SG162726A1 - Gene vector comprising mi-rna - Google Patents

Gene vector comprising mi-rna

Info

Publication number
SG162726A1
SG162726A1 SG201003686-1A SG2010036861A SG162726A1 SG 162726 A1 SG162726 A1 SG 162726A1 SG 2010036861 A SG2010036861 A SG 2010036861A SG 162726 A1 SG162726 A1 SG 162726A1
Authority
SG
Singapore
Prior art keywords
rna
gene vector
gene
vector
mirna sequence
Prior art date
Application number
SG201003686-1A
Other languages
English (en)
Inventor
Luigi Naldini
Brian David Brown
Original Assignee
San Raffaele Centro Fond
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 San Raffaele Centro Fond, Fond Telethon filed Critical San Raffaele Centro Fond
Publication of SG162726A1 publication Critical patent/SG162726A1/en

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    • C12N15/113—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
    • 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 (151)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8592384B2 (en) 2005-04-04 2013-11-26 The Board Of Regents Of The University Of Texas System Micro-RNA's that regulate muscle cells
DK2002003T3 (en) * 2005-05-27 2016-03-21 Ospedale San Raffaele Srl Gene vector comprising miRNA
WO2006128245A1 (en) * 2005-06-03 2006-12-07 Southern Adelaide Health Service-Flinders Medical Centre Targeting cells with altered microrna expression
DK2056882T3 (da) 2006-08-01 2012-11-19 Univ Texas Identifikation af en mikro-rna, som aktiverer ekspression af en tung kæde med beta-myosin
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
CA2694928A1 (en) 2007-07-31 2009-02-05 Board Of Regents, The University Of Texas System Micro-rnas that control myosin expression and myofiber identity
DK2182969T3 (en) 2007-07-31 2017-02-27 Univ Texas Micro-RNA family that modulates fibrosis and its applications
WO2009062169A2 (en) 2007-11-09 2009-05-14 The Board Of Regents Of The University Of Texas System Micro-rnas of the mir-15 family modulate cardiomyocyte survival and cardiac repair
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
WO2010045659A1 (en) 2008-10-17 2010-04-22 American Gene Technologies International Inc. Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules
US9938540B2 (en) 2008-11-12 2018-04-10 Ospedale San Raffaele S.R.L. 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
US8629119B2 (en) 2009-02-04 2014-01-14 The Board Of Regents, The University Of Texas System Dual targeting of MIR-208 and MIR-499 in the treatment of cardiac disorders
JP5871791B2 (ja) * 2009-04-29 2016-03-01 アカデミス・メディス・セントルム・ベイ・デ・ウニフェルジテイト・ファン・アムステルダムAcademisch Medisch Centrum Bij De Universiteit Van Amsterdam 心不全または心不全の危険性を中和、予防および/または決定する手段および方法
EP2425001A4 (en) 2009-04-30 2012-11-14 Univ California ANTI-HIV COMBINATION VECTORS, TARGETING VECTORS AND METHOD OF USE
MX2011011508A (es) * 2009-04-30 2012-02-13 San Raffaele Centro Fond Vector genico.
