SG10201804976YA - Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for Genome Editing - Google Patents

Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for Genome Editing

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Publication number
SG10201804976YA
SG10201804976YA SG10201804976YA SG10201804976YA SG10201804976YA SG 10201804976Y A SG10201804976Y A SG 10201804976YA SG 10201804976Y A SG10201804976Y A SG 10201804976YA SG 10201804976Y A SG10201804976Y A SG 10201804976YA SG 10201804976Y A SG10201804976Y A SG 10201804976YA
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Singapore
Prior art keywords
delivery
compositions
crispr
systems
therapeutic applications
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SG10201804976YA
Inventor
Cong Le
Benjamin Turitz Cox David
HEIDENREICH Matthias
jeffrey platt Randall
Swiech Lukasz
Zhang Feng
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Broad Inst Inc
Massachusetts Inst Technology
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Publication of SG10201804976YA publication Critical patent/SG10201804976YA/en

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Abstract

DELIVERY, USE AND THERAPEUTIC APPLICATIONS OF THE CRISPR-CAS SYSTEMS AND COMPOSITIONS FOR GENOME EDITING The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target 5 sequences. Provided are delivery systems and tissues or organ which are targeted as sites for delivery. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or 10 modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition. Figure 1A
SG10201804976YA 2013-12-12 2014-12-12 Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for Genome Editing SG10201804976YA (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201361915148P 2013-12-12 2013-12-12
US201361915107P 2013-12-12 2013-12-12
US201361915145P 2013-12-12 2013-12-12
US201361915153P 2013-12-12 2013-12-12
US201361915192P 2013-12-12 2013-12-12
US201361915176P 2013-12-12 2013-12-12
US201361915215P 2013-12-12 2013-12-12

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SG10201804976YA true SG10201804976YA (en) 2018-07-30

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EP (2) EP3079725B1 (en)
JP (3) JP7103750B2 (en)
KR (2) KR20250068794A (en)
CN (3) CN106061510B (en)
AU (3) AU2014361781B2 (en)
BR (1) BR112016013201B1 (en)
CA (1) CA2932478A1 (en)
DK (1) DK3079725T3 (en)
ES (1) ES2765481T3 (en)
IL (3) IL316141A (en)
MX (2) MX374529B (en)
SG (1) SG10201804976YA (en)
WO (1) WO2015089462A1 (en)
ZA (1) ZA201603714B (en)

Families Citing this family (265)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3613852A3 (en) 2011-07-22 2020-04-22 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
CN105121631B (en) 2013-02-20 2019-04-19 瑞泽恩制药公司 Genetic modification of rats
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
KR102186281B1 (en) 2013-04-16 2020-12-03 리제너론 파마슈티칼스 인코포레이티드 Targeted modification of rat genome
KR20160056869A (en) * 2013-06-17 2016-05-20 더 브로드 인스티튜트, 인코퍼레이티드 Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
JP2016536021A (en) 2013-11-07 2016-11-24 エディタス・メディシン，インコーポレイテッド CRISPR-related methods and compositions with governing gRNA
TW202346604A (en) 2013-11-15 2023-12-01 美商阿韋利諾美國實驗有限公司 Methods for multiplex detection of alleles associated with ophthalmic conditions
JP6174811B2 (en) 2013-12-11 2017-08-02 リジェネロン・ファーマシューティカルズ・インコーポレイテッドＲｅｇｅｎｅｒｏｎ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． Methods and compositions for targeted genomic modification
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
EP3114227B1 (en) * 2014-03-05 2021-07-21 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
ES2745769T3 (en) 2014-03-10 2020-03-03 Editas Medicine Inc CRISPR / CAS related procedures and compositions for treating Leber 10 congenital amaurosis (LCA10)
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
EP3981876A1 (en) 2014-03-26 2022-04-13 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
EP3177718B1 (en) 2014-07-30 2022-03-16 President and Fellows of Harvard College Cas9 proteins including ligand-dependent inteins
