SI2002003T1 - Genski vektor, ki vsebuje mi-RNA - Google Patents

Genski vektor, ki vsebuje mi-RNA

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Publication number
SI2002003T1
SI2002003T1 SI200632032A SI200632032A SI2002003T1 SI 2002003 T1 SI2002003 T1 SI 2002003T1 SI 200632032 A SI200632032 A SI 200632032A SI 200632032 A SI200632032 A SI 200632032A SI 2002003 T1 SI2002003 T1 SI 2002003T1
Authority
SI
Slovenia
Prior art keywords
rna
gene vector
gene
vector
Prior art date
Application number
SI200632032A
Other languages
English (en)
Inventor
Luigi Naldini
Brian David Brown
Original Assignee
Ospedale San Raffaele S.R.L.
Fondazione Telethon
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Ospedale San Raffaele S.R.L., Fondazione Telethon filed Critical Ospedale San Raffaele S.R.L.
Publication of SI2002003T1 publication Critical patent/SI2002003T1/sl

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    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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Families Citing this family (133)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2603881A1 (en) 2005-04-04 2006-10-12 The Board Of Regents Of The University Of Texas System Micro-rna's that regulate muscle cells
CA2609142C (en) * 2005-05-27 2016-02-09 Fondazione Centro San Raffaele Del Monte Tabor Therapeutic gene vectors comprising mirna target sequences
EP2377559A1 (en) * 2005-06-03 2011-10-19 Southern Adelaide Health Service - Flinders Medical Centre Targeting cells with altered microRNA expression
PL2056882T3 (pl) 2006-08-01 2013-03-29 Univ Texas Identyfikacja mikro-RNA, który aktywuje ekspresję łańcucha ciężkiego beta-miozyny
WO2008071959A1 (en) * 2006-12-12 2008-06-19 Oxford Biomedica (Uk) Limited Lentiviral vectors comprising micrornas
EP2125032A4 (en) * 2007-02-20 2011-02-23 Mayo Foundation TREATMENT OF CANCER USING VIRAL NUCLEIC ACID
AU2008283794B2 (en) 2007-07-31 2014-03-20 Board Of Regents, The University Of Texas System Micro-RNAs that control myosin expression and myofiber identity
US8440636B2 (en) 2007-07-31 2013-05-14 The Board Of Regents, The University Of Texas System Micro-RNA family that modulates fibrosis and uses thereof
KR20100099158A (ko) 2007-11-09 2010-09-10 보드 오브 리전츠 더 유니버시티 오브 텍사스 시스템 심근세포 생존과 심장 회복을 제어하는 MIR-15 집단의 마이크로RNAs
WO2009117418A2 (en) 2008-03-17 2009-09-24 The Board Of Regents Of The University Of Texas System Identification of micro-rnas involved in neuromuscular synapse maintenance and regeneration
US20110098346A1 (en) * 2008-05-19 2011-04-28 Agency For Science, Technology And Research Nucleic acid molecule and method of targeting gene expression to gliomas
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US20100183558A1 (en) 2008-10-17 2010-07-22 Zhennan Lai Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules
WO2010055413A1 (en) 2008-11-12 2010-05-20 Fondazione Centro San Raffaele Del Monte Tabor Gene vector for inducing transgene-specific immune tolerance
