DK0871754T3 - Fremgangsmåde til produktion af retrovirale emballeringscellelinjer, som producerer en retroviral supernatant med højeffektiv transduktionsgrad - Google Patents

Fremgangsmåde til produktion af retrovirale emballeringscellelinjer, som producerer en retroviral supernatant med højeffektiv transduktionsgrad

Info

Publication number
DK0871754T3
DK0871754T3 DK96945101.2T DK96945101T DK0871754T3 DK 0871754 T3 DK0871754 T3 DK 0871754T3 DK 96945101 T DK96945101 T DK 96945101T DK 0871754 T3 DK0871754 T3 DK 0871754T3
Authority
DK
Denmark
Prior art keywords
retroviral
producing
supernatant
pct
packaging cell
Prior art date
Application number
DK96945101.2T
Other languages
English (en)
Inventor
Richard J Rigg
Jingyi Chen
Jonathan S Dando
Ivan Plavec
Sean P Forestell
Ernst Bohnlein
Original Assignee
Systemix Inc
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 Systemix Inc filed Critical Systemix Inc
Application granted granted Critical
Publication of DK0871754T3 publication Critical patent/DK0871754T3/da

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/13011—Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/13043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/13011—Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13051—Methods of production or purification of viral material
    • C12N2740/13052—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Hybrid Cells (AREA)
  • Packages (AREA)
  • Immobilizing And Processing Of Enzymes And Microorganisms (AREA)
  • Led Devices (AREA)
DK96945101.2T 1995-12-15 1996-12-13 Fremgangsmåde til produktion af retrovirale emballeringscellelinjer, som producerer en retroviral supernatant med højeffektiv transduktionsgrad DK0871754T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US08/572,959 US5910434A (en) 1995-12-15 1995-12-15 Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
PCT/US1996/020777 WO1997021825A1 (en) 1995-12-15 1996-12-13 Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant

Publications (1)

Publication Number Publication Date
DK0871754T3 true DK0871754T3 (da) 2011-05-30

Family

ID=24290077

Family Applications (1)

Application Number Title Priority Date Filing Date
DK96945101.2T DK0871754T3 (da) 1995-12-15 1996-12-13 Fremgangsmåde til produktion af retrovirale emballeringscellelinjer, som producerer en retroviral supernatant med højeffektiv transduktionsgrad

Country Status (11)

Country Link
US (2) US5910434A (da)
EP (1) EP0871754B1 (da)
JP (1) JP2000501944A (da)
AT (1) ATE498689T1 (da)
AU (1) AU706543B2 (da)
CA (1) CA2238436C (da)
DE (1) DE69638334D1 (da)
DK (1) DK0871754T3 (da)
ES (1) ES2357736T3 (da)
PT (1) PT871754E (da)
WO (1) WO1997021825A1 (da)

