JP2020536939A5 - - Google Patents

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JP2020536939A5
JP2020536939A5 JP2020520799A JP2020520799A JP2020536939A5 JP 2020536939 A5 JP2020536939 A5 JP 2020536939A5 JP 2020520799 A JP2020520799 A JP 2020520799A JP 2020520799 A JP2020520799 A JP 2020520799A JP 2020536939 A5 JP2020536939 A5 JP 2020536939A5
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vector
virus
introduction
igm
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JP7427584B2 (ja
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JP2020520799A 2017-10-13 2018-10-12 抗ウイルス導入ベクターｉｇｍ応答を減弱化するための方法および組成物 Active JP7427584B2 (ja)

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JP2023159452A JP2024016844A (ja) 2017-10-13 2023-09-25 抗ウイルス導入ベクターｉｇｍ応答を減弱化するための方法および組成物

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US201762572297P 2017-10-13 2017-10-13
US62/572,297 2017-10-13
PCT/US2018/055660 WO2019075360A1 (en) 2017-10-13 2018-10-12 METHODS AND COMPOSITIONS FOR MITIGATING ANTI-VECTOR VIRAL TRANSFER IGM RESPONSES

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JP2020536939A JP2020536939A (ja) 2020-12-17
JP2020536939A5 true JP2020536939A5 (https=) 2021-11-25
JP7427584B2 JP7427584B2 (ja) 2024-02-05

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JP2023159452A Pending JP2024016844A (ja) 2017-10-13 2023-09-25 抗ウイルス導入ベクターｉｇｍ応答を減弱化するための方法および組成物

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US (1) US20190142974A1 (https=)
EP (1) EP3694543A1 (https=)
JP (2) JP7427584B2 (https=)
KR (1) KR20200086670A (https=)
CN (1) CN111542336A (https=)
AU (1) AU2018347583A1 (https=)
BR (1) BR112020007157A2 (https=)
CA (1) CA3078705A1 (https=)
IL (1) IL273825A (https=)
MX (1) MX2020003838A (https=)
WO (1) WO2019075360A1 (https=)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX374963B (es) 2011-04-29 2025-03-06 Selecta Biosciences Inc Nanoportadores sintéticos tolerogénicos para reducir las respuestas de anticuerpos.
BR122020023215B1 (pt) 2013-05-03 2022-11-22 Selecta Biosciences, Inc Composição e kit de nanocarreadores sintéticos tolerogênicos para reduzir ou prevenir anafilaxia em resposta a um antígeno não alergênico
JP7242180B2 (ja) 2014-09-07 2023-03-20 セレクタ バイオサイエンシーズ インコーポレーテッド 抗ウイルス導入ベクター免疫応答を減弱化するための方法および組成物
ES3036482T3 (en) 2017-03-11 2025-09-19 Cartesian Therapeutics Inc Methods and compositions related to combined treatment with anti-inflammatories and synthetic nanocarriers comprising an immunosuppressant
CN114206396A (zh) * 2019-05-28 2022-03-18 西莱克塔生物科技公司 用于减弱抗病毒转移载体免疫应答的方法和组合物
AU2020287627A1 (en) 2019-06-04 2021-11-18 Selecta Biosciences, Inc. Formulations and doses of PEGylated uricase
US20220288231A1 (en) * 2019-08-12 2022-09-15 Generation Bio Co. Methods and compositions for reducing gene or nucleic acid therapy-related immune responses
WO2021067598A1 (en) 2019-10-04 2021-04-08 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
AU2021225955A1 (en) * 2020-02-26 2022-09-22 Selecta Biosciences, Inc. Methods and compositions using synthetic nanocarriers comprising immunosuppressant
IL297274A (en) * 2020-04-14 2022-12-01 Selecta Biosciences Inc Methods and compositions for inducing autophagy
WO2022140622A1 (en) * 2020-12-22 2022-06-30 University Of Virginia Patent Foundation Hyaluronan is a driver of covid-19 severity
WO2022147575A1 (en) * 2021-01-04 2022-07-07 University Of Florida Research Foundation, Incorporated Methods and compositions for treatment of friedreich's ataxia