CN101787373B (zh) * 2009-01-23 2013-06-19 中国人民解放军第二军医大学东方肝胆外科医院 一种携带外源基因在包装细胞中高效生产的重组病毒载体、其构建方法及其用途
AU2010210605B2 (en) 2009-02-04 2015-08-13 Board Of Regents, The University Of Texas System Dual targeting of miR-208 and miR-499 in the treatment of cardiac disorders
EP2907879A3 (en) 2009-04-29 2015-10-14 Academisch Medisch Centrum bij de Universiteit van Amsterdam Means and methods for counteracting, preventing and/or determining heart failure, or a risk of heart failure
SG175839A1 (en) 2009-04-30 2011-12-29 San Raffaele Centro Fond Gene vector
US8728458B2 (en) 2009-04-30 2014-05-20 The Regents Of The University Of California Combination anti-HIV vectors, targeting vectors, and methods of use
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
CA2769670C (en) * 2009-07-31 2018-10-02 Ethris Gmbh Rna with a combination of unmodified and modified nucleotides for protein expression
DE102009050308A1 (de) 2009-10-22 2011-05-05 Ludwig-Maximilians-Universität München RNA mit einer Kombination aus unmodifizierten und modifizierten Nucleotiden zur Proteinexpression
WO2011020014A1 (en) 2009-08-14 2011-02-17 Regeneron Pharmaceuticals, Inc. Promoter-regulated differentiation-dependent self-deleting cassette
US9593347B2 (en) * 2010-04-16 2017-03-14 University of Pittsburgh—of the Commonwealth System of Higher Education Identification of mutations in herpes simplex virus envelope glycoproteins that enable or enhance vector retargeting to novel non-HSV receptors
CA2833912C (en) 2010-04-23 2021-09-21 University Of Massachusetts Aav-based treatment of cholesterol-related disorders
JP5963743B2 (ja) 2010-04-23 2016-08-03 ユニバーシティ オブ マサチューセッツ Cnsターゲティングaavベクターおよびその使用方法
CA3050894C (en) 2010-04-23 2022-10-18 University Of Massachusetts Multicistronic expression constructs
US9790491B2 (en) * 2010-07-12 2017-10-17 National University Corporation Tottori University Method for producing novel hiPSC by means of miRNA introduction
US9309534B2 (en) 2010-07-12 2016-04-12 Universidad Autonoma De Barcelona Gene therapy composition for use in diabetes treatment
WO2012065027A2 (en) * 2010-11-11 2012-05-18 University Of Miami Compositions, kits and methods for treatment of cardiovascular, immunological, and inflammatory diseases
EP2652151A2 (en) 2010-12-15 2013-10-23 Miragen Therapeutics Microrna inhibitors comprising locked nucleotides
WO2012143401A1 (en) 2011-04-18 2012-10-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Tissue-specific regulation of transgene expression
EP2699680B1 (en) 2011-04-21 2017-12-27 University of Massachusetts Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
CA2844012C (en) 2011-08-04 2021-03-16 Yeda Research And Development Co. Ltd. Micro-rnas and compositions comprising same for the treatment and diagnosis of serotonin-, adrenalin-, noradrenalin-, glutamate-, and corticotropin-releasing hormone- associated medical conditions
PL3572517T3 (pl) 2011-08-05 2021-10-04 Regeneron Pharmaceuticals, Inc. Myszy z humanizowanym uniwersalnym łańcuchem lekkim