Families Citing this family (208)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020173643A1 (en) * 1995-04-14 2002-11-21 Kappes John C. Fusion protein delivery system and uses thereof
AU732703B2 (en) * 1996-11-20 2001-04-26 Crucell Holland B.V. An improved method for the production and purification of adenoviral vectors
EP2325299A3 (en) * 1997-09-05 2011-10-05 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US7033595B1 (en) 1998-08-04 2006-04-25 Purdue Research Foundation Pseudotyped retroviruses and stable cell lines for their production
EP1930418B1 (en) 1998-09-04 2015-04-08 Genzyme Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
PT1109892E (pt) * 1998-09-04 2009-02-17 Targeted Genetics Corp Métodos para produzir preparações de vectores de aav recombinantes libertados, de título elevado, isentas de vírus auxiliares
EP1203016A2 (en) * 1999-08-09 2002-05-08 Incyte Genomics, Inc. Proteases and protease inhibitors
EP1210429A2 (en) * 1999-09-10 2002-06-05 Incyte Genomics, Inc. Apoptosis proteins
US6790614B1 (en) 1999-11-19 2004-09-14 Novartis Ag Selectable cell surface marker genes
EP1282727A1 (en) 2000-02-25 2003-02-12 Biofocus Discovery Limited Methods for identifying candidate polynucleotide molecules encoding a protease
AU2001240019A1 (en) * 2000-03-03 2001-09-17 Incyte Genomics, Inc. G-protein coupled receptors
US6849454B2 (en) 2000-03-07 2005-02-01 St. Jude Children's Research Hospital Highly efficient gene transfer into human repopulating stem cells by RD114 pseudotyped retroviral vector particles
AU2001266975A1 (en) 2000-06-16 2002-01-02 Incyte Genomics, Inc. G-protein coupled receptors
US7608704B2 (en) 2000-11-08 2009-10-27 Incyte Corporation Secreted proteins
US7122182B2 (en) * 2001-05-11 2006-10-17 Wellstat Biologics Corporation Oncolytic virus therapy
WO2003070902A2 (en) * 2002-02-20 2003-08-28 Incyte Corporation Receptors and membrane-associated proteins
AU2003237374A1 (en) * 2002-06-04 2003-12-19 Centers For Disease Control And Prevention Improved pseudotyped retroviruses
US20040009158A1 (en) * 2002-07-11 2004-01-15 Washington University Promotion of neovascularization using bone marrow-derived endothelial-progenitor cells
AU2003265431A1 (en) * 2002-08-13 2004-02-25 Incyte Corporation Cell adhesion and extracellular matrix proteins
US20070219353A1 (en) * 2002-09-03 2007-09-20 Incyte Corporation Immune Response Associated Proteins
AU2003279829A1 (en) * 2002-10-04 2004-05-04 Incyte Corp Protein modification and maintenance molecules
US20050164275A1 (en) * 2002-10-18 2005-07-28 Incyte Corporation Phosphodiesterases
WO2004044166A2 (en) * 2002-11-12 2004-05-27 Incyte Corporation Carbohydrate-associated proteins
WO2004044165A2 (en) * 2002-11-13 2004-05-27 Incyte Corporation Lipid-associated proteins
US20070009516A1 (en) * 2002-11-26 2007-01-11 Tran Uyen K Immune response-associated proteins
WO2004098539A2 (en) 2003-04-30 2004-11-18 Incyte Corporation Kinases and phosphatases
CA2545938A1 (en) * 2003-11-12 2005-05-26 Universite Laval High-titer retroviral packaging cells
TR201900968T4 (tr) 2004-09-24 2019-02-21 Mesoblast Inc Multipotansiyel genleşmiş mezenkimal prekürsör hücre soyu (memp) ve bunların kullanımı.
ES2645012T3 (es) 2004-09-24 2017-12-01 Mesoblast, Inc. Método para potenciar la proliferación y/o supervivencia de las células precursoras mesenquimales (MPC)
US8325826B2 (en) * 2005-03-09 2012-12-04 Qualcomm Incorporated Methods and apparatus for transmitting signals facilitating antenna control
US7826807B2 (en) * 2005-03-09 2010-11-02 Qualcomm Incorporated Methods and apparatus for antenna control in a wireless terminal
EP2399991B1 (en) 2005-04-12 2017-09-27 Mesoblast, Inc. Isolation of adult multipotential cells by tissue non-specific alkaline phosphatase
WO2006130855A2 (en) * 2005-06-01 2006-12-07 California Institute Of Technology Method of targeted gene delivery using viral vectors
US20090227032A1 (en) * 2005-12-13 2009-09-10 Kyoto University Nuclear reprogramming factor and induced pluripotent stem cells
US8129187B2 (en) * 2005-12-13 2012-03-06 Kyoto University Somatic cell reprogramming by retroviral vectors encoding Oct3/4. Klf4, c-Myc and Sox2
US8278104B2 (en) * 2005-12-13 2012-10-02 Kyoto University Induced pluripotent stem cells produced with Oct3/4, Klf4 and Sox2
EP2206724A1 (en) * 2005-12-13 2010-07-14 Kyoto University Nuclear reprogramming factor
WO2007124573A1 (en) * 2006-04-28 2007-11-08 Universite Laval High-titer retroviral packaging cells