JP6788584B2 (en) * 2014-10-31 2020-11-25 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 Massively Parallel Combinatorial Genetics on CRISPR
AU2015349692B2 (en) 2014-11-21 2021-10-28 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modification using paired guide RNAs
EP3626832B1 (en) 2014-11-25 2024-06-12 The Brigham and Women's Hospital, Inc. Method of identifying a person having a predisposition to or afflicted with a cardiometabolic disease
EP4400584A3 (en) 2014-12-03 2024-10-16 Agilent Technologies, Inc. Guide rna with chemical modifications
EP3230451B1 (en) 2014-12-12 2021-04-07 The Broad Institute, Inc. Protected guide rnas (pgrnas)
EP3230452B1 (en) 2014-12-12 2025-06-11 The Broad Institute, Inc. Dead guides for crispr transcription factors
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
WO2016100974A1 (en) 2014-12-19 2016-06-23 The Broad Institute Inc. Unbiased identification of double-strand breaks and genomic rearrangement by genome-wide insert capture sequencing
US10196613B2 (en) 2014-12-19 2019-02-05 Regeneron Pharmaceuticals, Inc. Stem cells for modeling type 2 diabetes
WO2016106236A1 (en) 2014-12-23 2016-06-30 The Broad Institute Inc. Rna-targeting system
EP3702456A1 (en) 2014-12-24 2020-09-02 The Broad Institute, Inc. Crispr having or associated with destabilization domains
WO2016108926A1 (en) 2014-12-30 2016-07-07 The Broad Institute Inc. Crispr mediated in vivo modeling and genetic screening of tumor growth and metastasis
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
EP3280803B1 (en) 2015-04-06 2021-05-26 The Board of Trustees of the Leland Stanford Junior University Chemically modified guide rnas for crispr/cas-mediated gene regulation
CA2981716C (en) 2015-04-10 2022-04-12 Feldan Bio Inc. Polypeptide-based shuttle agents for improving the transduction efficiency of polypeptide cargos to the cytosol of target eukaryotic cells, uses thereof, methods and kits relating to same
SG11201708653RA (en) 2015-04-24 2017-11-29 Editas Medicine Inc Evaluation of cas9 molecule/guide rna molecule complexes
FI3430134T3 (en) 2015-06-18 2023-01-13 Novel crispr enzymes and systems
AU2016279062A1 (en) 2015-06-18 2019-03-28 Omar O. Abudayyeh Novel CRISPR enzymes and systems
EP3313989B1 (en) 2015-06-29 2024-12-25 Ionis Pharmaceuticals, Inc. Modified crispr rna and modified single crispr rna and uses thereof
US10456452B2 (en) 2015-07-02 2019-10-29 Poseida Therapeutics, Inc. Compositions and methods for improved encapsulation of functional proteins in polymeric vesicles
BR112017028201A2 (en) * 2015-07-02 2018-08-28 Univ Johns Hopkins crisp / cas9 based treatments
ES2948559T3 (en) * 2015-07-25 2023-09-14 Habib Frost A system, device and method for providing a therapy or cure for cancer and other disease states
EP3334484B1 (en) 2015-08-12 2024-12-25 The General Hospital Corporation Compositions that promote hypoxia or the hypoxia response for the treatment and prevention of mitochondrial dysfunction
WO2017035416A2 (en) * 2015-08-25 2017-03-02 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
EP4530354A3 (en) * 2015-09-08 2025-09-10 Precision Biosciences, Inc. Treatment of retinitis pigmentosa using engineered meganucleases
FR3042506B1 (en) * 2015-10-16 2018-11-30 IFP Energies Nouvelles GENETIC TOOL FOR PROCESSING BACTERIA CLOSTRIDIUM
WO2017068077A1 (en) 2015-10-20 2017-04-27 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and products for genetic engineering
SG10202104041PA (en) 2015-10-23 2021-06-29 Harvard College Nucleobase editors and uses thereof
KR101651817B1 (en) 2015-10-28 2016-08-29 대한민국 Primer set for Preparation of NGS library and Method and Kit for making NGS library using the same
ES2905558T3 (en) 2015-11-13 2022-04-11 Avellino Lab Usa Inc Procedures for the treatment of corneal dystrophies
CA3005878A1 (en) 2015-11-19 2017-05-26 The Brigham And Women's Hospital, Inc. Lymphocyte antigen cd5-like (cd5l)-interleukin 12b (p40) heterodimers in immunity
KR20250044471A (en) 2015-11-30 2025-03-31 듀크 유니버시티 Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