WO2013043442A1 (en) * 2011-09-23 2013-03-28 Dharmacon, Inc. The introduction of modular vector elements during production of a lentivirus
AU2013201303C1 (en) 2011-10-06 2016-06-23 MiRagen Therapeutics, Inc. Control of whole body energy homeostasis by microRNA regulation
EP2584040A1 (en) 2011-10-17 2013-04-24 Pharmahungary 2000 Kft. Compounds for treatment of ischemic injury
US9878056B2 (en) 2012-04-02 2018-01-30 Modernatx, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
US20140010861A1 (en) 2012-04-02 2014-01-09 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
US10501513B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides for the production of oncology-related proteins and peptides
WO2013152230A1 (en) 2012-04-04 2013-10-10 The Trustees Of Columbia University In The City Of New York Smooth muscle specific inhibition for anti-restenotic therapy
US9163235B2 (en) 2012-06-21 2015-10-20 MiRagen Therapeutics, Inc. Inhibitors of the miR-15 family of micro-RNAs
CN103505743A (zh) * 2012-06-21 2014-01-15 北京命码生科科技有限公司 含功能性microRNA/siRNA的细胞微粒子及其应用
US9388408B2 (en) 2012-06-21 2016-07-12 MiRagen Therapeutics, Inc. Oligonucleotide-based inhibitors comprising locked nucleic acid motif
LT2922554T (lt) 2012-11-26 2022-06-27 Modernatx, Inc. Terminaliai modifikuota rnr
AU2013374345A1 (en) * 2013-01-17 2015-08-06 Moderna Therapeutics, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
SI3889173T1 (sl) 2013-02-15 2023-11-30 Bioverativ Therapeutics Inc. Optimiran gen dejavnika VIII
DK3184641T3 (da) 2013-10-28 2020-08-03 Univ Of Pittsburgh - Of The Commonwealth System Of Higher Education Onkolytisk hsv-vektor
US20150147774A1 (en) * 2013-11-22 2015-05-28 National University Of Singapore Expression construct for yeast and a method of using the construct
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
EP3750907A3 (en) 2014-03-18 2021-04-28 University of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
WO2015164786A1 (en) * 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
EP3160478A4 (en) 2014-06-30 2018-05-16 Bioverativ Therapeutics Inc. Optimized factor ix gene
EP3194430A1 (en) 2014-09-16 2017-07-26 Universitat Autònoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
JP7023108B2 (ja) 2014-10-21 2022-02-21 ユニバーシティ オブ マサチューセッツ 組み換えaavバリアントおよびその使用
CA2969599A1 (en) 2014-12-05 2016-06-09 Universitat Autonoma De Barcelona Viral vectors for the treatment of diabetes
JP2018503646A (ja) 2015-01-20 2018-02-08 ミラゲン セラピューティクス, インコーポレイテッド miR−92阻害剤およびその使用
US10584321B2 (en) 2015-02-13 2020-03-10 University Of Massachusetts Compositions and methods for transient delivery of nucleases
EP3285780A4 (en) 2015-04-24 2018-12-19 University of Massachusetts Modified aav constructions and uses thereof
EP3319631A4 (en) 2015-07-08 2019-01-09 American Gene Technologies International Inc. HIV PREMUNICATION AND IMMUNOTHERAPY
JP6990176B2 (ja) 2015-10-05 2022-02-03 モデルナティエックス インコーポレイテッド メッセンジャーリボ核酸薬物の治療投与のための方法
US10188750B1 (en) 2015-10-23 2019-01-29 University Of South Florida Self-replicating cell selective gene delivery compositions, methods, and uses thereof