US8225186B2 (en) 2006-07-14 2012-07-17 Qualcomm Incorporated Ecoding and decoding methods and apparatus for use in a wireless communication system
US7724853B2 (en) * 2006-07-14 2010-05-25 Qualcomm Incorporated Enabling mobile switched antennas
US7720485B2 (en) * 2006-07-14 2010-05-18 Qualcomm Incorporated Methods and apparatus related to assignment in a wireless communications system
PL2048955T3 (pl) * 2006-07-21 2013-11-29 California Inst Of Techn Nakierowane dostarczanie genu dla szczepienia komórek dendrytycznych
ES2569365T3 (es) 2006-11-29 2016-05-10 Nationwide Children's Hospital Inhibición de miostatina para la potenciación de músculo y/o la mejora de la función muscular
EP2155862A2 (en) 2007-05-04 2010-02-24 Whitehead Institute For Biomedical Research Ex vivo expansion of human hematopoietic stem cells
US9213999B2 (en) * 2007-06-15 2015-12-15 Kyoto University Providing iPSCs to a customer
JP2008307007A (ja) * 2007-06-15 2008-12-25 Bayer Schering Pharma Ag 出生後のヒト組織由来未分化幹細胞から誘導したヒト多能性幹細胞
EP3424514B1 (en) 2007-08-06 2022-05-11 Mesoblast, Inc. Tnap+ mesenchymal precursor cells for use in treating connective tissue diseases
TWI325401B (en) * 2007-12-17 2010-06-01 Duen Gang Mou Vessel structure
EP2268809B1 (en) 2008-05-02 2019-02-06 Kyoto University Method of nuclear reprogramming
JP5826629B2 (ja) 2008-08-18 2015-12-02 メゾブラスト，インコーポレーテッド モノクローナル抗体ｓｔｒｏ−４
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
US11219696B2 (en) 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9
NZ597804A (en) 2009-07-24 2013-10-25 Immune Design Corp Lentiviral vectors pseudotyped with a sindbis virus envelope glycoprotein
CN108744262A (zh) 2010-11-23 2018-11-06 普莱萨格生命科学公司 用于实体递送的治疗方法和组合物
US10196636B2 (en) 2011-04-21 2019-02-05 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of myotilin
ES2702496T3 (es) 2011-04-21 2019-03-01 Nationwide Childrens Hospital Inc Productos de virus recombinante y procedimientos para la inhibición de la expresión de la miotilina
US20130039888A1 (en) 2011-06-08 2013-02-14 Nationwide Children's Hospital Inc. Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders
WO2013016352A1 (en) 2011-07-25 2013-01-31 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of dux4
WO2013078316A1 (en) 2011-11-23 2013-05-30 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
US8323662B1 (en) 2012-03-30 2012-12-04 Immune Design Corp. Methods useful for generating highly mannosylated pseudotyped lentiviral vector particles comprising a Vpx protein
US9713635B2 (en) 2012-03-30 2017-07-25 Immune Design Corp. Materials and methods for producing improved lentiviral vector particles
EP2831095B1 (en) 2012-03-30 2018-11-28 Immune Design Corp. Lentiviral vector particles having improved transduction efficiency for cells expressing dc-sign
JP6314138B2 (ja) 2012-08-01 2018-04-18 ネイションワイド チルドレンズ ホスピタル 組換えアデノ随伴ウイルス９の髄腔内送達
JP6901823B2 (ja) 2012-08-01 2021-07-14 ユナイテッド セラピューティクス コーポレイション 間葉系幹細胞による肺動脈性高血圧症の処置
CA2880811C (en) 2012-08-01 2021-12-07 United Therapeutics Corporation Treatment of pulmonary arterial hypertension with prostacyclin-treated endothelial progenitor cells
JP2015529685A (ja) 2012-09-17 2015-10-08 ザ・リサーチ・インスティテュート・アット・ネイションワイド・チルドレンズ・ホスピタルＴｈｅ Ｒｅｓｅａｒｃｈ Ｉｎｓｔｉｔｕｔｅ Ａｔｎａｔｉｏｎｗｉｄｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ 筋萎縮性側索硬化症の処置のための組成物および方法
ES2963968T3 (es) 2013-01-09 2024-04-03 United Therapeutics Corp Tratamiento de la vasculopatía con prostaciclina y células madre mesenquimatosas
JP6461917B2 (ja) 2013-04-20 2019-01-30 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル エクソン２標的Ｕ７ｓｎＲＮＡポリヌクレオチド構築物の組換えアデノ随伴ウイルスによる送達
WO2014183071A2 (en) 2013-05-10 2014-11-13 Whitehead Institute For Biomedical Research In vitro production of red blood cells with sortaggable proteins
RS63942B1 (sr) 2013-08-27 2023-02-28 Res Inst Nationwide Childrens Hospital Proizvodi i postupci za lečenje amiotrofične lateralne skleroze
EP3065784A4 (en) 2013-11-05 2017-05-10 The Research Institute at Nationwide Children's Hospital COMPOSITIONS AND METHODS FOR INHIBITING NF- kB AND SOD-1 TO TREAT AMYOTROPHIC LATERAL SCLEROSIS
WO2015142984A1 (en) 2014-03-18 2015-09-24 Washington University Methods and compositions for red-shifted chromophore substitution for optogenetic applications
WO2015162302A2 (en) * 2014-04-25 2015-10-29 Genethon Treatment of hyperbilirubinemia
JP6986444B2 (ja) 2014-08-09 2021-12-22 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル Ｄｍｄ遺伝子のエクソン５内の内部リボソーム進入部位を活性化するための方法及び材料