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
EA022786B1 (ru) 2009-07-31 2016-03-31 Этрис Гмбх Рнк с комбинацией из немодифицированных и модифицированных нуклеотидов для экспрессии белков
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
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
DK2561073T3 (en) 2010-04-23 2016-12-12 Univ Massachusetts Aav vectors targeted to central nervous system and methods of use thereof
CA2833912C (en) 2010-04-23 2021-09-21 University Of Massachusetts Aav-based treatment of cholesterol-related disorders
CA3050894C (en) 2010-04-23 2022-10-18 University Of Massachusetts Multicistronic expression constructs
US9309534B2 (en) 2010-07-12 2016-04-12 Universidad Autonoma De Barcelona Gene therapy composition for use in diabetes treatment
WO2012008302A1 (ja) 2010-07-12 2012-01-19 国立大学法人鳥取大学 miRNA導入による新規hiPSC作製法
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
WO2012145624A2 (en) 2011-04-21 2012-10-26 University Of Massachusetts Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
ES2632212T3 (es) * 2011-08-04 2017-09-11 Yeda Research And Development Co. Ltd. miR-135 y composiciones que lo comprenden para el tratamiento de afecciones médicas asociadas con la serotonina
ES2872081T3 (es) 2011-08-05 2021-11-02 Regeneron Pharma Ratones con cadena ligera universal humanizada
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
WO2013151664A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins
US10501513B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides for the production of oncology-related proteins and peptides
US10501512B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides
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
EP2864482A4 (en) 2012-06-21 2016-02-10 Miragen Therapeutics Inc OLIGONUCLEOTIDE INHIBITORS COMPRISING A BLOCKED NUCLEIC ACID PATTERN
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
PT2922554T (pt) 2012-11-26 2022-06-28 Modernatx Inc Arn modificado nas porções terminais
EP3434774A1 (en) * 2013-01-17 2019-01-30 ModernaTX, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
RS61387B2 (sr) 2013-02-15 2024-06-28 Bioverativ Therapeutics Inc Gen optimizovanog faktora 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
WO2015127128A2 (en) 2014-02-19 2015-08-27 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
WO2015164786A1 (en) 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
US11008561B2 (en) 2014-06-30 2021-05-18 Bioverativ Therapeutics Inc. Optimized factor IX gene
US10973931B2 (en) 2014-09-16 2021-04-13 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
WO2016065001A1 (en) 2014-10-21 2016-04-28 University Of Massachusetts Recombinant aav variants and uses thereof
US11085055B2 (en) 2014-12-05 2021-08-10 Universitat Autonoma De Barcelona Viral vectors for the treatment of diabetes
HK1249108A1 (zh) 2015-01-20 2018-10-26 米拉根医疗股份有限公司 Mir-92抑制剂及其用途
EP3256170B1 (en) 2015-02-13 2020-09-23 University of Massachusetts Compositions and methods for transient delivery of nucleases
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
US11980663B2 (en) 2015-07-08 2024-05-14 American Gene Technologies International Inc. HIV pre-immunization and immunotherapy
EP3359670B2 (en) 2015-10-05 2024-02-14 ModernaTX, Inc. Methods for therapeutic administration of messenger ribonucleic acid drugs
EP3364949A4 (en) 2015-10-22 2019-07-31 ModernaTX, Inc. CANCER VACCINES
US10188750B1 (en) 2015-10-23 2019-01-29 University Of South Florida Self-replicating cell selective gene delivery compositions, methods, and uses thereof
IL297238B2 (en) 2016-01-15 2024-08-01 American Gene Tech Int Inc Methods and preparations for gamma-delta T-cell activation
US10137144B2 (en) 2016-01-15 2018-11-27 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta T-cells
AU2017212713B2 (en) 2016-01-27 2023-01-19 Oncorus, Inc. Oncolytic viral vectors and uses thereof
KR20180118659A (ko) 2016-02-01 2018-10-31 바이오버라티브 테라퓨틱스 인크. 최적화된 viii 인자 유전자
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
CA3012344A1 (en) 2016-02-12 2017-08-17 University Of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
ES2911448T3 (es) 2016-03-09 2022-05-19 American Gene Tech Int Inc Vectores combinados y métodos para el tratamiento del cáncer
ES2796488T3 (es) * 2016-04-08 2020-11-27 Krystal Biotech Inc Composiciones para el uso en métodos para el tratamiento de heridas, trastornos y enfermedades de la piel
US12128113B2 (en) * 2016-05-18 2024-10-29 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
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
WO2018017882A1 (en) 2016-07-21 2018-01-25 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
KR20190075964A (ko) 2016-10-13 2019-07-01 유니버시티 오브 매사추세츠 Aav 캡시드 설계
WO2018127702A1 (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
CN117821509A (zh) 2017-04-03 2024-04-05 编码治疗公司 组织选择性转基因表达
US11820999B2 (en) 2017-04-03 2023-11-21 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
KR102945203B1 (ko) * 2017-05-08 2026-03-31 시애틀 프로젝트 코포레이션 알파바이러스 신생항원 벡터
US11576872B2 (en) 2017-05-08 2023-02-14 Flagship Pioneering Innovations V, Inc. Compositions for facilitating membrane fusion and uses thereof
AU2018264996A1 (en) 2017-05-09 2019-12-05 University Of Massachusetts Methods of treating Amyotrophic Lateral Sclerosis (ALS)
CA3099990A1 (en) 2017-05-12 2018-11-15 University Of Massachusetts Viral vector production
KR102765034B1 (ko) 2017-05-24 2025-02-11 유니버시타트 아우토노마 데 바르셀로나 섬유아세포 성장 인자 21 (fgf21) 코딩 서열을 포함하는 바이러스 발현 컨스트럭트
US12559769B2 (en) 2017-06-16 2026-02-24 American Gene Technologies International Inc. Methods and compositions for the activation of tumor cytotoxicity via human gamma-delta t-cells
US20190016802A1 (en) 2017-07-14 2019-01-17 Immatics Biotechnologies Gmbh Dual specificity polypeptide molecule
MX2020000993A (es) * 2017-07-26 2020-07-22 Oncorus Inc Vectores virales oncolíticos y usos de estos.