WO2022221529A1 (en) 2021-04-16 2022-10-20 Asklepios Biopharmaceutical, Inc. Rational polyploid aav virions that cross the blood brain barrier and elicit reduced humoral response
US20230141563A1 (en) * 2021-10-12 2023-05-11 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
JP2024545594A (ja) * 2021-11-14 2024-12-10 カーティザン セラピューティクス，インコーポレーテッド ウイルスベクターでの複数回投薬
WO2023172624A1 (en) * 2022-03-09 2023-09-14 Selecta Biosciences, Inc. Immunosuppressants in combination with anti-igm agents and related dosing
US20230372535A1 (en) * 2022-03-25 2023-11-23 Selecta Biosciences, Inc. Synthetic nanocarriers comprising an immunosuppressant in combination with high affinity il-2 receptor agonists and anti-igm agents
WO2025256978A1 (en) 2024-06-13 2025-12-18 Bayer Aktiengesellschaft Variants of igm and igg cleaving enzymes
WO2025256977A1 (en) 2024-06-13 2025-12-18 Bayer Aktiengesellschaft Novel igm and igg cleaving enzymes

Family Cites Families (134)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4946929A (en) 1983-03-22 1990-08-07 Massachusetts Institute Of Technology Bioerodible articles useful as implants and prostheses having predictable degradation rates
US4638045A (en) 1985-02-19 1987-01-20 Massachusetts Institute Of Technology Non-peptide polyamino acid bioerodible polymers
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US4806621A (en) 1986-01-21 1989-02-21 Massachusetts Institute Of Technology Biocompatible, bioerodible, hydrophobic, implantable polyimino carbonate article
CA1340581C (en) 1986-11-20 1999-06-08 Joseph P. Vacanti Chimeric neomorphogenesis of organs by controlled cellular implantation using artificial matrices
US5804178A (en) 1986-11-20 1998-09-08 Massachusetts Institute Of Technology Implantation of cell-matrix structure adjacent mesentery, omentum or peritoneum tissue
US5736372A (en) 1986-11-20 1998-04-07 Massachusetts Institute Of Technology Biodegradable synthetic polymeric fibrous matrix containing chondrocyte for in vivo production of a cartilaginous structure
US5019379A (en) 1987-07-31 1991-05-28 Massachusetts Institute Of Technology Unsaturated polyanhydrides
US5010167A (en) 1989-03-31 1991-04-23 Massachusetts Institute Of Technology Poly(amide-and imide-co-anhydride) for biological application
GB8918616D0 (en) 1989-08-15 1989-09-27 Univ Glasgow Herpes simplex virus type 1 mutant
US5849572A (en) 1990-10-10 1998-12-15 Regents Of The University Of Michigan HSV-1 vector containing a lat promoter
US5804413A (en) 1992-07-31 1998-09-08 University Of Pittsburgh Of The Commonwealth System Of Higher Education Herpes simplex virus strains for gene transfer
US5399665A (en) 1992-11-05 1995-03-21 Massachusetts Institute Of Technology Biodegradable polymers for cell transplantation
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5512600A (en) 1993-01-15 1996-04-30 Massachusetts Institute Of Technology Preparation of bonded fiber structures for cell implantation
US5514378A (en) 1993-02-01 1996-05-07 Massachusetts Institute Of Technology Biocompatible polymer membranes and methods of preparation of three dimensional membrane structures
US5565215A (en) 1993-07-23 1996-10-15 Massachusettes Institute Of Technology Biodegradable injectable particles for imaging
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
AU687117B2 (en) 1993-10-25 1998-02-19 Canji, Inc. Recombinant adenoviral vector and methods of use
ATE336587T1 (de) 1994-06-10 2006-09-15 Genvec Inc Adenoviren-vektor systeme und zelllinien
