JP2024534124A - 逆位末端反復配列によるクローズドエンドdnaの作製 - Google Patents

逆位末端反復配列によるクローズドエンドdnaの作製 Download PDF

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JP2024534124A
JP2024534124A JP2024512008A JP2024512008A JP2024534124A JP 2024534124 A JP2024534124 A JP 2024534124A JP 2024512008 A JP2024512008 A JP 2024512008A JP 2024512008 A JP2024512008 A JP 2024512008A JP 2024534124 A JP2024534124 A JP 2024534124A
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nucleic acid
acid molecule
itr
sequence
promoter
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JP2024534124A5 (enExample
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アジャイ・メゴディア
クリスティアン・ミュラー
トンヤオ・リウ
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バイオベラティブ セラピューティクス インコーポレイテッド
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
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    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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JP2024512008A 2021-08-23 2022-08-22 逆位末端反復配列によるクローズドエンドdnaの作製 Pending JP2024534124A (ja)

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US202163236215P 2021-08-23 2021-08-23
US63/236,215 2021-08-23
PCT/US2022/075280 WO2023028455A1 (en) 2021-08-23 2022-08-22 Closed-end dna production with inverted terminal repeat sequences

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JP2024534124A true JP2024534124A (ja) 2024-09-18
JP2024534124A5 JP2024534124A5 (enExample) 2025-08-29

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US (1) US20230091932A1 (enExample)
EP (1) EP4392445A1 (enExample)
JP (1) JP2024534124A (enExample)
KR (1) KR20240049825A (enExample)
CN (1) CN117881696A (enExample)
AR (1) AR126845A1 (enExample)
AU (1) AU2022334714A1 (enExample)
CA (1) CA3229345A1 (enExample)
CO (1) CO2024001242A2 (enExample)
IL (1) IL310963A (enExample)
MX (1) MX2024002192A (enExample)
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NZ762100A (en) 2017-08-09 2025-12-19 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof
EP3833766A1 (en) 2018-08-09 2021-06-16 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
CA3262218A1 (en) 2022-07-19 2024-01-25 Rampart Bioscience, Inc. CIRCULAR NON-IMMUNOGENEOUS NON-VIRAL DNA VECTORS
EP4692314A1 (en) * 2023-03-24 2026-02-11 Nanjing GenScript Biotech Co., Ltd. Host cell for improving stability of plasmid
WO2024249298A2 (en) * 2023-05-26 2024-12-05 Generation Bio Co. Modified closed-ended dna (cedna) vectors, compositions, and uses thereof

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4704362A (en) 1977-11-08 1987-11-03 Genentech, Inc. Recombinant cloning vehicle microbial polypeptide expression
EP0832981A1 (en) 1987-02-17 1998-04-01 Pharming B.V. DNA sequences to target proteins to the mammary gland for efficient secretion
US5633076A (en) 1989-12-01 1997-05-27 Pharming Bv Method of producing a transgenic bovine or transgenic bovine embryo
MX9204374A (es) 1991-07-25 1993-03-01 Idec Pharma Corp Anticuerpo recombinante y metodo para su produccion.
ATE236987T1 (de) 1992-11-13 2003-04-15 Idec Pharma Corp Konsensus-kozak-sequenzen zur säugetier- exprimierung
US5736137A (en) 1992-11-13 1998-04-07 Idec Pharmaceuticals Corporation Therapeutic application of chimeric and radiolabeled antibodies to human B lymphocyte restricted differentiation antigen for treatment of B cell lymphoma
US5827690A (en) 1993-12-20 1998-10-27 Genzyme Transgenics Corporatiion Transgenic production of antibodies in milk
GB9524973D0 (en) 1995-12-06 1996-02-07 Lynxvale Ltd Viral vectors
AU737155B2 (en) 1997-03-14 2001-08-09 Biogen Idec Inc. Method for integrating genes at specific sites in mammalian cells via homologous recombination and vectors for accomplishing the same
GB0911870D0 (en) * 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
WO2014168953A1 (en) * 2013-04-08 2014-10-16 University Of Iowa Research Foundation Chimeric adeno-associated virus/ bocavirus parvovirus vector
SMT202000531T1 (it) * 2014-03-09 2020-11-10 Univ Pennsylvania Composizioni utili in trattamento di deficit di ornitina transcarbamilasi(otc)
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
EP3687580A1 (en) * 2017-09-27 2020-08-05 Sigilon Therapeutics, Inc. Methods, compositions, and implantable elements comprising active cells
EP3736286A1 (en) * 2019-05-09 2020-11-11 Biotest AG Single chain factor viii molecule
JP2023535632A (ja) * 2020-07-27 2023-08-18 アンジャリウム バイオサイエンシズ エージー Dna分子の組成物、その作製方法、及びその使用方法

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MX2024002192A (es) 2024-03-13
US20230091932A1 (en) 2023-03-23
CN117881696A (zh) 2024-04-12
CA3229345A1 (en) 2023-03-02
IL310963A (en) 2024-04-01
AU2022334714A1 (en) 2024-04-04
CO2024001242A2 (es) 2024-04-29
AR126845A1 (es) 2023-11-22
EP4392445A1 (en) 2024-07-03
KR20240049825A (ko) 2024-04-17
WO2023028455A1 (en) 2023-03-02
TW202328174A (zh) 2023-07-16

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