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PL2002003T3 - Wektor genetyczny zawierający mi-RNA - Google Patents

Wektor genetyczny zawierający mi-RNA

Info

Publication number
PL2002003T3
PL2002003T3 PL06795291T PL06795291T PL2002003T3 PL 2002003 T3 PL2002003 T3 PL 2002003T3 PL 06795291 T PL06795291 T PL 06795291T PL 06795291 T PL06795291 T PL 06795291T PL 2002003 T3 PL2002003 T3 PL 2002003T3
Authority
PL
Poland
Prior art keywords
rna
gene vector
gene
vector
Prior art date
Application number
PL06795291T
Other languages
English (en)
Inventor
Luigi Naldini
Brian David Brown
Original Assignee
Ospedale San Raffaele Srl
Fond 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 Srl, Fond Telethon filed Critical Ospedale San Raffaele Srl
Publication of PL2002003T3 publication Critical patent/PL2002003T3/pl

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    • C12N15/1137—Non-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 (143)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1877557A2 (en) 2005-04-04 2008-01-16 The Board of Regents of The University of Texas System Micro-rna's that regulate muscle cells
CN101287834B (zh) * 2005-05-27 2016-11-09 圣拉法埃莱医院有限公司 基因载体
WO2006128245A1 (en) * 2005-06-03 2006-12-07 Southern Adelaide Health Service-Flinders Medical Centre Targeting cells with altered microrna expression
CA2659364C (en) 2006-08-01 2017-08-22 Board Of Regents Of The University Of Texas System Identification of a micro-rna that activates expression of .beta.-myosin heavy chain
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 CANCER TREATMENT WITH VIRAL NUCLEIC ACID
CN101808649B (zh) 2007-07-31 2014-05-21 得克萨斯系统大学董事会 控制肌球蛋白表达和肌纤维身份的微小rna
BRPI0815383A2 (pt) 2007-07-31 2015-09-08 Univ Texas família de micro-rna que modula fibrose e usos da mesma
CN101951924B (zh) * 2007-11-09 2015-06-24 得克萨斯系统大学董事会 Mir-15家族的微小rna调控心肌细胞存活和心脏修复
JP5653899B2 (ja) 2008-03-17 2015-01-14 ボード・オブ・リージエンツ，ザ・ユニバーシテイ・オブ・テキサス・システム 神経筋シナプスの維持および再生に関与するマイクロｒｎａの同定
CN102099472A (zh) * 2008-05-19 2011-06-15 新加坡科技研究局 使基因表达靶向于神经胶质瘤的核酸分子和方法
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
DK2364362T3 (en) 2008-11-12 2016-01-25 Ospedale San Raffaele Srl Gene vector FOR INDUCTION OF IMMUNE TOLERANCE transgene
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
KR20110128838A (ko) 2009-02-04 2011-11-30 보드 오브 리전츠 더 유니버시티 오브 텍사스 시스템 심장 질환 치료에서 ｍｉｒ－208 및 ｍｉｒ－499의 이중 표적화
DK2425016T3 (en) 2009-04-29 2015-06-29 Amc Amsterdam MEANS AND METHODS TO REMEDY THE, PREVENT AND / OR diagnose heart failure or EN risk of cardiac failure
CN102596255B (zh) * 2009-04-30 2017-10-13 圣拉法埃莱医院有限公司 基因载体
WO2010127166A2 (en) 2009-04-30 2010-11-04 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
DE102009050308A1 (de) 2009-10-22 2011-05-05 Ludwig-Maximilians-Universität München RNA mit einer Kombination aus unmodifizierten und modifizierten Nucleotiden zur Proteinexpression
KR20120097484A (ko) 2009-07-31 2012-09-04 에트리스 게엠베하 단백질 발현을 위한 비변형된 뉴클레오티드 및 변형된 뉴클레오티드의 조합을 가진 ｒｎａ
WO2011020005A1 (en) * 2009-08-14 2011-02-17 Regeneron Pharmaceuticals, Inc. miRNA-REGULATED DIFFERENTIATION-DEPENDENT SELF-DELETING CASSETTE
WO2011130749A2 (en) 2010-04-16 2011-10-20 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
WO2011133901A2 (en) 2010-04-23 2011-10-27 University Of Massachusetts Aav-based treatment of cholesterol-related disorders
EP3536781A1 (en) 2010-04-23 2019-09-11 University of Massachusetts Cns targeting aav vectors and methods of use thereof