US6007845A (en) 1994-07-22 1999-12-28 Massachusetts Institute Of Technology Nanoparticles and microparticles of non-linear hydrophilic-hydrophobic multiblock copolymers
GB9415319D0 (en) 1994-07-29 1994-09-21 Medical Res Council HSV viral vector
US5846782A (en) 1995-11-28 1998-12-08 Genvec, Inc. Targeting adenovirus with use of constrained peptide motifs
US5716404A (en) 1994-12-16 1998-02-10 Massachusetts Institute Of Technology Breast tissue engineering
US6123727A (en) 1995-05-01 2000-09-26 Massachusetts Institute Of Technology Tissue engineered tendons and ligaments
IL160406A0 (en) 1995-06-15 2004-07-25 Crucell Holland Bv A cell harbouring nucleic acid encoding adenoritus e1a and e1b gene products
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
US5801030A (en) 1995-09-01 1998-09-01 Genvec, Inc. Methods and vectors for site-specific recombination
US5837511A (en) 1995-10-02 1998-11-17 Cornell Research Foundation, Inc. Non-group C adenoviral vectors
US6095148A (en) 1995-11-03 2000-08-01 Children's Medical Center Corporation Neuronal stimulation using electrically conducting polymers
US5902599A (en) 1996-02-20 1999-05-11 Massachusetts Institute Of Technology Biodegradable polymer networks for use in orthopedic and dental applications
US6893664B1 (en) 1996-06-17 2005-05-17 Powderject Research Limited Particle delivery techniques
AU735648B2 (en) 1996-07-12 2001-07-12 Ariad Pharmaceuticals, Inc. Materials and method for treating or preventing pathogenic fungal infection
US5814500A (en) 1996-10-31 1998-09-29 The Johns Hopkins University School Of Medicine Delivery construct for antisense nucleic acids and methods of use
EP0968226A4 (en) 1996-12-27 2001-02-14 Icn Pharmaceuticals OLIGOAPTAMERS WITH G-ENRICHED REGIONS AND METHODS FOR MODULATING AN IMMUNE RESPONSE
US5849561A (en) 1997-05-22 1998-12-15 Cornell Research Foundation, Inc. Method for the production of non-group C adenoviral vectors
EP0996735B1 (en) 1997-06-09 2003-12-10 Genvec, Inc. Chimeric vectors comprising a phage packaging site and a portion derived from the genome of a eukaryotic virus
US5837752A (en) 1997-07-17 1998-11-17 Massachusetts Institute Of Technology Semi-interpenetrating polymer networks
EP0894853A1 (en) 1997-07-24 1999-02-03 Gesellschaft für Biotechnologische Forschung mbH (GBF) Transcriptional silencer protein NRF, nucleic acid molecules encoding it and their use
AU8605598A (en) 1997-07-31 1999-02-22 University Of Pittsburgh Targeted hsv vectors
JP2001517455A (ja) 1997-09-23 2001-10-09 ジェンベク、インコーポレイティッド 真核生物ウイルスベクター構築用プラスミド
US6632922B1 (en) 1998-03-19 2003-10-14 The Regents Of The University Of California Methods and compositions for controlled polypeptide synthesis
US6686446B2 (en) 1998-03-19 2004-02-03 The Regents Of The University Of California Methods and compositions for controlled polypeptide synthesis
US6506577B1 (en) 1998-03-19 2003-01-14 The Regents Of The University Of California Synthesis and crosslinking of catechol containing copolypeptides
IL139040A0 (en) 1998-04-22 2001-11-25 Genvec Inc Efficient purification of adenovirus
US6436392B1 (en) 1998-05-20 2002-08-20 University Of Iowa Research Foundation Adeno-associated virus vectors
US5965358A (en) 1998-08-26 1999-10-12 Genvec, Inc. Method for assessing the relative purity of viral gene transfer vector stocks
WO2000020040A1 (en) 1998-10-08 2000-04-13 University Of Massachusetts Controlling gene expression in living cells
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
JP4751556B2 (ja) 2000-02-28 2011-08-17 ジーンシーグス， インコーポレイテッド ナノカプセルカプセル化システムおよび方法