WO2017099494A1 (en) 2015-12-08 2017-06-15 기초과학연구원 Genome editing composition comprising cpf1, and use thereof
EP3984550A1 (en) 2015-12-11 2022-04-20 Massachusetts Eye & Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells
US11208652B2 (en) * 2016-02-04 2021-12-28 President And Fellows Of Harvard College Mitochondrial genome editing and regulation
US20190032088A1 (en) * 2016-02-26 2019-01-31 Cellectis Micelle based system nuclease encapsulation for in-vivo gene editing
EP3426715A1 (en) * 2016-03-12 2019-01-16 The Regents of the University of California Biodegradable vectors for efficient rna delivery
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
CA3018729A1 (en) 2016-03-23 2017-09-28 The Regents Of The University Of California Methods of treating mitochondrial disorders
US12011488B2 (en) 2016-03-23 2024-06-18 The Regents Of The University Of California Methods of treating mitochondrial disorders
WO2017165862A1 (en) 2016-03-25 2017-09-28 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
WO2017175072A1 (en) * 2016-04-08 2017-10-12 Feldan Bio Inc. Peptide shuttle based gene disruption
EP3443080B1 (en) * 2016-04-13 2021-08-11 University of Massachusetts Repairing compound heterozygous recessive mutations by allele exchange
EP3443081A4 (en) 2016-04-13 2019-10-30 Duke University CRISPR / CAS9-BASED REPRESSORS TO INACTIVATE IN VIVO GENE TARGETS AND METHODS OF USE
ES2865481T3 (en) 2016-04-29 2021-10-15 Poseida Therapeutics Inc Poly (histidine) -based micelles for complexation and delivery of proteins and nucleic acids
KR102356542B1 (en) 2016-05-20 2022-01-28 리제너론 파마슈티칼스 인코포레이티드 A method for disrupting immunological resistance using multiple guide RNAs
RU2771383C2 (en) * 2016-06-03 2022-05-04 Энсэрм (Энститю Насьональ Де Ла Санте Э Де Ла Решерш Медикаль) FOOD RATION-CONTROLLED EXPRESSION OF NUCLEIC ACID ENCODING Cas9 NUCLEASE, AND ITS APPLICATIONS
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
WO2017218852A1 (en) * 2016-06-15 2017-12-21 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
ES2989940T3 (en) * 2016-06-29 2024-11-28 Crispr Therapeutics Ag Materials and methods for the treatment of Friedreich's ataxia and other related disorders
CA3029874A1 (en) * 2016-07-05 2018-01-11 The Johns Hopkins University Crispr/cas9-based compositions and methods for treating retinal degenerations
JP7490211B2 (en) 2016-07-19 2024-05-27 デューク ユニバーシティ Therapeutic Applications of CPF1-Based Genome Editing
JP6875500B2 (en) * 2016-07-28 2021-05-26 インスティテュート フォー ベーシック サイエンスＩｎｓｔｉｔｕｔｅ Ｆｏｒ Ｂａｓｉｃ Ｓｃｉｅｎｃｅ Pharmaceutical composition for treating eye diseases containing Cas9 protein and guide RNA
WO2018026976A1 (en) 2016-08-02 2018-02-08 Editas Medicine, Inc. Compositions and methods for treating cep290 associated disease
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
WO2018031683A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
EP3500671B1 (en) 2016-08-17 2024-07-10 The Broad Institute, Inc. Method of selecting target sequences for the design of guide rnas
EP3500670B1 (en) 2016-08-17 2024-07-10 The Broad Institute, Inc. Method for selecting target sequences for guide rna of crispr systems
JP7050215B2 (en) * 2016-08-19 2022-04-08 ツールゲン インコーポレイテッド Artificially manipulated angiogenesis regulatory system
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
EP3512943B1 (en) * 2016-09-14 2023-04-12 Yeda Research and Development Co. Ltd. Crisp-seq, an integrated method for massively parallel single cell rna-seq and crispr pooled screens
GB201616202D0 (en) * 2016-09-23 2016-11-09 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of eye deisease
KR102626671B1 (en) 2016-10-12 2024-01-18 펠단 바이오 인코포레이티드 Rationally-designed synthetic peptide shuttle agents for delivering polypeptide cargo from the extracellular space to the cytoplasm and/or nucleus of a target eukaryotic cell, uses thereof, methods and kits related thereto