US9546369B2 (en) 2010-04-23 2017-01-17 University Of Massachusetts Multicistronic expression constructs
WO2012007458A1 (en) 2010-07-12 2012-01-19 Universidad Autónoma De Barcelona Gene therapy composition for use in diabetes treatment
US9476041B2 (en) * 2010-07-12 2016-10-25 National University Corporation Tottori University Method for producing novel hipsc by means of siRNA introduction
EP2638159B1 (en) * 2010-11-11 2019-04-24 University of Miami Compositions, kits and methods for treatment of cardiovascular, immunological, and inflammatory diseases
BR112013012319A2 (pt) 2010-12-15 2019-09-24 Miragen Therapeutics inibidores de micro rna compreendendo nucleotídeos bloqueados
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
EP2739736B1 (en) 2011-08-04 2017-04-19 Yeda Research and Development Co. Ltd. Mir-135 and compositions comprising same for the treatment of serotonin associated medical conditions
FI3865581T3 (fi) 2011-08-05 2024-11-02 Regeneron Pharma Humanisoituja universaaleja kevytketjun hiiriä
EP2758531B1 (en) * 2011-09-23 2017-04-12 GE Healthcare Dharmacon, Inc. The introduction of modular vector elements during production of a lentivirus
EP2763703B1 (en) 2011-10-06 2018-02-14 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
US10501513B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides for the production of oncology-related proteins and peptides
HK1206636A1 (zh) 2012-04-02 2016-01-15 Modernatx, Inc. 用於制备肿瘤相关蛋白和多肽的修饰的多核苷酸
US9303079B2 (en) 2012-04-02 2016-04-05 Moderna Therapeutics, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
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
SMT202200337T1 (it) 2012-11-26 2022-09-14 Modernatx Inc Rna modificato al livello del terminale
CA2897941A1 (en) * 2013-01-17 2014-07-24 Moderna Therapeutics, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
FI2956477T4 (fi) 2013-02-15 2024-04-24 Bioverativ Therapeutics Inc Optimoitu tekijä viii:n geeni
EP3778906B1 (en) 2013-10-28 2025-07-02 University of Pittsburgh- Of the Commonwealth System of Higher Education Oncolytic hsv vector and use thereof in
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
JP6756700B2 (ja) 2014-03-18 2020-09-16 ユニバーシティ オブ マサチューセッツ 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法
EP3134522B1 (en) 2014-04-25 2021-10-06 University of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
WO2016004113A1 (en) 2014-06-30 2016-01-07 Biogen Ma 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
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
RU2020140209A (ru) 2014-10-21 2021-01-25 Юниверсити Оф Массачусетс Варианты рекомбинантных aav и их применения
ES2824457T3 (es) 2014-12-05 2021-05-12 UNIV AUTòNOMA DE BARCELONA Vectores virales para el tratamiento de la diabetes
SG10201906716QA (en) 2015-01-20 2019-08-27 Miragen Therapeutics Inc Mir-92 inhibitors and uses thereof
US10584321B2 (en) 2015-02-13 2020-03-10 University Of Massachusetts Compositions and methods for transient delivery of nucleases
CA3021949C (en) 2015-04-24 2023-10-17 University Of Massachusetts Modified aav constructs and uses thereof
EP3319631A4 (en) 2015-07-08 2019-01-09 American Gene Technologies International Inc. HIV PREMUNICATION AND IMMUNOTHERAPY
EP3359670B2 (en) 2015-10-05 2024-02-14 ModernaTX, Inc. Methods for therapeutic administration of messenger ribonucleic acid drugs
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
CN108883100B (zh) 2016-01-15 2022-11-25 美国基因技术国际有限公司 用于活化γ-δT细胞的方法和组合物
EP4089166A1 (en) 2016-01-27 2022-11-16 Oncorus, Inc. Oncolytic viral vectors and uses thereof