US10137144B2 (en) 2016-01-15 2018-11-27 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta T-cells
IL310925A (en) 2016-01-15 2024-04-01 American Gene Tech Int Inc Methods and preparations for activating GAMMA-DELTA T cells
SG10202107138TA (en) 2016-01-27 2021-08-30 Oncorus Inc Oncolytic viral vectors and uses thereof
PL3411478T3 (pl) 2016-02-01 2022-10-03 Bioverativ Therapeutics Inc. Geny zoptymalizowanego czynnika VIII
JP7153332B2 (ja) 2016-02-08 2022-10-14 アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド Hivワクチン接種および免疫療法
WO2017139643A1 (en) 2016-02-12 2017-08-17 University Of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
EP4036231A1 (en) 2016-03-09 2022-08-03 American Gene Technologies International Inc. Combination vectors and methods for treating cancer
US11976292B2 (en) 2016-06-08 2024-05-07 American Gene Technologies International Inc. Non-integrating viral delivery system and methods related thereto
EP3478321A4 (en) 2016-06-30 2020-04-22 Oncorus, Inc. PSEUDOTYPIZED ONCOLYTIC VIRAL ADMINISTRATION OF THERAPEUTIC POLYPEPTIDES
CA3028982A1 (en) 2016-07-08 2018-01-11 American Gene Technologies International Inc. Hiv pre-immunization and immunotherapy
GB201612214D0 (en) 2016-07-14 2016-08-31 Univ Oxford Innovation Ltd Method
JP7176756B2 (ja) 2016-07-21 2022-11-22 アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド パーキンソン病を処置するためのウイルスベクター
CN110088285A (zh) * 2016-07-26 2019-08-02 森迪生物科学公司 时空调节子
JP2018029521A (ja) * 2016-08-24 2018-03-01 国立研究開発法人農業・食品産業技術総合研究機構 miR398標的核酸配列を含む、遺伝子導入のためのウイルスベクター
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
CA3040483A1 (en) 2016-10-13 2018-04-19 University Of Massachusetts Aav capsid designs
CA3048313A1 (en) 2017-01-06 2018-07-12 Stabilitech Biopharma Ltd Virus
CN107384868A (zh) * 2017-02-17 2017-11-24 中国农业科学院上海兽医研究所 永生化原代绵羊肺细胞系的构建及其应用
CA3057142A1 (en) 2017-04-03 2018-10-11 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
KR20200034668A (ko) 2017-05-08 2020-03-31 플래그쉽 파이어니어링 이노베이션스 브이, 인크. 막 융합을 촉진시키기 위한 조성물 및 그의 용도
TW202333779A (zh) * 2017-05-08 2023-09-01 美商磨石生物公司 阿爾法病毒新抗原載體
US11859179B2 (en) 2017-05-09 2024-01-02 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (ALS)
JP2020519294A (ja) 2017-05-12 2020-07-02 ユニバーシティ オブ マサチューセッツ ウイルスベクター産生
DK3428194T3 (da) 2017-07-14 2021-11-15 Immatics Biotechnologies Gmbh Forbedret polypeptidmolekyle med dobbelt specificitet
JP7249323B2 (ja) 2017-07-26 2023-03-30 オンコラス, インコーポレイテッド 腫瘍溶解性ウイルスベクター及びその使用
IT201700086403A1 (it) * 2017-07-27 2019-01-27 Univ Del Piemonte Orientale Endothelial-specific promoter sequences and uses thereof
KR20200035130A (ko) 2017-08-09 2020-04-01 바이오버라티브 테라퓨틱스 인크. 핵산 분자 및 이의 용도
EP3444347A1 (en) 2017-08-18 2019-02-20 Universität Heidelberg Use of anti-crispr polypeptides for specific activation of cas nucleases
CN118006607A (zh) 2017-09-22 2024-05-10 马萨诸塞大学 Sod1双表达载体及其用途
KR20230110812A (ko) * 2017-10-10 2023-07-25 난트바이오 인코포레이티드 바이러스 생성 페이로드에 대해 독성이 낮은 변형된ec7 세포