US10842886B2 (en) 2014-10-10 2020-11-24 Research Institute At Nationwide Children's Hospital Guided injections for AAV gene transfer to muscle
CA2966884A1 (en) 2014-11-05 2016-05-12 Research Institute At Nationwide Children's Hospital Methods and materials for producing recombinant viruses in eukaryotic microalgae
MA41451A (fr) 2015-02-04 2017-12-12 Univ Washington Constructions anti-tau
SG11201706767RA (en) 2015-02-23 2017-09-28 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
US10755078B2 (en) * 2015-04-10 2020-08-25 President And Fellows Of Harvard College Methods and devices for live cell imaging analysis
US10017832B2 (en) 2015-08-25 2018-07-10 Washington University Compositions and methods for site specific recombination at asymmetric sites
CA2998636A1 (en) 2015-09-17 2017-03-23 Research Institute At Nationwide Children's Hospital Methods and materials for galgt2 gene therapy
JP2018532402A (ja) 2015-09-24 2018-11-08 クリスパー セラピューティクス アーゲー Ｒｎａプログラム可能エンドヌクレアーゼの新規のファミリーならびにゲノム編集および他の適用におけるそれらの使用
JP2019507579A (ja) 2015-10-28 2019-03-22 クリスパー セラピューティクス アーゲー デュシェンヌ型筋ジストロフィーの処置のための材料および方法
EP3371305A1 (en) 2015-11-06 2018-09-12 Crispr Therapeutics AG Materials and methods for treatment of glycogen storage disease type 1a
JP7418957B2 (ja) 2015-11-16 2024-01-22 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル タイチン系ミオパチー及び他のタイチノパチーの治療のための材料及び方法
CA3006618A1 (en) 2015-12-01 2017-06-08 Crispr Therapeutics Ag Materials and methods for treatment of alpha-1 antitrypsin deficiency
AU2016370590B2 (en) 2015-12-14 2023-11-02 The Trustees Of The University Of Pennsylvania Composition for treatment of Crigler-Najjar syndrome
BR112018012894A2 (pt) 2015-12-23 2018-12-04 Crispr Therapeutics Ag materiais e métodos para tratamento de esclerose lateral amiotrófica e/ou degeneração lobular frontotemporal
US11938193B2 (en) 2016-01-08 2024-03-26 Washington University Compositions comprising chemerin and methods of use thereof
WO2017134529A1 (en) 2016-02-02 2017-08-10 Crispr Therapeutics Ag Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
EP3416689B1 (en) 2016-02-18 2023-01-18 CRISPR Therapeutics AG Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
WO2017147509A1 (en) 2016-02-25 2017-08-31 Marco Colonna Compositions comprising trem2 and methods of use thereof
EP3420109A4 (en) 2016-02-26 2019-11-06 Research Institute at Nationwide Children's Hospital RECOMBINANT VIRUS PRODUCTS AND METHOD OF INDUCING DUX4 EXON SKIPPING
EP3429632B1 (en) 2016-03-16 2023-01-04 CRISPR Therapeutics AG Materials and methods for treatment of hereditary haemochromatosis
CN109714954A (zh) 2016-04-02 2019-05-03 全国儿童医院研究所 用于组织特异性表达的修饰的u6启动子系统
RS65490B1 (sr) 2016-04-15 2024-05-31 Res Inst Nationwide Childrens Hospital Vektor adeno-asociranog virusa za isporuku β-sarkoglikana i mikrorna-29 i tretman mišićne distrofije
MA45477A (fr) 2016-04-15 2019-02-20 Res Inst Nationwide Childrens Hospital Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire
BR112018071321A2 (pt) 2016-04-18 2019-02-26 Crispr Therapeutics Ag materiais e métodos para tratamento de hemoglobinopatias
WO2017191503A1 (en) 2016-05-05 2017-11-09 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
EP3478313B1 (en) 2016-06-29 2022-05-04 CRISPR Therapeutics AG Materials and methods for treatment of amyotrophic lateral sclerosis (als) and other related disorders
WO2018002783A1 (en) 2016-06-29 2018-01-04 Crispr Therapeutics Ag Materials and methods for treatment of friedreich ataxia and other related disorders
WO2018002812A1 (en) 2016-06-29 2018-01-04 Crispr Therapeutics Ag Materials and methods for treatment of myotonic dystrophy type 1 (dm1) and other related disorders
JP7305534B2 (ja) 2016-07-06 2023-07-10 バーテックス ファーマシューティカルズ インコーポレイテッド 疼痛関連障害を処置するための物質及び方法
JP2019520079A (ja) 2016-07-06 2019-07-18 クリスパー セラピューティクス アクチェンゲゼルシャフト 疼痛関連障害を処置するための物質及び方法
WO2018007871A1 (en) 2016-07-08 2018-01-11 Crispr Therapeutics Ag Materials and methods for treatment of transthyretin amyloidosis
WO2018020323A2 (en) 2016-07-25 2018-02-01 Crispr Therapeutics Ag Materials and methods for treatment of fatty acid disorders
CA3041514A1 (en) 2016-10-24 2018-05-03 United Therapeutics Corporation Enhancement of msc immunomodulatory properties by treprostinil
JP7106534B2 (ja) 2016-11-17 2022-07-26 ネイションワイド チルドレンズ ホスピタル， インコーポレイテッド メチル－ｃｐｇ結合タンパク質２をコードする組換えアデノ随伴ウイルスの髄腔内送達
CN110191955B (zh) 2016-12-13 2024-05-31 西雅图儿童医院（Dba西雅图儿童研究所） 在体外和体内对工程化的细胞中表达的化学物质诱导的信号传导复合物进行外源性药物激活的方法