SI3411478T1 (sl) 2016-02-01 2022-10-28 Bioverativ Therapeutics Inc. Optimirani geni dejavnika VIII
WO2017139065A1 (en) 2016-02-08 2017-08-17 American Gene Technologies International Inc. Hiv vaccination and immunotherapy
US11060088B2 (en) 2016-02-12 2021-07-13 University Of Massachusetts Anti-angiogenic miRNA therapeutics for inhibiting corneal neovascularization
WO2017156311A2 (en) 2016-03-09 2017-09-14 American Gene Technologies International Inc. Combination vectors and methods for treating cancer
KR20250133993A (ko) * 2016-04-08 2025-09-09 크리스탈 바이오테크, 인크. 피부의 상처, 장애 및 질환의 치료를 위한 조성물 및 방법
EP3458107B1 (en) * 2016-05-18 2024-03-13 ModernaTX, Inc. Polynucleotides encoding jagged1 for the treatment of alagille syndrome
WO2017213697A1 (en) 2016-06-08 2017-12-14 American Gene Technologies International Inc. Non-integrating viral delivery system and methods related thereto
SG11201811600PA (en) 2016-06-30 2019-01-30 Oncorus Inc Pseudotyped oncolytic viral delivery of therapeutic polypeptides
JP6971492B2 (ja) 2016-07-08 2021-11-24 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予備免疫化および免疫療法
GB201612214D0 (en) 2016-07-14 2016-08-31 Univ Oxford Innovation Ltd Method
US11583562B2 (en) 2016-07-21 2023-02-21 American Gene Technologies International Inc. Viral vectors for treating Parkinson's disease
CN110073000A (zh) * 2016-07-26 2019-07-30 森迪生物科学公司 遗传擦除子
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
KR20240173209A (ko) 2016-10-13 2024-12-10 유니버시티 오브 매사추세츠 Aav 캡시드 설계
US12016949B2 (en) 2017-01-06 2024-06-25 Iosbio 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
CN110869507A (zh) 2017-05-08 2020-03-06 旗舰先锋创新V股份有限公司 促进膜融合的组合物和其用途
CA3062591A1 (en) * 2017-05-08 2018-11-15 Gritstone Oncology, Inc. Alphavirus neoantigen vectors
EP3622073A4 (en) 2017-05-09 2021-01-06 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
HRP20211744T1 (hr) 2017-07-14 2022-02-04 Immatics Biotechnologies Gmbh Poboljšana molekula polipeptida dvostruke specifičnosti
SG11202000691XA (en) 2017-07-26 2020-02-27 Oncorus Inc Oncolytic viral vectors and uses thereof
IT201700086403A1 (it) * 2017-07-27 2019-01-27 Univ Del Piemonte Orientale Endothelial-specific promoter sequences and uses thereof
MX2020001593A (es) 2017-08-09 2020-07-13 Bioverativ Therapeutics Inc Moleculas de acido nucleico y usos de las mismas.
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
JP2021509253A (ja) * 2017-10-10 2021-03-25 ナントバイオ，インコーポレイテッド ウイルス生成ペイロードに対して低い毒性を有する改変ｅｃ７細胞
JP7401432B2 (ja) 2017-12-01 2023-12-19 エンコーデッド セラピューティクス， インコーポレイテッド 操作されたｄｎａ結合タンパク質
IL275273B2 (en) * 2017-12-15 2024-09-01 Medizinische Hochschule Hannover Improved compound for treatment of heart failure
EP3731850A4 (en) 2017-12-29 2021-12-01 Oncorus, Inc. ONCOLYTIC VIRUS DELIVERY OF THERAPEUTIC POLYPEPTIDES
IL315325B1 (en) 2018-01-04 2025-07-01 Iconic Therapeutics Inc Anti-tissue-mediated antibodies, antibody-drug conjugates, and related methods
CN111918674A (zh) 2018-02-01 2020-11-10 比奥维拉迪维治疗股份有限公司 表达因子viii的慢病毒载体的用途
US10716866B2 (en) 2018-03-29 2020-07-21 Generoath Co., Ltd Pharmaceutical composition comprising AIMP2-DX2 for preventing or treating neuronal diseases and use thereof
KR20210021473A (ko) 2018-05-15 2021-02-26 플래그쉽 파이어니어링 이노베이션스 브이, 인크. 푸소솜 조성물 및 그의 용도
AU2019301053A1 (en) 2018-07-09 2021-01-28 Flagship Pioneering Innovations V, Inc. Fusosome compositions and uses thereof
MX2021001599A (es) 2018-08-09 2021-07-02 Bioverativ Therapeutics Inc Moleculas de acido nucleico y sus usos para la terapia genica no viral.