IL275273B1 (en) * 2017-12-15 2024-05-01 Medizinische Hochschule Hannover An improved compound for the treatment of heart failure
EP3731850A4 (en) 2017-12-29 2021-12-01 Oncorus, Inc. ONCOLYTIC VIRUS DELIVERY OF THERAPEUTIC POLYPEPTIDES
SG11202006400UA (en) 2018-01-04 2020-08-28 Iconic Therapeutics Inc Anti-tissue factor antibodies, antibody-drug conjugates, and related methods
SG11202007114VA (en) 2018-02-01 2020-08-28 Bioverativ Therapeutics Inc Use of lentiviral vectors expressing factor viii
US20210228627A1 (en) 2018-05-15 2021-07-29 Flagship Pioneering Innovations V, Inc. Fusosome compositions and uses thereof
EP3820509A1 (en) 2018-07-09 2021-05-19 Flagship Pioneering Innovations V, Inc. Fusosome compositions and uses thereof
AU2019319984A1 (en) 2018-08-09 2021-03-04 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
SG11202102058UA (en) 2018-08-30 2021-03-30 Tenaya Therapeutics Inc Cardiac cell reprogramming with myocardin and ascl1
JP2022513347A (ja) 2018-10-11 2022-02-07 アイドゲノーシッシェ テヒニッシェ ホッホシューレ チューリッヒ 高度にコンパクトな多入力論理ゲートをコードする核酸ベクターを使用して疾患を処置する方法
EP3876952A4 (en) 2018-11-05 2022-08-24 American Gene Technologies International Inc. VECTOR SYSTEM FOR EXPRESSING REGULATORY RNA
US20230043255A1 (en) 2018-11-14 2023-02-09 Flagship Pioneering Innovations V, Inc. Fusosome compositions for t cell delivery
EP3880832A1 (en) 2018-11-14 2021-09-22 Flagship Pioneering Innovations V, Inc. Fusosome compositions for hematopoietic stem cell delivery
US20220008557A1 (en) 2018-11-14 2022-01-13 Flagship Pioneering Innovations V, Inc. Fusosome compositions for cns delivery
EP3891297A4 (en) * 2018-12-03 2022-09-07 California Institute of Technology IN SITU READING OF DNA BARCODES
TW202039855A (zh) 2018-12-06 2020-11-01 美商百歐維拉提夫治療公司 表現因子ix之慢病毒載體的用途
EP3908326A4 (en) * 2018-12-21 2022-10-26 The Trustees of The University of Pennsylvania COMPOSITIONS FOR DRG-SPECIFIC REDUCTION OF TRANSGENE EXPRESSION
EP3906039A4 (en) * 2019-01-04 2023-01-18 Oncorus, Inc. ENCAPSULATED RNA POLYNUCLEOTIDES AND METHODS OF USE
KR102248420B1 (ko) * 2019-03-15 2021-05-06 주식회사 제너로스 miR-142-3p의 표적 서열을 포함하는 재조합 벡터
CN114026236A (zh) * 2019-04-24 2022-02-08 宾夕法尼亚州大学信托人 可用于治疗雷特综合征的组合物
BR122024002387A2 (pt) 2019-05-30 2024-03-12 Gritstone Bio, Inc. Vetores de adenovírus, composição farmacêutica, sequência de nucleotídeo isolada, célula isolada, vetor, kit, usos de um vetor, método para fabricar o vetor, métodos para produzir um vírus e vetor viral
CN114521143A (zh) 2019-09-19 2022-05-20 吉尼松公司 减轻fkrp心脏毒性的基因治疗表达***
KR20220097891A (ko) 2019-09-30 2022-07-08 바이오버라티브 테라퓨틱스 인크. 렌티바이러스 벡터 제형
KR102461417B1 (ko) * 2019-11-11 2022-11-01 주식회사 온코크로스 아데노바이러스 벡터를 포함하는 전립선암 진단용 조성물
IL298138A (en) * 2020-05-12 2023-01-01 Univ Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2021255245A2 (en) 2020-06-19 2021-12-23 Genethon Gene therapy expression system allowing an adequate expression in the muscles and in the heart of sgcg
AU2021297965A1 (en) 2020-06-24 2023-02-23 Bioverativ Therapeutics Inc. Methods for the removal of free factor VIII from preparations of lentiviral vectors modified to express said protein