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
CA3077594C (en) * 2017-10-10 2022-11-29 Nantbio, Inc. Modified ec7 cells having low toxicity to viral production payloads
CN118345115A (zh) 2017-12-01 2024-07-16 编码治疗公司 工程化的dna结合蛋白
IL275273B2 (en) * 2017-12-15 2024-09-01 Medizinische Hochschule Hannover Improved compound for treatment of heart failure
WO2019133847A1 (en) 2017-12-29 2019-07-04 Oncorus, Inc. Oncolytic viral delivery of therapeutic polypeptides
KR20240134250A (ko) 2018-01-04 2024-09-06 아이코닉 테라퓨틱스, 인코포레이티드 항-조직 인자 항체, 항체-약물 결합체, 및 관련 방법
US20210038744A1 (en) 2018-02-01 2021-02-11 Bioverativ Therapeutics Inc. Use of lentiviral vectors expressing factor viii
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
CN112367973A (zh) 2018-05-15 2021-02-12 旗舰先锋创新V股份有限公司 融合剂脂质体组合物和其用途
CA3105953A1 (en) 2018-07-09 2020-01-16 Flagship Pioneering Innovations V, Inc. Fusosome compositions and uses thereof
JP7602454B2 (ja) 2018-08-09 2024-12-18 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 核酸分子及び非ウイルス遺伝子治療のためのそれらの使用
KR20210102870A (ko) 2018-08-30 2021-08-20 테나야 테라퓨틱스, 인코포레이티드 미오카르딘 및 ascl1을 사용한 심장 세포 재프로그래밍
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
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
KR20210090650A (ko) 2018-11-07 2021-07-20 그릿스톤 바이오, 인코포레이티드 알파바이러스 신생항원 벡터 및 인터페론 억제제
KR20210131991A (ko) 2018-11-14 2021-11-03 플래그쉽 파이어니어링 이노베이션스 브이, 인크. 구획-특이적 카고 전달을 위한 조성물 및 방법
US20230048166A1 (en) 2018-11-14 2023-02-16 Flagship Pioneering Innovations V, Inc. Fusosome compositions for hematopoietic stem cell delivery
WO2020102503A2 (en) 2018-11-14 2020-05-22 Flagship Pioneering Innovations V, Inc. Fusosome compositions for t cell delivery
EP3880179A2 (en) * 2018-11-14 2021-09-22 Flagship Pioneering Innovations V, Inc. Fusosome compositions for cns delivery
US11421273B2 (en) * 2018-12-03 2022-08-23 California Institute Of Technology In situ readout of DNA barcodes
CA3121786A1 (en) 2018-12-06 2020-06-11 Bioverativ Therapeutics Inc. Use of lentiviral vectors expressing factor ix
MX2021007600A (es) * 2018-12-21 2021-08-11 Univ Pennsylvania Composiciones para la reduccion de la expresion transgenica especifica de drg.
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
WO2020219766A1 (en) * 2019-04-24 2020-10-29 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
SG11202113048SA (en) 2019-05-29 2021-12-30 Encoded Therapeutics Inc Compositions and methods for selective gene regulation
SG11202113187WA (en) 2019-05-30 2021-12-30 Gritstone Bio Inc Modified adenoviruses
EP4031673A1 (en) 2019-09-19 2022-07-27 Genethon Gene therapy expression system alleviating cardiac toxicity of fkrp
JP7803014B2 (ja) 2019-09-30 2026-01-21 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 レンチウイルスベクター製剤
KR102461417B1 (ko) * 2019-11-11 2022-11-01 주식회사 온코크로스 아데노바이러스 벡터를 포함하는 전립선암 진단용 조성물
US20230210883A1 (en) * 2020-03-13 2023-07-06 Duke University Compositions for the treatment of nash and associated disorders and methods of using same
MX2022012059A (es) * 2020-03-30 2023-02-14 Greffex Inc Aumento de la produccion de vectores de genetransferencia a base de adenovirus.