JP7450945B2 (ja) 2018-08-30 2024-03-18 テナヤ セラピューティクス， インコーポレイテッド ミオカルディンおよびａｓｃｌ１を用いた心細胞リプログラミング
CA3114621A1 (en) 2018-09-28 2020-04-02 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
US20210381001A1 (en) 2018-10-11 2021-12-09 Eidgenössische Technische Hochschule Zürich A method to treat disease using a nucleic acid vector encoding a highly compact multi-input logic gate
WO2020097049A1 (en) 2018-11-05 2020-05-14 American Gene Technologies International Inc. Vector system for expressing regulatory rna
CA3120082A1 (en) * 2018-11-14 2020-05-22 Flagship Pioneering Innovations V, Inc. Fusosome compositions for cns delivery
US20230048166A1 (en) 2018-11-14 2023-02-16 Flagship Pioneering Innovations V, Inc. Fusosome compositions for hematopoietic stem cell delivery
JP2022507453A (ja) 2018-11-14 2022-01-18 フラッグシップ パイオニアリング イノベーションズ ブイ， インコーポレイテッド Ｔ細胞送達のためのフソソーム組成物
EP3891297A4 (en) * 2018-12-03 2022-09-07 California Institute of Technology In situ readout 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
CN113646005A (zh) * 2018-12-21 2021-11-12 宾夕法尼亚州大学信托人 用于drg特异性降低转基因表达的组合物
CA3124524A1 (en) * 2019-01-04 2020-07-09 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
IL317797A (en) 2019-05-30 2025-02-01 Gritstone Bio Inc Modified adenovirus
EP4031673A1 (en) 2019-09-19 2022-07-27 Genethon Gene therapy expression system alleviating cardiac toxicity of fkrp
EP4038182A1 (en) 2019-09-30 2022-08-10 Bioverativ Therapeutics Inc. Lentiviral vector formulations
KR102461417B1 (ko) * 2019-11-11 2022-11-01 주식회사 온코크로스 아데노바이러스 벡터를 포함하는 전립선암 진단용 조성물
WO2021184006A1 (en) * 2020-03-13 2021-09-16 Duke University Compositions for the treatment of nash and associated disorders and methods of using same
EP4127190A4 (en) * 2020-03-30 2024-05-29 Greffex, Inc. IMPROVING THE PRODUCTION OF ADENOVIRUS-BASED GENETIC TRANSFER VECTORS
MX2022014258A (es) * 2020-05-12 2023-02-22 Univ Pennsylvania Composiciones para la reducción específica de un fármaco de la expresión de transgén.
CN115996758A (zh) 2020-06-19 2023-04-21 吉尼松公司 使sgcg在肌肉和心脏中充分表达的基因治疗表达系统
KR20230074703A (ko) 2020-06-24 2023-05-31 바이오버라티브 테라퓨틱스 인크. 유리 인자 viii을 상기 단백질이 발현되도록 변형된 렌티바이러스 벡터의 제제로부터 제거하는 방법
WO2022008711A1 (en) 2020-07-10 2022-01-13 Genethon A novel muscle-specific promoter
JP2023541108A (ja) 2020-08-06 2023-09-28 グリットストーン バイオ インコーポレイテッド マルチエピトープワクチンカセット
TW202227466A (zh) 2020-08-23 2022-07-16 美商百歐維拉提夫治療公司 用於改進封閉端ＤＮＡ（ｃｅＤＮＡ）的產生之經修飾的桿狀病毒系統
KR102626543B1 (ko) * 2020-09-14 2024-01-23 주식회사 제너로스 AIMP2-DX2 및 miR-142의 표적 핵산을 포함하는 재조합 벡터
KR20220035693A (ko) * 2020-09-14 2022-03-22 주식회사 제너로스 표적화된 유전자 전달을 위한 아데노-부속 바이러스 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
EP4221760A4 (en) * 2020-09-30 2025-08-13 Generoath Co Ltd METHODS OF TREATING AGE-RELATED MACULAR DISEASES USING AIMP2-DX2 AND OPTIONALLY A TARGET SEQUENCE FOR MIR-142 AND RELATED COMPOSITIONS
US20220372165A1 (en) 2021-05-05 2022-11-24 Immatics Biotechnologies Gmbh Antigen binding proteins specifically binding prame
AU2022271727A1 (en) * 2021-05-13 2023-12-14 Akouos, Inc. Gene therapy delivery compositions and methods for treating hearing loss
CN117836319A (zh) 2021-08-23 2024-04-05 比奥维拉迪维治疗股份有限公司 优化的因子viii基因
WO2023028440A2 (en) 2021-08-23 2023-03-02 Bioverativ Therapeutics Inc. Baculovirus expression system