US9982267B2 (en) 2016-10-12 2018-05-29 Feldan Bio Inc. Rationally-designed synthetic peptide shuttle agents for delivering polypeptide cargos from an extracellular space to the cytosol and/or nucleus of a target eukaryotic cell, uses thereof, methods and kits relating to same
US11629170B2 (en) 2016-10-12 2023-04-18 Feldan Bio Inc. Rationally-designed synthetic peptide shuttle agents for delivering polypeptide cargos from an extracellular space to the cytosol and/or nucleus of a target eukaryotic cell, uses thereof, methods and kits relating to same
EP3526320A1 (en) 2016-10-14 2019-08-21 President and Fellows of Harvard College Aav delivery of nucleobase editors
CN108018314A (en) * 2016-11-03 2018-05-11 优美佳生物技术有限公司 Method and composition for cell reprogramming
US12227578B2 (en) 2016-11-11 2025-02-18 The Broad Institute, Inc. Modulation of intestinal epithelial cell differentiation, maintenance and/or function through T cell action
KR101917287B1 (en) 2016-12-19 2018-11-13 한국과학기술연구원 self-assembled ribonucleoprotein nanoparticles
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
WO2018129129A1 (en) * 2017-01-05 2018-07-12 Rutgers, The State University Of New Jersey Targeted gene editing platform independent of dna double strand break and uses thereof
WO2018131551A1 (en) * 2017-01-13 2018-07-19 学校法人自治医科大学 Aav vector for disrupting clotting-related factor gene on liver genome
TW201839136A (en) 2017-02-06 2018-11-01 瑞士商諾華公司 Composition and method for treating hemochromatosis
EP3576696A4 (en) 2017-02-06 2020-09-16 Children's Medical Center Corporation MATERIALS AND METHODS FOR DELIVERING NUCLEIC ACIDS TO COCHLEA AND AURIAL CELLS
WO2018147343A1 (en) * 2017-02-07 2018-08-16 Edigene Corporation Method of treating diseases associated with elevated kras expression using crispr-gndm system
EP3594339A4 (en) * 2017-03-06 2020-12-30 Institute For Basic Science COMPOSITION WITH C2CL ENDONUCLEASE FOR DIELECTRIC CALIBRATION AND PROCEDURE FOR DIELECTRIC CALIBRATION WITH IT
EP3592381A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Cancer vaccine
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
JP2020510439A (en) 2017-03-10 2020-04-09 プレジデント アンド フェローズ オブ ハーバード カレッジ Base-editing factor from cytosine to guanine
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
KR102454284B1 (en) * 2017-03-15 2022-10-12 더 브로드 인스티튜트, 인코퍼레이티드 Novel cas13b orthologues crispr enzymes and systems
WO2018176009A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
WO2018175924A1 (en) 2017-03-24 2018-09-27 The Broad Institute, Inc. Methods and compositions for regulating innate lymphoid cell inflammatory responses
WO2018183908A1 (en) 2017-03-31 2018-10-04 Dana-Farber Cancer Institute, Inc. Compositions and methods for treating ovarian tumors
EP3610266A4 (en) 2017-04-12 2021-04-21 Massachusetts Eye and Ear Infirmary TUMOR SIGNATURE OF METASTASIS, COMPOSITIONS OF SUBSTANCES AND USES THEREOF
WO2018191520A1 (en) 2017-04-12 2018-10-18 The Broad Institute, Inc. Respiratory and sweat gland ionocytes
US12350368B2 (en) 2017-04-14 2025-07-08 The Broad Institute, Inc. Delivery of large payloads
EP3612232A1 (en) 2017-04-21 2020-02-26 The Broad Institute, Inc. Targeted delivery to beta cells
JOP20190257A1 (en) * 2017-04-28 2019-10-28 Novartis Ag 6-6 fused bicyclic heteroaryl compounds and their use as lats inhibitors
WO2018204777A2 (en) 2017-05-05 2018-11-08 The Broad Institute, Inc. Methods for identification and modification of lncrna associated with target genotypes and phenotypes
WO2018209158A2 (en) 2017-05-10 2018-11-15 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
US12226479B2 (en) 2017-05-11 2025-02-18 The General Hospital Corporation Methods and compositions of use of CD8+ tumor infiltrating lymphocyte subtypes and gene signatures thereof
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
US12297436B2 (en) 2017-05-18 2025-05-13 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
AU2018270088B2 (en) 2017-05-18 2024-05-16 Massachusetts Institute Of Technology Systems, methods, and compositions for targeted nucleic acid editing
