JP2020500162A5 - - Google Patents

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JP2020500162A5
JP2020500162A5 JP2019520884A JP2019520884A JP2020500162A5 JP 2020500162 A5 JP2020500162 A5 JP 2020500162A5 JP 2019520884 A JP2019520884 A JP 2019520884A JP 2019520884 A JP2019520884 A JP 2019520884A JP 2020500162 A5 JP2020500162 A5 JP 2020500162A5
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cell
transgene
gal
protein
composition
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JP7042263B2 (ja
JP2020500162A (ja
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JP2019520884A 2016-10-20 2017-10-19 ファブリー病の治療のための方法および組成物 Active JP7042263B2 (ja)

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JP2022039062A JP7418485B2 (ja) 2016-10-20 2022-03-14 ファブリー病の治療のための方法および組成物

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US201662410543P 2016-10-20 2016-10-20
US62/410,543 2016-10-20
US201762444093P 2017-01-09 2017-01-09
US62/444,093 2017-01-09
US201762458324P 2017-02-13 2017-02-13
US62/458,324 2017-02-13
US201762502058P 2017-05-05 2017-05-05
US62/502,058 2017-05-05
US201762516373P 2017-06-07 2017-06-07
US62/516,373 2017-06-07
US201762552792P 2017-08-31 2017-08-31
US62/552,792 2017-08-31
PCT/US2017/057328 WO2018075736A1 (en) 2016-10-20 2017-10-19 Methods and compositions for the treatment of fabry disease

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JP2020500162A5 true JP2020500162A5 (enExample) 2020-10-08
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US (2) US11219695B2 (enExample)
EP (2) EP4667565A2 (enExample)
JP (2) JP7042263B2 (enExample)
KR (1) KR102712926B1 (enExample)
CN (1) CN110022904B (enExample)
AU (1) AU2017345430B2 (enExample)
BR (1) BR112019007210A2 (enExample)
CA (1) CA3039673A1 (enExample)
ES (1) ES3047063T3 (enExample)
IL (1) IL266047B2 (enExample)
MX (1) MX2019004487A (enExample)
WO (1) WO2018075736A1 (enExample)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7042263B2 (ja) 2016-10-20 2022-03-25 サンガモ セラピューティクス, インコーポレイテッド ファブリー病の治療のための方法および組成物
US20220184185A1 (en) * 2018-07-25 2022-06-16 Modernatx, Inc. Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders
EP3867380A2 (en) 2018-10-18 2021-08-25 Intellia Therapeutics, Inc. Compositions and methods for expressing factor ix
MX2021004278A (es) * 2018-10-18 2021-09-08 Intellia Therapeutics Inc Composiciones y metodos para la expresion de transgenes a partir de un locus de albumina.
CN113423419A (zh) * 2019-01-04 2021-09-21 桑格摩生物治疗股份有限公司 用于治疗法布里病的方法和组合物
SG11202108502UA (en) * 2019-02-04 2021-09-29 Freeline Therapeutics Ltd Polynucleotides
US20220267794A1 (en) * 2019-03-27 2022-08-25 Sigilon Therapeutics, Inc. Compositions, devices, and methods for treating fabry disease
AU2020289581B2 (en) 2019-06-07 2024-11-21 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized albumin locus
CN113058041B (zh) * 2020-08-27 2022-04-05 华东师范大学 一种用于治疗庞贝氏病的产品
WO2021183895A1 (en) * 2020-03-13 2021-09-16 Biomarin Pharmaceutical Inc. Treatment of fabry disease with aav gene therapy vectors
KR102438536B1 (ko) * 2020-04-28 2022-09-05 케이비바이오메드 주식회사 경구용 핵산 분자 전달 복합체
EP4181671A4 (en) * 2020-07-15 2024-08-28 The Jackson Laboratory Humanized mouse models for sars-cov-2 infection
KR20230131246A (ko) * 2021-01-14 2023-09-12 스파크 테라퓨틱스, 인코포레이티드 파브리병을 치료하기 위한 조성물 및 방법
KR20240099332A (ko) * 2021-11-03 2024-06-28 상가모 테라퓨틱스, 인코포레이티드 파브리병의 치료를 위한 바이러스 벡터 작제물의 사용 방법

Family Cites Families (141)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4217344A (en) 1976-06-23 1980-08-12 L'oreal Compositions containing aqueous dispersions of lipid spheres