EP3585899A1 (en) 2017-02-22 2020-01-01 CRISPR Therapeutics AG Materials and methods for treatment of primary hyperoxaluria type 1 (ph1) and other alanine-glyoxylate aminotransferase (agxt) gene related conditions or disorders
WO2018154439A1 (en) 2017-02-22 2018-08-30 Crispr Therapeutics Ag Materials and methods for treatment of spinocerebellar ataxia type 1 (sca1) and other spinocerebellar ataxia type 1 protein (atxn1) gene related conditions or disorders
CN110662838B (zh) 2017-02-22 2024-05-28 克里斯珀医疗股份公司 用于基因编辑的组合物和方法
EP3585900B1 (en) 2017-02-22 2022-12-21 CRISPR Therapeutics AG Materials and methods for treatment of spinocerebellar ataxia type 2 (sca2) and other spinocerebellar ataxia type 2 protein (atxn2) gene related conditions or disorders
WO2018154418A1 (en) 2017-02-22 2018-08-30 Crispr Therapeutics Ag Materials and methods for treatment of early onset parkinson's disease (park1) and other synuclein, alpha (snca) gene related conditions or disorders
MA47800A (fr) 2017-03-17 2020-01-22 Univ Newcastle Délivrance par vecteur viral adéno-associé d'un fragment de micro-dystrophine pour traiter la dystrophie musculaire
MA52112B1 (fr) 2017-03-17 2023-08-31 Res Inst Nationwide Childrens Hospital Administration par vecteur à virus adéno-associé de micro-dystrophine spécifique du muscle pour traiter la dystrophie musculaire
US11622977B2 (en) 2017-05-12 2023-04-11 Crispr Therapeutics Ag Materials and methods for engineering cells and uses thereof in immuno-oncology
CN111065741A (zh) 2017-07-08 2020-04-24 吉尼松公司 脊髓性肌萎缩症的治疗
SG11202003464VA (en) 2017-10-17 2020-05-28 Crispr Therapeutics Ag Compositions and methods for gene editing for hemophilia a
JP7213238B2 (ja) 2017-10-18 2023-01-26 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 筋ジストロフィーを治療するための筋特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達
EP3697916A4 (en) 2017-10-20 2021-08-11 Research Institute at Nationwide Children's Hospital METHODS AND MATERIALS FOR NT-3 GENE THERAPY
MA50849A (fr) 2017-10-26 2020-09-02 Vertex Pharma Substances et procédés pour le traitement d'hémoglobinopathies
SG11202004406VA (en) 2017-11-08 2020-06-29 Avexis Inc Means and method for preparing viral vectors and uses of same
US20210363521A1 (en) 2017-11-09 2021-11-25 Vertex Pharmaceuticals Incorporated CRISPR/CAS Systems For Treatment of DMD
CN111727251A (zh) 2017-11-21 2020-09-29 克里斯珀医疗股份公司 用于治疗常染色体显性色素性视网膜炎的材料和方法
US11578323B2 (en) 2017-12-14 2023-02-14 Bayer Healthcare Llc RNA-programmable endonuclease systems and their use in genome editing and other applications
EP3728594A1 (en) 2017-12-21 2020-10-28 CRISPR Therapeutics AG Materials and methods for treatment of usher syndrome type 2a
EP3728595A1 (en) 2017-12-21 2020-10-28 CRISPR Therapeutics AG Materials and methods for treatment of usher syndrome type 2a and/or non-syndromic autosomal recessive retinitis pigmentosa (arrp)
WO2019140330A1 (en) 2018-01-12 2019-07-18 Casebia Therapeutics Limited Liability Partnership Compositions and methods for gene editing by targeting transferrin
US11268077B2 (en) 2018-02-05 2022-03-08 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
MA51787A (fr) 2018-02-05 2020-12-16 Vertex Pharma Substances et méthodes de traitement d'hémoglobinopathies
MA51869A (fr) 2018-02-16 2020-12-23 Bayer Healthcare Llc Compositions et méthodes pour l'édition génique par ciblage du fibrinogène-alpha
US20210054353A1 (en) 2018-03-19 2021-02-25 Crispr Therapeutics Ag Novel rna-programmable endonuclease systems and uses thereof
WO2019204668A1 (en) 2018-04-18 2019-10-24 Casebia Therapeutics Limited Liability Partnership Compositions and methods for knockdown of apo(a) by gene editing for treatment of cardiovascular disease
SG11202007874XA (en) 2018-04-27 2020-09-29 Seattle Childrens Hospital Dba Seattle Childrens Res Inst Rapamycin resistant cells
JP7478675B2 (ja) 2018-06-08 2024-05-07 ノバルティス アーゲー 薬物製品の効力を測定するための細胞ベースアッセイ
GB201809588D0 (en) 2018-06-12 2018-07-25 Univ Bristol Materials and methods for modulating intraocular and intracranial pressure
BR112020025995A2 (pt) 2018-06-18 2021-03-23 Research Institute At Nationwide Children's Hospital administração de microdistrofina músculo-específica por vetor de vírus adeno-associado para tratar a distrofia muscular
CA3104471A1 (en) 2018-06-18 2019-12-26 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies
AU2019295779A1 (en) 2018-06-29 2021-01-28 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2A