JP7862321B2 (ja) * 2020-04-27 2026-05-19 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Cdkl5欠損症(cdd)の治療において有用な組成物
IL298138A (en) * 2020-05-12 2023-01-01 Univ Pennsylvania Compositions for drg-specific reduction of transgene expression
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
JP2023537565A (ja) 2020-06-24 2023-09-04 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 タンパク質を発現するように改変されたレンチウイルスベクターの調製物から遊離第viii因子を除去する方法
BR112023000305A2 (pt) 2020-07-10 2023-03-21 Genethon Promotor, sistema de expressão e composição farmacêutica
IL300026A (en) 2020-08-06 2023-03-01 Gritstone Bio Inc Multiepitope vaccine cassettes
AU2021330936A1 (en) 2020-08-23 2023-05-04 Bioverativ Therapeutics Inc. MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA)
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
WO2022233956A1 (en) 2021-05-05 2022-11-10 Immatics Biotechnologies Gmbh Antigen binding proteins specifically binding prame
MX2023013394A (es) * 2021-05-13 2023-12-14 Akouos Inc Composiciones de suministro de terapia genica y metodos para tratar perdida auditiva.
US20230084036A1 (en) 2021-08-23 2023-03-16 Bioverativ Therapeutics Inc. Optimized factor viii genes
WO2023028440A2 (en) 2021-08-23 2023-03-02 Bioverativ Therapeutics Inc. Baculovirus expression system
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
CN119343154A (zh) * 2022-05-13 2025-01-21 杰内罗蒂股份有限公司 使用AIMP2-DX2和任选的miR-142靶序列及其组合物治疗视网膜退行性疾病的方法
KR20260042228A (ko) 2023-07-24 2026-03-30 스트랜드 세러퓨틱스 인코포레이티드 체외 세포 요법을 위한 조작된 면역 세포를 생산하기 위한 rna 기반 합성 회로
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.
CA2144040A1 (fr) 1993-07-13 1995-01-26 Michel Perricaudet Vecteurs adenoviraux defectifs et utilisation en therapie genique
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
CZ297278B6 (cs) 1996-08-07 2006-10-11 Darwin Discovery Limited Deriváty hydroxamové a karboxylové kyseliny a jejich pouzití
US6126939A (en) 1996-09-03 2000-10-03 Yeda Research And Development Co. Ltd. Anti-inflammatory dipeptide and pharmaceutical composition thereof
IL138021A0 (en) * 1998-02-25 2001-10-31 Dow Chemical Co High affinity humanized anti-tag-72 monoclonal antibodies
AU752494B2 (en) * 1998-02-25 2002-09-19 Dow Chemical Company, The High affinity humanized ANTI-CEA monoclonal antibodies
JP4810042B2 (ja) * 2000-01-21 2011-11-09 バイオヴェックス リミテッド ヘルペスウイルス株
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
EP2428570B1 (en) 2001-09-28 2018-05-23 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Microrna molecules
DK2348119T3 (en) 2002-02-01 2017-08-21 Oxford Biomedica (Uk) Ltd Polycistronic lentiviral vector
US6998391B2 (en) 2002-02-07 2006-02-14 Supergen.Inc. Method for treating diseases associated with abnormal kinase activity
US7226919B2 (en) 2002-12-18 2007-06-05 Vertex Pharmaceuticals Inc. Compositions useful as inhibitors of protein kinases
CA2513679A1 (en) 2003-01-17 2004-08-05 University Of Florida Small interference rna gene therapy
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
WO2005013901A2 (en) 2003-07-31 2005-02-17 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for use in modulation of small non-coding rnas
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
WO2005056761A2 (en) * 2003-12-05 2005-06-23 Massachusetts Institute Of Technology REAGENTS AND METHODS FOR IDENTIFICATION OF RNAi PATHWAY GENES AND CHEMICAL MODULATORS OF RNAi
EP2990410A1 (en) * 2004-08-10 2016-03-02 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
DK2002003T3 (en) * 2005-05-27 2016-03-21 Ospedale San Raffaele Srl Gene vector comprising miRNA
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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