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4186183A (en) 1978-03-29 1980-01-29 The United States Of America As Represented By The Secretary Of The Army Liposome carriers in chemotherapy of leishmaniasis
US4261975A (en) 1979-09-19 1981-04-14 Merck & Co., Inc. Viral liposome particle
US4485054A (en) 1982-10-04 1984-11-27 Lipoderm Pharmaceuticals Limited Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV)
US4501728A (en) 1983-01-06 1985-02-26 Technology Unlimited, Inc. Masking of liposomes from RES recognition
US4946787A (en) 1985-01-07 1990-08-07 Syntex (U.S.A.) Inc. N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
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
US4774085A (en) 1985-07-09 1988-09-27 501 Board of Regents, Univ. of Texas Pharmaceutical administration systems containing a mixture of immunomodulators
US5422251A (en) 1986-11-26 1995-06-06 Princeton University Triple-stranded nucleic acids
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5176996A (en) 1988-12-20 1993-01-05 Baylor College Of Medicine Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
AU7979491A (en) 1990-05-03 1991-11-27 Vical, Inc. Intracellular delivery of biologically active substances by means of self-assembling lipid complexes
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5420032A (en) 1991-12-23 1995-05-30 Universitge Laval Homing endonuclease which originates from chlamydomonas eugametos and recognizes and cleaves a 15, 17 or 19 degenerate double stranded nucleotide sequence
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
US5487994A (en) 1992-04-03 1996-01-30 The Johns Hopkins University Insertion and deletion mutants of FokI restriction endonuclease
US5356802A (en) 1992-04-03 1994-10-18 The Johns Hopkins University Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease
US5792632A (en) 1992-05-05 1998-08-11 Institut Pasteur Nucleotide sequence encoding the enzyme I-SceI and the uses thereof
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US6242568B1 (en) 1994-01-18 2001-06-05 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
CA2181548C (en) 1994-01-18 2009-11-03 Carlos F. Barbas, Iii Zinc finger protein derivatives and methods therefor
JP4285766B2 (ja) 1994-03-23 2009-06-24 オハイオ ユニバーシティ 緻密にした核酸および細胞へのデリバリ
US5585245A (en) 1994-04-22 1996-12-17 California Institute Of Technology Ubiquitin-based split protein sensor
DE69535829D1 (de) 1994-08-20 2008-10-16 Gendaq Ltd Verbesserung in bezug auf bindungsproteine bei der erkennung von dna
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
US6544761B2 (en) * 1994-12-13 2003-04-08 Human Genome Sciences, Inc. Human tissue inhibitor of metalloproteinase-4
US5789538A (en) 1995-02-03 1998-08-04 Massachusetts Institute Of Technology Zinc finger proteins with high affinity new DNA binding specificities
US5925523A (en) 1996-08-23 1999-07-20 President & Fellows Of Harvard College Intraction trap assay, reagents and uses thereof
US6458574B1 (en) * 1996-09-12 2002-10-01 Transkaryotic Therapies, Inc. Treatment of a α-galactosidase a deficiency
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
US6342345B1 (en) 1997-04-02 2002-01-29 The Board Of Trustees Of The Leland Stanford Junior University Detection of molecular interactions by reporter subunit complementation
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
WO2000009153A1 (en) * 1997-10-29 2000-02-24 Genzyme Corporation Compositions and methods for treating lysosomal storage disease
US6410248B1 (en) 1998-01-30 2002-06-25 Massachusetts Institute Of Technology General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites
ES2341926T3 (es) 1998-03-02 2010-06-29 Massachusetts Institute Of Technology Poliproteinas con dedos de cinc que tienen enlazadores mejorados.