US20210324417A1 (en) 2018-08-29 2021-10-21 Research Institute At Nationwide Children's Hospital Products and methods for inhibition of expression of mutant gars protein
BR112021007403A2 (pt) 2018-10-17 2021-12-07 Bayer Healthcare Llc Composições e métodos para entrega de transgenes
MX2021006359A (es) 2018-11-30 2021-08-11 Novartis Ag Vectores virales de vaa y sus usos.
CA3122319A1 (en) 2018-12-21 2020-06-25 Genethon Expression cassettes for gene therapy vectors
AU2019419494A1 (en) 2018-12-31 2021-07-15 Research Institute At Nationwide Children's Hospital DUX4 RNA silencing using RNA targeting CRISPR-Cas13b
CA3127801A1 (en) 2019-02-04 2020-08-13 Research Institute At Nationwide Children's Hospital Adeno-associated virus delivery of cln6 polynucleotide
SG11202107986YA (en) 2019-02-04 2021-08-30 Res Inst Nationwide Childrens Hospital Adeno-associated virus delivery of cln3 polynucleotide
WO2020168362A1 (en) 2019-02-15 2020-08-20 Crispr Therapeutics Ag Gene editing for hemophilia a with improved factor viii expression
EP3930765A1 (en) 2019-02-26 2022-01-05 Research Institute at Nationwide Children's Hospital Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy
EP3937963A2 (en) 2019-03-12 2022-01-19 CRISPR Therapeutics AG Novel high fidelity rna-programmable endonuclease systems and uses thereof
EP3955969A1 (en) 2019-04-15 2022-02-23 Sanford Research Gene therapy for treating or preventing visual effects in batten disease
US20210047649A1 (en) 2019-05-08 2021-02-18 Vertex Pharmaceuticals Incorporated Crispr/cas all-in-two vector systems for treatment of dmd
CA3141017A1 (en) 2019-05-17 2020-11-26 Research Institute At Nationwide Children's Hospital Improved delivery of gene therapy vectors to retinal cells using a glycoside hydrolase enzyme
EP3990636A1 (en) 2019-06-28 2022-05-04 CRISPR Therapeutics AG Materials and methods for controlling gene editing
KR20220035937A (ko) 2019-07-25 2022-03-22 노파르티스 아게 조절 가능한 발현 시스템
CA3150964A1 (en) 2019-08-21 2021-02-25 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy
US20220378945A1 (en) 2019-10-18 2022-12-01 Research Institute At Nationwide Children's Hospital Gene therapy targeting cochlear cells
JP2022552014A (ja) 2019-10-18 2022-12-14 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル Ｉｒｆ２ｂｐｌ遺伝子の変異に関連する障害の治療のための材料および方法
EP4061831A1 (en) 2019-11-22 2022-09-28 Research Institute at Nationwide Children's Hospital Materials and methods for treatment of disorders associated with the ighmbp2 gene
AU2020408225A1 (en) 2019-12-20 2022-07-14 Research Institute At Nationwide Children's Hospital Optimized gene therapy for targeting muscle in muscle diseases
CA3171959A1 (en) 2020-02-18 2021-08-26 Research Institute At Nationwide Children's Hospital Aav-mediated targeting of mirna in the treatment of x-linked disorders
CA3174863A1 (en) 2020-04-14 2021-10-21 Ana BUJ BELLO Vectors for the treatment of acid ceramidase deficiency
AU2021293197A1 (en) 2020-06-15 2023-02-09 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery for muscular dystrophies
WO2022018638A1 (en) 2020-07-21 2022-01-27 Crispr Therapeutics Ag Genome-editing compositions and methods to modulate faah for treatment of neurological disorders
TW202227634A (zh) 2020-09-08 2022-07-16 美商薩羅塔治療公司 表現γ—肌聚醣之腺相關病毒載體之全身性遞送及肌肉失養症之治療
WO2022060841A2 (en) 2020-09-15 2022-03-24 Research Institute At Nationwide Children's Hospital Aav-mediated homology-independent targeted integration gene editing for correction of diverse dmd mutations in patients with muscular dystrophy
CA3194122A1 (en) 2020-09-28 2022-03-31 Research Institute At Nationwide Children's Hospital Products and methods for treating muscular dystrophy
WO2022070107A1 (en) 2020-09-30 2022-04-07 Crispr Therapeutics Ag Materials and methods for treatment of amyotrophic lateral sclerosis
IL303230A (en) 2020-11-30 2023-07-01 Res Inst Nationwide Childrens Hospital Preparations and methods for the treatment of facial-shoulder-arm muscle atrophy
WO2022133246A1 (en) 2020-12-17 2022-06-23 Vertex Pharmaceuticals Incorporated Compositions and methods for editing beta-globin for treatment of hemaglobinopathies
EP4284413A1 (en) 2021-01-27 2023-12-06 Research Institute at Nationwide Children's Hospital Materials and methods for the treatment of lysosomal acid lipase deficiency (lal-d)
JP2024505575A (ja) 2021-02-03 2024-02-06 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル Ｄｕｘ４過剰発現に関連する疾患を治療するための組成物及び方法
WO2022170038A1 (en) 2021-02-05 2022-08-11 Amicus Therapeutics, Inc. Adeno-associated virus delivery of cln3 polynucleotide