WO2022008711A1 (en) 2020-07-10 2022-01-13 Genethon A novel muscle-specific promoter
WO2022032196A2 (en) 2020-08-06 2022-02-10 Gritstone Bio, Inc. Multiepitope vaccine cassettes
BR112023002395A2 (pt) 2020-08-23 2023-03-21 Bioverativ Therapeutics Inc Sistema de baculovírus modificado para produção aprimorada de dna com extremidades fechadas (cedna)
KR20220035693A (ko) * 2020-09-14 2022-03-22 주식회사 제너로스 표적화된 유전자 전달을 위한 아데노-부속 바이러스 벡터
KR102626543B1 (ko) * 2020-09-14 2024-01-23 주식회사 제너로스 AIMP2-DX2 및 miR-142의 표적 핵산을 포함하는 재조합 벡터
JP7121086B2 (ja) * 2020-09-25 2022-08-17 ウニベルシダッド アウトノマ デ バルセロナ 脂肪組織に形質導入するのに有用なアデノ随伴ウイルスベクター
JP2023544142A (ja) * 2020-09-30 2023-10-20 ジェネロス カンパニー, リミテッド AIMP2-DX2および必要に応じてmiR-142の標的配列ならびにその組成物を使用した加齢黄斑疾患を処置する方法
EP4334361A1 (en) 2021-05-05 2024-03-13 Immatics Biotechnologies GmbH Antigen binding proteins specifically binding prame
KR20240049821A (ko) 2021-08-23 2024-04-17 바이오버라티브 테라퓨틱스 인크. 바큘로바이러스 발현 시스템
AR126846A1 (es) 2021-08-23 2023-11-22 Bioverativ Therapeutics Inc Genes del factor viii optimizados
KR20240067122A (ko) 2021-09-30 2024-05-16 바이오버라티브 테라퓨틱스 인크. 감소된 면역원성을 갖는 인자 viii 폴리펩타이드를 암호화하는 핵산
WO2023142040A1 (en) * 2022-01-29 2023-08-03 Virogin Biotech Canada Ltd. Transcriptional and translational dual regulated oncolytic herpes simplex virus vectors
WO2023218430A1 (en) * 2022-05-13 2023-11-16 Generoath Co., Ltd. Methods of treating retinal degenerative diseases using aimp2-dx2 and optionally a target sequence for mir‑142 and compositions thereof

Family Cites Families (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS56169586A (en) 1981-05-02 1981-12-26 Tokyo Tanabe Co Ltd Novel beta-galactosidase
FR2705361B1 (fr) 1993-05-18 1995-08-04 Centre Nat Rech Scient Vecteurs viraux et utilisation en thérapie génique.
FR2705686B1 (fr) 1993-05-28 1995-08-18 Transgene Sa Nouveaux adénovirus défectifs et lignées de complémentation correspondantes.
ES2310924T3 (es) 1993-07-13 2009-01-16 Centelion Vectores adenovirales defectivos y utilizacion en terapia genica.
WO1995003400A1 (en) 1993-07-23 1995-02-02 Johns Hopkins University School Of Medicine Recombinationally targeted cloning in yeast artificial chromosomes
US7252989B1 (en) 1994-04-04 2007-08-07 Board Of Regents, The University Of Texas System Adenovirus supervector system
FR2724945B1 (fr) 1994-09-27 1996-12-27 Centre Nat Rech Scient Vecteurs viraux et utilisation en therapie genique
IL116816A (en) 1995-01-20 2003-05-29 Rhone Poulenc Rorer Sa Cell for the production of a defective recombinant adenovirus or an adeno-associated virus and the various uses thereof
FR2730504B1 (fr) 1995-02-13 1997-03-28 Rhone Poulenc Rorer Sa Procede de preparation de genomes d'adenovirus recombinants
US5723315A (en) 1996-08-23 1998-03-03 Genetics Institute, Inc. Secreted proteins and polynucleotides encoding them
ES2217425T3 (es) 1996-08-07 2004-11-01 Darwin Discovery Limited Derivados del acido hidroxamico y del acido carboxilico con actividad inhibitoria de la mmp y del tnf.