WO2018220211A1 (en) * 2017-06-02 2018-12-06 Institut National De La Sante Et De La Recherche Medicale (Inserm) Viral vector combining gene therapy and genome editing approaches for gene therapy of genetic disorders
EP3635113A4 (en) 2017-06-05 2021-03-17 Fred Hutchinson Cancer Research Center GENOMIC SAFE HARBORS FOR GENETIC THERAPIES IN HUMAN STEM CELLS AND MANIPULATED NANOPARTICLES TO DELIVER TARGETED GENETIC THERAPIES
EP3638218A4 (en) 2017-06-14 2021-06-09 The Broad Institute, Inc. COMPOSITIONS AND METHODS TARGETING COMPONENT 3 OF THE COMPLEMENT TO INHIBIT TUMOR GROWTH
MY200418A (en) 2017-06-15 2023-12-25 Univ California Targeted non-viral dna insertions
SG11201911623WA (en) 2017-06-15 2020-01-30 Toolgen Inc Platform for expressing protein of interest in liver
US11773407B2 (en) * 2017-06-26 2023-10-03 Arizona Board Of Regents On Behalf Of Arizona State University CRISPR logic circuits for safer and controllable gene therapies
EP3645054A4 (en) 2017-06-26 2021-03-31 The Broad Institute, Inc. COMPOSITIONS BASED ON CRISPR / CAS-ADENIN-DEAMINASE, SYSTEMS AND METHODS FOR TARGETED NUCLEIC ACID EDITING
WO2019005853A2 (en) * 2017-06-26 2019-01-03 Arizona Board Of Regents On Behalf Of Arizona State University Crispr-based synthetic gene circuits as next generation gene therapy of inner ear
US20200123567A1 (en) * 2017-06-26 2020-04-23 Samira Kiani A universal platform to enhance crispr-based gene editing for in vivo therapies
KR102007457B1 (en) * 2017-06-30 2019-08-05 동국대학교 산학협력단 Nanocomplex comprising Cas9 protein, guide RNA and amphipathic peptide, and uses of the same
WO2019014581A1 (en) 2017-07-14 2019-01-17 The Broad Institute, Inc. Methods and compositions for modulating cytotoxic lymphocyte activity
WO2019018440A1 (en) 2017-07-17 2019-01-24 The Broad Institute, Inc. Cell atlas of the healthy and ulcerative colitis human colon
CN111801345A (en) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 Methods and compositions using an evolved base editor for Phage Assisted Continuous Evolution (PACE)
BR102017016440A2 (en) 2017-07-31 2019-03-19 Universidade Federal Do Rio Grande Do Sul COMPOSITION FOR CENTRAL NERVOUS SYSTEM GENE THERAPY, OBTAINING AND USE PROCESS
EP3675913A4 (en) * 2017-08-28 2021-06-02 University of Florida Research Foundation, Incorporated METHODS AND COMPOSITIONS FOR TREATMENT OF CONE-ROD DYSTROPHY
WO2019046211A1 (en) * 2017-08-28 2019-03-07 Wisconsin Alumni Research Foundation Nanocapsules for delivering ribonucleoproteins
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
EP3684397A4 (en) * 2017-09-21 2021-08-18 The Broad Institute, Inc. SYSTEMS, PROCEDURES AND COMPOSITIONS FOR TARGETED NUCLEIC ACID EDITING
EP3692152B1 (en) 2017-10-04 2025-09-03 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
MX2019014690A (en) * 2017-10-16 2020-02-07 Illumina Inc TECHNIQUES BASED ON DEEP LEARNING FOR THE TRAINING OF DEEP CONVOLUTIONAL NEURONAL NETWORKS.
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
CN119265240A (en) 2017-10-20 2025-01-07 弗莱德哈钦森癌症中心 Systems and methods for generating B cells genetically modified to express selected antibodies
CN111867635A (en) * 2017-10-20 2020-10-30 法国国家卫生及研究医学协会 A method of expressing a polynucleotide of interest in a subject's cone photoreceptors comprising subretinal delivery of a therapeutically effective amount of a recombinant AAV9-derived vector
KR102503130B1 (en) 2017-10-27 2023-02-24 더 리전트 오브 더 유니버시티 오브 캘리포니아 Targeted replacement of endogenous T cell receptors
EP3707258A1 (en) * 2017-11-06 2020-09-16 Editas Medicine, Inc. Methods, compositions and components for crispr-cas9 editing of cblb in t cells for immunotherapy
GB2568255A (en) * 2017-11-08 2019-05-15 Evox Therapeutics Ltd Exosomes comprising RNA therapeutics
US12171783B2 (en) 2017-11-13 2024-12-24 The Broad Institute, Inc. Methods and compositions for targeting developmental and oncogenic programs in H3K27M gliomas
US12221720B2 (en) 2017-11-13 2025-02-11 The Broad Institute, Inc. Methods for determining spatial and temporal gene expression dynamics during adult neurogenesis in single cells
CA3082450A1 (en) * 2017-11-21 2019-05-31 Crispr Therapeutics Ag Materials and methods for treatment of autosomal dominant retinitis pigmentosa