EP4301462A1 (en) 2021-03-04 2024-01-10 Research Institute at Nationwide Children's Hospital Products and methods for treatment of dystrophin-based myopathies using crispr-cas9 to correct dmd exon duplications
CN115427561B (zh) 2021-03-09 2024-06-04 辉大(上海)生物科技有限公司 工程化CRISPR/Cas13系统及其用途
JP2024515623A (ja) 2021-04-13 2024-04-10 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 髄腔内送達によってピット・ホプキンス症候群を治療するためのメチル－ｃｐｇ結合タンパク質２をコードする組換えアデノ随伴ウイルス
AU2022262420A1 (en) 2021-04-23 2023-11-16 Research Institute At Nationwide Children's Hospital Products and methods for treating muscular dystrophy
WO2022234295A1 (en) 2021-05-07 2022-11-10 Ucl Business Ltd Abca4 genome editing
WO2022245675A1 (en) 2021-05-17 2022-11-24 Sarepta Therapeutics, Inc. Production of recombinant aav vectors for treating muscular dystrophy
EP4108263A3 (en) 2021-06-02 2023-03-22 Research Institute at Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
EP4101928A1 (en) 2021-06-11 2022-12-14 Bayer AG Type v rna programmable endonuclease systems
AU2022290382A1 (en) 2021-06-11 2023-11-23 Bayer Aktiengesellschaft Type v rna programmable endonuclease systems
WO2023283962A1 (en) 2021-07-16 2023-01-19 Huigene Therapeutics Co., Ltd. Modified aav capsid for gene therapy and methods thereof
CN118159656A (zh) 2021-08-11 2024-06-07 坚固生物科技公司 肌营养不良的治疗
EP4144841A1 (en) 2021-09-07 2023-03-08 Bayer AG Novel small rna programmable endonuclease systems with impoved pam specificity and uses thereof
WO2023042104A1 (en) 2021-09-16 2023-03-23 Novartis Ag Novel transcription factors
AU2022360382A1 (en) 2021-10-07 2024-05-09 Research Institute At Nationwide Children's Hospital Products and methods for myelin protein zero silencing and treating cmt1b disease
WO2023060233A1 (en) 2021-10-08 2023-04-13 Amicus Therapeutics, Inc. Biomarkers for lysosomal storage diseases
JP2023059858A (ja) 2021-10-15 2023-04-27 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 自己相補的アデノ随伴ウイルスベクター及び筋ジストロフィーの治療におけるその使用
US20230279431A1 (en) 2021-11-30 2023-09-07 Research Institute At Nationwide Children's Hospital Self-Complementary Adeno-Associated Virus Vector and its Use in Treatment of Muscular Dystrophy
EP4198134A1 (en) 2021-12-16 2023-06-21 Genethon Gamma-sarcoglycan gene transfer increase using modified itr sequences
EP4198048A1 (en) 2021-12-16 2023-06-21 Genethon Calpain-3 gene transfer increase using modified itr sequences
EP4198046A1 (en) 2021-12-16 2023-06-21 Genethon Alpha-sarcoglycan gene transfer increase using modified itr sequences
EP4198047A1 (en) 2021-12-16 2023-06-21 Genethon Fukutin related protein gene transfer increase using modified itr sequences
AU2022420596A1 (en) 2021-12-21 2024-07-11 Research Institute At Nationwide Children's Hospital Materials and methods for the treatment of limb girdle muscular dystrophy
WO2023118068A1 (en) 2021-12-23 2023-06-29 Bayer Aktiengesellschaft Novel small type v rna programmable endonuclease systems
WO2023168400A2 (en) 2022-03-03 2023-09-07 Research Institute At Nationwide Children's Hospital Materials and methods for the treatment of eif2b5 mutations and diseases resulting therefrom
WO2023196818A1 (en) 2022-04-04 2023-10-12 The Regents Of The University Of California Genetic complementation compositions and methods
WO2023214346A1 (en) 2022-05-06 2023-11-09 Novartis Ag Novel recombinant aav vp2 fusion polypeptides
WO2023240177A1 (en) 2022-06-08 2023-12-14 Research Instiitute At Nationwide Children's Hospital Products and methods for treating diseases or conditions associated with mutant or pathogenic kcnq3 expression
WO2023237587A1 (en) 2022-06-10 2023-12-14 Bayer Aktiengesellschaft Novel small type v rna programmable endonuclease systems
WO2024011115A1 (en) 2022-07-06 2024-01-11 Research Institute At Nationwide Children's Hospital Adeno-associated virus delivery of cln1 polynucleotide
WO2024035782A1 (en) 2022-08-10 2024-02-15 Aav Gene Therapeutics, Inc. Aav-mediated intramuscular delivery of insulin
WO2024081706A1 (en) 2022-10-11 2024-04-18 Research Institute At Nationwide Children's Hospital Adeno-associated virus delivery to treat spinal muscular atrophy with respiratory distress type 1 (smard1) and charcot-marie-tooth type 2s (cmt2s)
WO2024092126A1 (en) 2022-10-27 2024-05-02 Cargo Therapeutics, Inc. Compositions and methods for improved immunotherapies
WO2024129743A2 (en) 2022-12-13 2024-06-20 Bluerock Therapeutics Lp Engineered type v rna programmable endonucleases and their uses