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
US7030215B2 (en) 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
CA2394850C (en) 1999-12-06 2012-02-07 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
EP1254369B1 (en) 2000-02-08 2010-10-06 Sangamo BioSciences, Inc. Cells for drug discovery
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
AU2001263155A1 (en) 2000-05-16 2001-11-26 Massachusetts Institute Of Technology Methods and compositions for interaction trap assays
US20030073652A1 (en) * 2000-05-17 2003-04-17 Harvey Pollard Ex-vivo and in vivo factor XII gene therapy for hemophilia A and B
AU2001271614B2 (en) 2000-07-03 2007-05-31 Catalent Pharma Solutions, Llc Host cells containing multiple integrating vectors
MXPA03000765A (es) 2000-07-24 2004-05-21 Gendel Ltd Suministro de polipeptido il-2.
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
GB0024550D0 (enExample) 2000-10-06 2000-11-22 Oxford Biomedica Ltd
US7419829B2 (en) 2000-10-06 2008-09-02 Oxford Biomedica (Uk) Limited Vector system
AU2002215389A1 (en) 2000-10-11 2002-08-28 Transkaryotic Therapies, Inc. Optimized messenger rna
US7067317B2 (en) 2000-12-07 2006-06-27 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
KR100750929B1 (ko) 2001-07-10 2007-08-22 삼성전자주식회사 색 보정 기능을 갖는 액정 표시 장치 및 이의 구동 장치및 그 방법
WO2003016496A2 (en) 2001-08-20 2003-02-27 The Scripps Research Institute Zinc finger binding domains for cnn
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
ATE347593T1 (de) 2002-01-23 2006-12-15 Univ Utah Res Found Zielgerichtete chromosomale mutagenese mit zinkfingernukleasen
EP1504092B2 (en) 2002-03-21 2014-06-25 Sangamo BioSciences, Inc. Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
US7074596B2 (en) 2002-03-25 2006-07-11 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Synthesis and use of anti-reverse mRNA cap analogues
US7361635B2 (en) 2002-08-29 2008-04-22 Sangamo Biosciences, Inc. Simultaneous modulation of multiple genes
WO2004037977A2 (en) 2002-09-05 2004-05-06 California Institute Of Thechnology Use of chimeric nucleases to stimulate gene targeting
US7446098B2 (en) * 2003-02-18 2008-11-04 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiencies
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US7253273B2 (en) 2004-04-08 2007-08-07 Sangamo Biosciences, Inc. Treatment of neuropathic pain with zinc finger proteins
JP4903689B2 (ja) 2004-04-08 2012-03-28 サンガモ バイオサイエンシズ インコーポレイテッド 神経障害および神経変性症状を治療するための方法と組成物
AU2005287278B2 (en) 2004-09-16 2011-08-04 Sangamo Biosciences, Inc. Compositions and methods for protein production
US8008468B2 (en) 2005-02-16 2011-08-30 Benitec, Inc. RNAi expression constructs with liver-specific enhancer/promoter
WO2006121866A2 (en) 2005-05-05 2006-11-16 The Arizona Board Of Regents On Behalf Of The University Of Arizona Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences
US20090263900A1 (en) 2008-04-14 2009-10-22 Sangamo Biosciences, Inc. Linear donor constructs for targeted integration
EP1913149A4 (en) 2005-07-26 2009-08-05 Sangamo Biosciences Inc TARGETED INTEGRATION AND EXPRESSION OF EXOGENOUS NUCLEIC ACID SEQUENCES
CA2626262C (en) 2005-10-18 2015-09-08 Homme W. Hellinga Rationally-designed meganucleases with altered sequence specificity and dna-binding affinity
BRPI0618583A2 (pt) * 2005-11-18 2011-09-06 Tokyo Metropolitan Org Med Res enzima altamente funcional tendo especifidade ao substrato modificada
EP2019839B1 (en) 2006-05-25 2011-12-07 Sangamo BioSciences, Inc. Methods and compositions for gene inactivation
DE602007005634D1 (de) 2006-05-25 2010-05-12 Sangamo Biosciences Inc Variante foki-spaltungshälften-domänen