US20220290186A1 (en) * 2017-12-06 2022-09-15 Generation Bio Co. Gene editing using a modified closed-ended dna (cedna)
WO2019113506A1 (en) 2017-12-07 2019-06-13 The Broad Institute, Inc. Methods and compositions for multiplexing single cell and single nuclei sequencing
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND PROCEDURES FOR PREDICTING REPAIR RESULTS IN GENE ENGINEERING
WO2019123429A1 (en) * 2017-12-21 2019-06-27 Casebia Therapeutics Llp Materials and methods for treatment of usher syndrome type 2a
US11994512B2 (en) 2018-01-04 2024-05-28 Massachusetts Institute Of Technology Single-cell genomic methods to generate ex vivo cell systems that recapitulate in vivo biology with improved fidelity
CN109507429A (en) * 2018-02-14 2019-03-22 复旦大学 Method based on CRISPR/cas9 and peroxidase APEX2 system identification analysis specific position interaction protein
CA3093490A1 (en) 2018-03-05 2019-09-12 Children's Medical Center Corporation Compositions and methods for delivering nucleic acids to cochlear and vestibular cells
US20200405884A1 (en) * 2018-03-09 2020-12-31 Board Of Regents, The University Of Texas System Crispr-nanoparticles and methods of use in brain disorders
KR20200132924A (en) 2018-03-14 2020-11-25 에디타스 메디신, 인코포레이티드 Systems and methods for the treatment of hemoglobinosis
US20190284553A1 (en) * 2018-03-15 2019-09-19 KSQ Therapeutics, Inc. Gene-regulating compositions and methods for improved immunotherapy
EP4524247A3 (en) * 2018-03-23 2025-06-11 Massachusetts Eye and Ear Infirmary Crispr/cas9-mediated exon-skipping approach for ush2a-associated usher syndrome
EP3773520A4 (en) * 2018-04-09 2021-12-22 Orgenesis Inc. Bioxomes particles, redoxomes, method and composition
EP3781711B1 (en) 2018-04-19 2024-11-20 Massachusetts Institute Of Technology Single-stranded break detection in double-stranded dna
SI3560330T1 (en) 2018-04-24 2022-08-31 KWS SAAT SE & Co. KGaA Plants with improved digestibility and marker haplotypes
JP2021521825A (en) * 2018-04-24 2021-08-30 リガンダル・インコーポレイテッド Genome editing method and composition
US11957695B2 (en) 2018-04-26 2024-04-16 The Broad Institute, Inc. Methods and compositions targeting glucocorticoid signaling for modulating immune responses
WO2019210268A2 (en) 2018-04-27 2019-10-31 The Broad Institute, Inc. Sequencing-based proteomics
EP3787693A4 (en) * 2018-04-30 2022-03-09 Oregon Health & Science University METHODS OF GENE THERAPY
US20190336585A1 (en) * 2018-05-03 2019-11-07 John Lawrence Mee Method for sustainable human cognitive enhancement
US20210393796A1 (en) * 2018-05-03 2021-12-23 Cognigenics, Inc. Methods for sustainable human cognitive enhancement
WO2019213660A2 (en) 2018-05-04 2019-11-07 The Broad Institute, Inc. Compositions and methods for modulating cgrp signaling to regulate innate lymphoid cell inflammatory responses
WO2019217942A1 (en) 2018-05-11 2019-11-14 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
WO2019217943A1 (en) * 2018-05-11 2019-11-14 Beam Therapeutics Inc. Methods of editing single nucleotide polymorphism using programmable base editor systems
CN112941155B (en) * 2018-05-14 2023-07-14 北京艾克伦医疗科技有限公司 DNA primer pair with stem-loop structure and application thereof
CN108587998B (en) * 2018-05-17 2021-05-18 浙江大学 Exosome, preparation method of exosome and application of exosome in preparation of medicine for treating skin superficial tumors
US20190359985A1 (en) * 2018-05-22 2019-11-28 John Lawrence Mee Adjustable method for sustainable human cognitive enhancement
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
CN108707627A (en) * 2018-05-31 2018-10-26 深圳市免疫基因治疗研究院 A kind of MLD slow virus carriers, slow virus and its preparation method and application
US20210371932A1 (en) 2018-06-01 2021-12-02 Massachusetts Institute Of Technology Methods and compositions for detecting and modulating microenvironment gene signatures from the csf of metastasis patients
US12036240B2 (en) 2018-06-14 2024-07-16 The Broad Institute, Inc. Compositions and methods targeting complement component 3 for inhibiting tumor growth