US6126939A (en) 1996-09-03 2000-10-03 Yeda Research And Development Co. Ltd. Anti-inflammatory dipeptide and pharmaceutical composition thereof
JP2002504372A (ja) * 1998-02-25 2002-02-12 ザ ダウ ケミカル カンパニー 高親和性ヒト型化抗−ceaモノクロ−ナル抗体
IL138021A0 (en) 1998-02-25 2001-10-31 Dow Chemical Co High affinity humanized anti-tag-72 monoclonal antibodies
GB2375113B (en) * 2000-01-21 2004-10-20 Biovex Ltd A modified, oncolytic, non-laboratory HSV strain and its use in treating cancer
WO2003006465A1 (en) 2001-07-13 2003-01-23 Cv Therapeutics, Inc. Partial and full agonist of a adenosine receptors
US20030198627A1 (en) 2001-09-01 2003-10-23 Gert-Jan Arts siRNA knockout assay method and constructs
EP2385122B1 (en) * 2001-09-28 2018-04-25 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. MicroRNA molecules
EP1504108B1 (en) 2002-02-01 2013-04-17 Oxford Biomedica (UK) Limited Lentiviral vector
US6998391B2 (en) 2002-02-07 2006-02-14 Supergen.Inc. Method for treating diseases associated with abnormal kinase activity
ATE425160T1 (de) 2002-12-18 2009-03-15 Vertex Pharma Benzisoxazolderivate, die sich als inhibitoren von proteinkinasen eigen
CN1738648A (zh) 2003-01-17 2006-02-22 佛罗里达大学研究基金会有限公司 小干扰rna基因治疗
WO2004066183A2 (en) 2003-01-22 2004-08-05 European Molecular Biology Laboratory Microrna
WO2004094642A2 (en) * 2003-04-24 2004-11-04 Fondazione Centro San Raffaele Del Monte Tabor Lentiviral vectors carrying synthetic bi-directional promoters and uses thereof
EP2530157B1 (en) 2003-07-31 2016-09-28 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of miRNAs
WO2005047505A2 (en) 2003-08-07 2005-05-26 Whitehead Institute For Biomedical Research Methods and products for expression of micro rnas
US20050142581A1 (en) 2003-09-04 2005-06-30 Griffey Richard H. Microrna as ligands and target molecules
US20050266552A1 (en) * 2003-12-05 2005-12-01 Doench John G Reagents and methods for identification of RNAi pathway genes and chemical modulators of RNAi
US7582744B2 (en) * 2004-08-10 2009-09-01 Alnylam Pharmaceuticals, Inc. Chemically modified oligonucleotides
US20060056344A1 (en) * 2004-09-10 2006-03-16 Interdigital Technology Corporation Seamless channel change in a wireless local area network
US20060265771A1 (en) * 2005-05-17 2006-11-23 Lewis David L Monitoring microrna expression and function
CA2609142C (en) * 2005-05-27 2016-02-09 Fondazione Centro San Raffaele Del Monte Tabor Therapeutic gene vectors comprising mirna target sequences
US20070054872A1 (en) * 2005-08-24 2007-03-08 Mirus Bio Corporation Regulatable or conditional expression systems
US20090004668A1 (en) * 2007-06-22 2009-01-01 The Board Of Trustees Of The Leland Stanford Junior University Pre-miRNA loop-modulated target regulation

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EP2002003A2 (en) 2008-12-17
US10000757B2 (en) 2018-06-19
KR20080041601A (ko) 2008-05-13
CN101287834B (zh) 2016-11-09
JP2013226139A (ja) 2013-11-07
WO2007000668A2 (en) 2007-01-04
US11753643B2 (en) 2023-09-12
US9556438B2 (en) 2017-01-31
SG162726A1 (en) 2010-07-29
DK2002003T3 (en) 2016-03-21
ES2564823T3 (es) 2016-03-29
HUE027013T2 (en) 2016-10-28
CN101287834A (zh) 2008-10-15
US20100041737A1 (en) 2010-02-18
WO2007000668A3 (en) 2007-12-21
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JP2008545406A (ja) 2008-12-18
CA2609142C (en) 2016-02-09
KR101373548B1 (ko) 2014-03-25
CA2609142A1 (en) 2007-01-04
KR20130114758A (ko) 2013-10-17
EP2002003B1 (en) 2015-12-30
PL2002003T3 (pl) 2016-06-30
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US20150167003A1 (en) 2015-06-18

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