US6168941B1 (en) 2000-04-07 2001-01-02 Genvec, Inc. Method of producing adenoviral vector stocks
CA2407897A1 (en) 2000-05-05 2001-11-15 Cytos Biotechnology Ag Molecular antigen arrays and vaccines
ATE318923T1 (de) 2000-06-01 2006-03-15 Univ North Carolina Doppelsträngige parvovirus-vektoren
CA2319928A1 (en) 2000-09-18 2002-03-18 Vasogen Ireland Limited Apoptosis-mimicking synthetic entities and use thereof in medical treatments
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
US6447995B1 (en) 2000-10-04 2002-09-10 Genvec, Inc. Utilizing intrinsic fluorescence to detect adenovirus
GB0025414D0 (en) 2000-10-16 2000-11-29 Consejo Superior Investigacion Nanoparticles
CA2440367A1 (en) 2000-10-20 2002-08-15 Canji, Inc. Aptamer-mediated regulation of gene expression
US7122181B2 (en) 2000-12-19 2006-10-17 Research Development Foundation Lentiviral vector-mediated gene transfer and uses thereof
JP4608210B2 (ja) 2001-05-31 2011-01-12 ノバルティス バクシンズ アンド ダイアグノスティックス，インコーポレーテッド キメラアルファウイルスレプリコン粒子
ATE527347T1 (de) 2001-08-02 2011-10-15 Inst Clayton De La Rech Verfahren und zusammensetzungen im zusammenhang mit verbesserten lentivirusvektor- produktionssystemen
WO2003020797A1 (en) 2001-08-30 2003-03-13 The Regents Of The University Of California Transition metal initiators for controlled poly (beta-peptide) synthesis from beta-lactam monomers
ES2330202T5 (es) 2001-09-06 2014-01-20 Alphavax, Inc. Sistemas vectores basados en replicones de alfavirus
RU2305708C2 (ru) 2001-10-02 2007-09-10 Энститю Клейтон Де Ля Решерш Рекомбинантный лентивирусный вектор, клетка-хозяин, трансдуцированная лентивирусным вектором, способ ее трансдукции и применение
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
DE60333487D1 (de) 2002-12-13 2010-09-02 Genetix Pharmaceuticals Inc Therapeutische retrovirus-vektoren für gentherapie
US7510872B2 (en) 2003-02-26 2009-03-31 Nationwide Children's Hospital Recombinant adeno-associated virus production
AU2004224761A1 (en) 2003-03-26 2004-10-07 Cytos Biotechnology Ag HIV-peptide-carrier-conjugates
US7186699B2 (en) 2003-06-03 2007-03-06 Cell Genesys, Inc. Method for treating cancer by vector-mediated delivery of one or more anti-angiogenic or pro-apoptotic genes
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
WO2005038035A2 (en) 2003-10-15 2005-04-28 University Of Iowa Research Foundation Methods for producing and using in vivo pseudotyped retroviruses
MXPA06006738A (es) 2003-12-19 2006-08-31 Univ North Carolina Metodos para fabricar micro- y nano-estructuras aisladas utilizando litografia suave o de impresion.
ES2423060T3 (es) 2004-03-12 2013-09-17 Alnylam Pharmaceuticals, Inc. Agentes iRNA que tienen como diana al VEGF
EP2933332A1 (en) 2004-06-28 2015-10-21 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
WO2006080951A2 (en) 2004-07-01 2006-08-03 Yale University Targeted and high density drug loaded polymeric materials
DE602005027479D1 (de) 2004-09-24 2011-05-26 Alnylam Pharmaceuticals Inc Targeting von zwischenprodukten zur gegenstrangreplikation von einzelstrangigen viren durch rnai
US20060089324A1 (en) 2004-10-22 2006-04-27 Sailen Barik RNAi modulation of RSV, PIV and other respiratory viruses and uses thereof
US7790878B2 (en) 2004-10-22 2010-09-07 Alnylam Pharmaceuticals, Inc. RNAi modulation of RSV, PIV and other respiratory viruses and uses thereof
NZ560936A (en) 2005-02-03 2010-04-30 Benitec Inc RNAI expression constructs
MX2007010008A (es) 2005-02-16 2008-01-18 Lentigen Corp Vectores lentivirales y su uso.