CA3232988A1 (en) 2021-09-30 2023-04-06 Bioverativ Therapeutics Inc. Nucleic acids encoding factor viii polypeptides with reduced immunogenicity
WO2023142040A1 (en) * 2022-01-29 2023-08-03 Virogin Biotech Canada Ltd. Transcriptional and translational dual regulated oncolytic herpes simplex virus vectors
US20250281573A1 (en) * 2022-05-13 2025-09-11 Generoath Co., Ltd. Methods of Treating Retinal Degenerative Diseases Using AIMP2-DX2 and Optionally a Target Sequence for miR-142 and Compositions Thereof
WO2025024559A1 (en) 2023-07-24 2025-01-30 Strand Therapeutics Inc. Rna-based synthetic circuit for producing engineered immune cells for an extracorporeal cell therapy
US12281311B1 (en) * 2024-02-20 2025-04-22 Wyvern Pharmaceuticals Inc. Composition for regulating production of interfering ribonucleic acid

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.
CN1115414C (zh) 1993-07-13 2003-07-23 罗纳-布朗克罗莱尔股份有限公司 用于基因治疗的病毒载体
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
ATE266000T1 (de) 1996-08-07 2004-05-15 Darwin Discovery Ltd Hydroxamsäure- und carbonsäure-derivate mit mmp und tnf hemmender wirkung
US6126939A (en) 1996-09-03 2000-10-03 Yeda Research And Development Co. Ltd. Anti-inflammatory dipeptide and pharmaceutical composition thereof
WO1999043816A1 (en) 1998-02-25 1999-09-02 The Dow Chemical Company High affinity humanized anti-tag-72 monoclonal antibodies
IL138022A0 (en) 1998-02-25 2001-10-31 Dow Chemical Co High affinity humanized anti-cea monoclonal antibodies
WO2001053505A2 (en) * 2000-01-21 2001-07-26 Biovex Limited Herpes virus strains for gene therapy
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
WO2003029459A2 (en) * 2001-09-28 2003-04-10 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Micro-rna molecules
SI1504108T1 (sl) 2002-02-01 2013-07-31 Oxford Biomedica (Uk) Limited Lentiviralni vektor
US6998391B2 (en) 2002-02-07 2006-02-14 Supergen.Inc. Method for treating diseases associated with abnormal kinase activity
WO2004058749A1 (en) 2002-12-18 2004-07-15 Vertex Pharmaceuticals Incorporated Benzisoxazole derivatives useful as inhibitors of protein kinases
US7763722B2 (en) 2003-01-17 2010-07-27 University Of Florida Research Foundation, Inc. Small interference RNA gene therapy
WO2004066183A2 (en) 2003-01-22 2004-08-05 European Molecular Biology Laboratory Microrna
EP1616012B1 (en) 2003-04-24 2007-12-19 Fondazione Centro San Raffaele Del Monte Tabor 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
US8106180B2 (en) * 2003-08-07 2012-01-31 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
CN101287834B (zh) * 2005-05-27 2016-11-09 圣拉法埃莱医院有限公司 基因载体
US20070054872A1 (en) * 2005-08-24 2007-03-08 Mirus Bio Corporation Regulatable or conditional expression systems
WO2009002462A1 (en) * 2007-06-22 2008-12-31 The Board Of Trustees Of The Leland Stanford Junior University Pre-mirna loop-modulated target regulation

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JP2013226139A (ja) 2013-11-07
US10000757B2 (en) 2018-06-19
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US20190032059A1 (en) 2019-01-31
KR20080041601A (ko) 2008-05-13
WO2007000668A2 (en) 2007-01-04
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