US8110379B2 (en) 2007-04-26 2012-02-07 Sangamo Biosciences, Inc. Targeted integration into the PPP1R12C locus
PT3252161T (pt) 2007-06-06 2022-02-01 Genzyme Corp Terapia genética para doenças do armazenamento lisossomal
DK2167523T3 (da) 2007-06-19 2014-09-08 Univ Louisiana State Syntese og anvendelse af anti-reverse phosphorothioatanaloger af messenger-rna-cappen
US8790345B2 (en) 2007-08-21 2014-07-29 Zimmer, Inc. Titanium alloy with oxidized zirconium for a prosthetic implant
US8563314B2 (en) 2007-09-27 2013-10-22 Sangamo Biosciences, Inc. Methods and compositions for modulating PD1
SI2252313T1 (sl) * 2008-02-12 2015-08-31 Amicus Therapeutics, Inc. Postopek napovedovanja odgovora na zdravljenje bolezni s farmakološkim spremljevalcem
DE102008025277A1 (de) 2008-05-27 2009-12-03 Merck Patent Gmbh Glaszusammensetzung
SG191561A1 (en) 2008-08-22 2013-07-31 Sangamo Biosciences Inc Methods and compositions for targeted single-stranded cleavage and targeted integration
JP5681114B2 (ja) 2008-12-04 2015-03-04 サンガモ バイオサイエンシーズ, インコーポレイテッド 亜鉛フィンガーヌクレアーゼを使用したラットのゲノム編集
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
BR112012002291A2 (pt) 2009-07-31 2016-11-29 Ethris Gmbh "polirribonucleotídeo com uma sequência que codifica uma proteína ou fragmento de proteína, implante, e, processo para a seleção de sequências de nucleotídeos"
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
DK2534173T3 (da) 2010-02-08 2019-10-14 Sangamo Therapeutics Inc Manipulerede spaltnings-halvdomæner
EP2534163B1 (en) 2010-02-09 2015-11-04 Sangamo BioSciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
JP5437895B2 (ja) 2010-04-20 2014-03-12 株式会社ジャパンディスプレイ 表示装置及びその製造方法
KR101880536B1 (ko) 2010-04-26 2018-07-23 상가모 테라퓨틱스, 인코포레이티드 아연 핑거 뉴클레아제를 사용하는 로사 좌위의 게놈 편집
KR101974036B1 (ko) 2010-05-03 2019-04-30 상가모 테라퓨틱스, 인코포레이티드 아연 핑거 모듈을 연결하기 위한 조성물
JP6208580B2 (ja) 2010-05-17 2017-10-04 サンガモ セラピューティクス, インコーポレイテッド 新規のdna結合タンパク質及びその使用
CA2993567C (en) 2010-07-21 2022-06-28 Sangamo Biosciences, Inc. Methods and compositions for modification of a t-cell receptor gene
CA2848417C (en) 2011-09-21 2023-05-02 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
CA2852955C (en) 2011-10-27 2021-02-16 Sangamo Biosciences, Inc. Methods and compositions for modification of the hprt locus
JP6144691B2 (ja) 2011-11-16 2017-06-07 サンガモ セラピューティクス, インコーポレイテッド 修飾されたdna結合タンパク質およびその使用
CN113896787A (zh) 2011-12-22 2022-01-07 弗·哈夫曼-拉罗切有限公司 表达载体元件组合、新的生产用细胞产生方法及其在重组产生多肽中的用途
US20140001721A1 (en) 2012-06-28 2014-01-02 John C. Benko Ergonomic cart
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
HUE051612T2 (hu) * 2012-07-11 2021-03-01 Sangamo Therapeutics Inc Eljárások és készítmények lizoszomális tárolási betegségek kezelésére
AU2013329186B2 (en) 2012-10-10 2019-02-14 Sangamo Therapeutics, Inc. T cell modifying compounds and uses thereof
CA2892448A1 (en) 2012-12-05 2014-06-12 Sangamo Biosciences, Inc. Methods and compositions for regulation of metabolic disorders
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
US10604771B2 (en) 2013-05-10 2020-03-31 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
CN116083487A (zh) 2013-05-15 2023-05-09 桑格摩生物治疗股份有限公司 用于治疗遗传病状的方法和组合物
CA2920899C (en) 2013-08-28 2023-02-28 Sangamo Biosciences, Inc. Compositions for linking dna-binding domains and cleavage domains
TWI793159B (zh) 2013-10-23 2023-02-21 美商健臻公司 重組醣蛋白及其用途
EP3080274B1 (en) * 2013-12-09 2020-06-03 Sangamo Therapeutics, Inc. Methods and compositions for genome engineering
GB201322798D0 (en) 2013-12-20 2014-02-05 Oxford Biomedica Ltd Production system
MX378789B (es) 2014-05-08 2025-03-11 Sangamo Biosciences Inc Métodos y composiciones para tratar la enfermedad de huntington.