JP2008541730A (ja) 2005-05-23 2008-11-27 ヴァクシン インコーポレイテッド 高力価かつ複製コンピテントアデノウイルス不含である組換えアデノウイルスベクターの迅速な作成法
WO2007002718A2 (en) 2005-06-27 2007-01-04 Alnylam Pharmaceuticals, Inc. Rnai modulation of hif-1 and theraputic uses thereof
DE602006019916D1 (de) 2005-12-12 2011-03-10 Canji Inc Adenovirale expressionsvektoren mit einer expressionskassette im e1-bereich und einer inaktivierten e2b-polymerase
GB0526211D0 (en) 2005-12-22 2006-02-01 Oxford Biomedica Ltd Viral vectors
ES2465996T3 (es) 2006-05-25 2014-06-09 Sangamo Biosciences, Inc. Métodos y composiciones para la inactivación genética
CN103849629B (zh) 2006-06-21 2017-06-09 尤尼克尔Ip股份有限公司 具有经修饰的用于在昆虫细胞中产生aav的aav‑rep78翻译起始密码子的载体
ES2586210T3 (es) 2006-12-14 2016-10-13 Sangamo Biosciences, Inc. Proteínas de dedo de zinc no canónicas optimizadas
CA2683063A1 (en) 2007-04-09 2008-10-16 Chimeros, Inc. Self-assembling nanoparticle drug delivery system
WO2008127532A1 (en) 2007-04-12 2008-10-23 Emory University Novel strategies for delivery of active agents using micelles and particles
CA2685127C (en) 2007-04-23 2019-01-08 Alnylam Pharmaceuticals, Inc. Glycoconjugates of rna interference agents
DK2185192T3 (en) 2007-08-03 2019-02-18 Pasteur Institut LENTIVIRAL TRANSFER VECTORS AND THEIR MEDICAL USES
JP2011500569A (ja) 2007-10-12 2011-01-06 マサチューセッツ インスティテュート オブ テクノロジー ワクチンナノテクノロジー
EP2215223B1 (en) 2007-10-31 2013-05-01 Precision Biosciences, Inc. Rationally-designed single-chain meganucleases with non-palindromic recognition sequences
JP2011505809A (ja) 2007-12-07 2011-03-03 プレシジョン バイオサイエンシズ，インク． ヒトゲノムのＤＮａｓｅ高感受性領域に見出される認識配列を有する合理的に設計されたメガヌクレアーゼ
WO2009106999A2 (en) 2008-02-28 2009-09-03 Deutsches Krebsforschungszentrum, Stiftung Des Öffentlichen Rechts Hollow nanoparticles and uses thereof
WO2009114321A2 (en) 2008-03-11 2009-09-17 Precision Biosciencs, Inc. Rationally-designed meganucleases for maize genome engineering
WO2009146179A1 (en) 2008-04-15 2009-12-03 University Of Iowa Research Foundation Zinc finger nuclease for the cftr gene and methods of use thereof
WO2010033722A2 (en) 2008-09-17 2010-03-25 Isogenis, Inc. Construction of fully-deleted adenovirus-based gene delivery vectors and uses thereof
CN101676291B (zh) 2008-09-18 2012-05-09 上海海和药物研究开发有限公司 一类雷帕霉素碳酸酯类似物、其药物组合物及其制备方法和用途
BR122020021379B1 (pt) 2008-10-24 2021-05-11 Sarepta Therapeutics, Inc. oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular
US20120015899A1 (en) 2008-10-25 2012-01-19 Plant Bioscience, Limited Modified plant virus particles and uses therefor
WO2010114948A2 (en) 2009-04-02 2010-10-07 University Of Florida Research Foundation, Inc. An inducible system for highly efficient production of recombinant adeno-associated virus (raav) vectors
EP2480659A2 (en) 2009-09-24 2012-08-01 Cellectis Meganuclease reagents of uses thereof for treating genetic diseases caused by frame shift/non sense mutations
WO2011043719A1 (en) 2009-10-05 2011-04-14 Ya-Fang Mei Replicating viral vectors for gene therapy
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
TR201816523T4 (tr) 2009-11-12 2018-11-21 Univ Western Australia Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler.