US12357580B2 (en) 2018-06-19 2025-07-15 The Board Of Regents Of The University Of Texas System Lipid nanoparticle compositions for delivery of mRNA and long nucleic acids
US20220000988A1 (en) * 2018-06-23 2022-01-06 John Lawrence Mee Reversible method for sustainable human cognitive enhancement
US20190390193A1 (en) * 2018-06-23 2019-12-26 John Lawrence Mee Reversible method for sustainable human cognitive enhancement
JP2021528975A (en) 2018-06-26 2021-10-28 ザ・ブロード・インスティテュート・インコーポレイテッド Compositions, systems, and methods for amplification using CRISPR / CAS and transposases
AU2019294630A1 (en) 2018-06-26 2020-12-17 Massachusetts Institute Of Technology Crispr effector system based amplification methods, systems, and diagnostics
IL260445B (en) * 2018-07-05 2019-08-29 Lempo Therapeutics Ltd Methods and compositions for modulating myeloperoxidase (mpo) expression
WO2020021312A1 (en) 2018-07-26 2020-01-30 Uniwersytet Jagiellonski In vivo delivery system of the genome dna modifying enzymes and the use thereof
EP3827847A4 (en) * 2018-07-26 2023-07-19 Toolgen Incorporated GENE EDIT OF ANTICOAGULANTS
WO2020028823A1 (en) * 2018-08-03 2020-02-06 Beam Therapeutics Inc. Multi-effector nucleobase editors and methods of using same to modify a nucleic acid target sequence
JP2021532815A (en) 2018-08-07 2021-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド New Cas12b enzyme and system
US20210324357A1 (en) 2018-08-20 2021-10-21 The Brigham And Women's Hospital, Inc. Degradation domain modifications for spatio-temporal control of rna-guided nucleases
WO2020041380A1 (en) 2018-08-20 2020-02-27 The Broad Institute, Inc. Methods and compositions for optochemical control of crispr-cas9
CA3111483A1 (en) 2018-09-04 2020-03-12 The Board Of Regents Of The University Of Texas System Compositions and methods for organ specific delivery of nucleic acids
GB2592505B (en) 2018-09-04 2023-05-03 Univ Texas Compositions and methods for organ specific delivery of nucleic acids
CN109402115B (en) * 2018-09-06 2024-02-02 广州普世利华科技有限公司 gRNA of target Rett mutant gene RNA, detection method of Rett mutant gene and detection kit
US20210317479A1 (en) 2018-09-06 2021-10-14 The Broad Institute, Inc. Nucleic acid assemblies for use in targeted delivery
EP3849565A4 (en) 2018-09-12 2022-12-28 Fred Hutchinson Cancer Research Center REDUCING CD33 EXPRESSION FOR SELECTIVE PROTECTION OF THERAPEUTIC CELLS
CA3113095A1 (en) 2018-09-18 2020-03-26 Vnv Newco Inc. Arc-based capsids and uses thereof
US20210355492A1 (en) * 2018-09-21 2021-11-18 City University Of Hong Kong Cargo loaded extracellular vesicles
US12331320B2 (en) 2018-10-10 2025-06-17 The Research Foundation For The State University Of New York Genome edited cancer cell vaccines
US20220411783A1 (en) 2018-10-12 2022-12-29 The Broad Institute, Inc. Method for extracting nuclei or whole cells from formalin-fixed paraffin-embedded tissues
US20210379057A1 (en) 2018-10-16 2021-12-09 Massachusetts Institute Of Technology Nutlin-3a for use in treating a mycobacterium tuberculosis infection
CN109957568B (en) * 2018-10-16 2024-02-02 广州普世利华科技有限公司 gRNA for targeting HBB RNA, C2C 2-based HBB mutation detection method and detection kit
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
US20210401008A1 (en) 2018-11-08 2021-12-30 Triton Algae Innovations, Inc. Compositions and methods for incorporating heme from algae in edible products
US12165743B2 (en) 2018-11-09 2024-12-10 The Broad Institute, Inc. Compressed sensing for screening and tissue imaging
US12402610B2 (en) 2018-11-09 2025-09-02 The Broad Institute, Inc. Methods and compositions for modulating innate lymphoid cell pathogenic effectors
EP3894568A2 (en) * 2018-12-12 2021-10-20 Flagship Pioneering Innovations V, Inc. Anellosomes for delivering secreted therapeutic modalities
WO2020123906A1 (en) * 2018-12-13 2020-06-18 The General Hospital Corporation Biologically informed and accurate sequence alignment
