1 (en) | 2017-04-27 | 2018-04-27 | Therapeutic formulations containing cd34+ stem cells derived from negative selection |
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EP (1) | EP3615044A4 (ja) |
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