SG11201700404WA (en) 2014-07-18 2017-02-27 Celltheon Corp Methods and compositions for expression of polypeptides in a cell
US9816074B2 (en) 2014-07-25 2017-11-14 Sangamo Therapeutics, Inc. Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells
WO2016019144A2 (en) 2014-07-30 2016-02-04 Sangamo Biosciences, Inc. Gene correction of scid-related genes in hematopoietic stem and progenitor cells
MA41346A (fr) 2015-01-12 2017-11-21 Juno Therapeutics Inc Eléments régulateurs post-transcriptionnels d'hépatite modifiée
US10179918B2 (en) 2015-05-07 2019-01-15 Sangamo Therapeutics, Inc. Methods and compositions for increasing transgene activity
HUE068603T2 (hu) 2015-06-23 2025-01-28 Childrens Hospital Philadelphia Módosított IX-es faktor, valamint készítmények, eljárások és alkalmazások sejtekbe, szervekbe és szövetekbe történõ géntranszferhez
RS63986B1 (sr) 2015-10-28 2023-03-31 Acuitas Therapeutics Inc Novi lipidi i lipidne formulacije nanočestica za isporuku nukleinskih kiselina
KR102766526B1 (ko) 2015-10-28 2025-02-13 상가모 테라퓨틱스, 인코포레이티드 간-특이적 작제물인, 인자 viii 발현 카세트 및 이의 사용 방법
JP7012650B2 (ja) 2016-02-02 2022-01-28 サンガモ セラピューティクス, インコーポレイテッド Dna結合ドメインと切断ドメインとを連結するための組成物
SG11201901364VA (en) 2016-08-24 2019-03-28 Sangamo Therapeutics Inc Engineered target specific nucleases
JP7042263B2 (ja) 2016-10-20 2022-03-25 サンガモ セラピューティクス, インコーポレイテッド ファブリー病の治療のための方法および組成物
AU2017374042C1 (en) 2016-12-09 2024-07-11 Acuitas Therapeutics, Inc. Delivery of target specific nucleases
US11357834B2 (en) 2017-01-10 2022-06-14 Amicus Therapeutics, Inc. Recombinant α-galactosidase A for treatment of Fabry disease
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
KR102735096B1 (ko) 2017-10-11 2024-11-28 (주)셀트리온 고발현 및 고기능성 목적 단백질의 생산을 위한 발현 카세트 및 이의 이용
MX2020008272A (es) 2018-02-08 2020-09-21 Sangamo Therapeutics Inc Nucleasas diseñadas específicas de blancos.
WO2020077114A2 (en) 2018-10-10 2020-04-16 Amicus Therapeutics, Inc. Disulfide bond stabilized polypeptide compositions and methods of use
CN113423419A (zh) 2019-01-04 2021-09-21 桑格摩生物治疗股份有限公司 用于治疗法布里病的方法和组合物

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