Family Cites Families (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4754065A (en) * 1984-12-18 1988-06-28 Cetus Corporation Precursor to nucleic acid probe
US4683202A (en) * 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4683195A (en) * 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US4800159A (en) * 1986-02-07 1989-01-24 Cetus Corporation Process for amplifying, detecting, and/or cloning nucleic acid sequences
US5278056A (en) * 1988-02-05 1994-01-11 The Trustees Of Columbia University In The City Of New York Retroviral packaging cell lines and process of using same
AU3069189A (en) * 1988-02-05 1989-08-25 Trustees Of Columbia University In The City Of New York, The Retroviral packaging cell lines and processes of using same
US5591624A (en) * 1988-03-21 1997-01-07 Chiron Viagene, Inc. Retroviral packaging cell lines
US5716826A (en) * 1988-03-21 1998-02-10 Chiron Viagene, Inc. Recombinant retroviruses
EP0432216A1 (en) * 1988-09-01 1991-06-19 Whitehead Institute For Biomedical Research Recombinant retroviruses with amphotropic and ecotropic host ranges
US5061620A (en) * 1990-03-30 1991-10-29 Systemix, Inc. Human hematopoietic stem cell
WO1992005266A2 (en) * 1990-09-21 1992-04-02 Viagene, Inc. Packaging cells
AU650085B2 (en) * 1990-11-13 1994-06-09 Immunex Corporation Bifunctional selectable fusion genes
CA2104396C (en) * 1991-02-19 2000-10-10 Jiing-Kuan Yee Viral particles having altered host range
EP0689601B1 (en) * 1993-02-22 2006-10-04 The Rockefeller University Production of high titer helper-free retroviruses by transient transfection
WO1994028143A1 (en) * 1993-05-21 1994-12-08 Targeted Genetics Corporation Bifunctional selectable fusion genes based on the cytosine deaminase (cd) gene
US5378605A (en) 1993-06-08 1995-01-03 Thomas Jefferson University Method of detecting hepatitis B variants having deletions within the X region of the virus genome
US5834256A (en) * 1993-06-11 1998-11-10 Cell Genesys, Inc. Method for production of high titer virus and high efficiency retroviral mediated transduction of mammalian cells
US5498537A (en) * 1994-03-09 1996-03-12 Cellco, Inc. Serum-free production of packaged viral vector
CA2196208A1 (en) * 1994-08-17 1996-02-22 Michael Pensiero Retroviral vectors produced by producer cell lines resistant to lysis by human serum

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