WO2011072246A2 (en) 2009-12-10 2011-06-16 Regents Of The University Of Minnesota Tal effector-mediated dna modification
EP2660318A1 (en) 2010-02-09 2013-11-06 Sangamo BioSciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
US20130156845A1 (en) 2010-04-29 2013-06-20 Alnylam Pharmaceuticals, Inc. Lipid formulated single stranded rna
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
EP2675484B1 (en) 2011-02-14 2018-05-30 The Children's Hospital of Philadelphia Improved aav8 vector with enhanced functional activity and methods of use thereof
US20140155469A1 (en) 2011-04-19 2014-06-05 The Research Foundation Of State University Of New York Adeno-associated-virus rep sequences, vectors and viruses
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
JP2014530607A (ja) 2011-10-11 2014-11-20 アリオフタ・アーゲーＡｌｉｏｐｈｔｈａ Ａｇ 人工転写因子の送達による受容体発現の調節
EP3730618A1 (en) 2011-11-18 2020-10-28 Alnylam Pharmaceuticals, Inc. Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases
US20150166969A1 (en) 2012-02-24 2015-06-18 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
AU2013240248B2 (en) 2012-03-26 2018-12-20 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Delivery of packaged RNA to mammalian cells
NZ701693A (en) 2012-04-18 2017-02-24 The Children’S Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using aav capsid variants
US9738879B2 (en) 2012-04-27 2017-08-22 Duke University Genetic correction of mutated genes
AU2013296321B2 (en) 2012-08-03 2019-05-16 Alnylam Pharmaceuticals, Inc. Modified RNAi agents
HUE054260T2 (hu) 2012-09-06 2021-08-30 Univ Chicago Antiszensz polinukleotidok exon-ugrás indukálására és eljárások disztrófiák kezelésére
JP2015529466A (ja) 2012-09-14 2015-10-08 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 鎌状赤血球症の幹細胞遺伝子治療のためのレンチウイルスベクター
US9181535B2 (en) 2012-09-24 2015-11-10 The Chinese University Of Hong Kong Transcription activator-like effector nucleases (TALENs)
US9624510B2 (en) 2013-03-01 2017-04-18 The Wistar Institute Adenoviral vectors comprising partial deletions of E3
US20140329762A1 (en) 2013-03-15 2014-11-06 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
BR122020023215B1 (pt) * 2013-05-03 2022-11-22 Selecta Biosciences, Inc Composição e kit de nanocarreadores sintéticos tolerogênicos para reduzir ou prevenir anafilaxia em resposta a um antígeno não alergênico
EA038792B1 (ru) 2013-05-22 2021-10-20 Элнилэм Фармасьютикалз, Инк. КОМПОЗИЦИИ НА ОСНОВЕ RNAi Serpina1 И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
US9873907B2 (en) 2013-05-29 2018-01-23 Agilent Technologies, Inc. Method for fragmenting genomic DNA using CAS9
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
JP7242180B2 (ja) * 2014-09-07 2023-03-20 セレクタ バイオサイエンシーズ インコーポレーテッド 抗ウイルス導入ベクター免疫応答を減弱化するための方法および組成物
KR102601922B1 (ko) 2014-11-05 2023-11-15 셀렉타 바이오사이언시즈, 인크. 안정한 과-포화 상태로 라파마이신을 포함하는 합성 나노담체와 관련된 방법 및 조성물
MX2019008143A (es) * 2017-01-07 2020-01-13 Selecta Biosciences Inc Dosificación sistemática de inmunosupresores acoplados a nanoportadores sintéticos.

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