CN111321171A (en) * 2018-12-14 2020-06-23 江苏集萃药康生物科技有限公司 Method for preparing gene targeting animal model by applying CRISPR/Cas9 mediated ES targeting technology
WO2020131862A1 (en) 2018-12-17 2020-06-25 The Broad Institute, Inc. Crispr-associated transposase systems and methods of use thereof
US20220062394A1 (en) 2018-12-17 2022-03-03 The Broad Institute, Inc. Methods for identifying neoantigens
KR20210118402A (en) * 2018-12-23 2021-09-30 씨에스엘 베링 엘엘씨 Hematopoietic stem cell-gene therapy for Wiskott-Aldrich syndrome
KR102386498B1 (en) * 2018-12-27 2022-04-15 한양대학교 산학협력단 composition for gene editing based on CRISPR-CAS
WO2020139031A1 (en) * 2018-12-27 2020-07-02 한양대학교 산학협력단 Crispr-cas-based composition for gene correction
US11739156B2 (en) 2019-01-06 2023-08-29 The Broad Institute, Inc. Massachusetts Institute of Technology Methods and compositions for overcoming immunosuppression
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
EP3931326A1 (en) * 2019-02-25 2022-01-05 Editas Medicine, Inc. Crispr/rna-guided nuclease-related methods and compositions for treating rho-associated autosomal-dominant retinitis pigmentosa (adrp)
AU2020229871B2 (en) * 2019-02-28 2023-05-18 Regeneron Pharmaceuticals, Inc. Adeno-associated virus vectors for the delivery of therapeutics
US20220170013A1 (en) * 2019-03-06 2022-06-02 The Broad Institute, Inc. T:a to a:t base editing through adenosine methylation
WO2020191246A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
CN113891727A (en) * 2019-03-27 2022-01-04 全国儿童医院研究所 Generation of Chimeric Antigen Receptor (CAR) -primary NK cells for cancer immunotherapy using a combination of CAS9/RNP and AAV viruses
CA3040645A1 (en) 2019-04-18 2020-10-18 Feldan Bio Inc. Peptide-based non-proteinaceous cargo delivery
CN110129368B (en) * 2019-04-22 2021-03-09 中国科学院脑科学与智能技术卓越创新中心 AAV vector for infecting support cell and hair cell
CN114096684B (en) 2019-05-13 2024-11-19 科沃施种子欧洲股份两合公司 Drought tolerance of corn
WO2020236972A2 (en) 2019-05-20 2020-11-26 The Broad Institute, Inc. Non-class i multi-component nucleic acid targeting systems
AR118995A1 (en) 2019-05-25 2021-11-17 Kws Saat Se & Co Kgaa HAPLOID INDUCTION ENHANCER
US20220243178A1 (en) 2019-05-31 2022-08-04 The Broad Institute, Inc. Methods for treating metabolic disorders by targeting adcy5
CN110241098B (en) * 2019-06-05 2021-04-30 复旦大学 Truncated high specificity variant of CRISPR nuclease SpCas9 of Streptococcus pyogenes and its application
CN114258398A (en) 2019-06-13 2022-03-29 总医院公司 Engineered human endogenous virus-like particles and methods of using the same for delivery to cells
CN112143701A (en) * 2019-06-26 2020-12-29 中国科学院脑科学与智能技术卓越创新中心 Method and reagent for inhibiting choroidal neovascularization based on RNA (ribonucleic acid) site-specific editing
CN114729383A (en) 2019-07-02 2022-07-08 弗莱德哈钦森癌症研究中心 Recombinant AD35 vector and related gene therapy improvement
WO2021021349A1 (en) * 2019-07-30 2021-02-04 Nxgen Medicine, Inc. Methods of treating pain conditions and compositions related thereto
EP3772542A1 (en) 2019-08-07 2021-02-10 KWS SAAT SE & Co. KGaA Modifying genetic variation in crops by modulating the pachytene checkpoint protein 2
US20220333172A1 (en) * 2019-08-16 2022-10-20 The Jackson Laboratory Live cell imaging of non-repetitive genomic loci
US20220298501A1 (en) 2019-08-30 2022-09-22 The Broad Institute, Inc. Crispr-associated mu transposase systems
US11981922B2 (en) 2019-10-03 2024-05-14 Dana-Farber Cancer Institute, Inc. Methods and compositions for the modulation of cell interactions and signaling in the tumor microenvironment
WO2021072328A1 (en) 2019-10-10 2021-04-15 The Broad Institute, Inc. Methods and compositions for prime editing rna
WO2021074367A1 (en) 2019-10-17 2021-04-22 KWS SAAT SE & Co. KGaA Enhanced disease resistance of crops by downregulation of repressor genes
EP4054651A1 (en) * 2019-11-08 2022-09-14 Regeneron Pharmaceuticals, Inc. Crispr and aav strategies for x-linked juvenile retinoschisis therapy
CN111001044A (en) * 2019-12-30 2020-04-14 上海申淇医疗科技有限公司 Medicine balloon, preparation of medicine coated on medicine balloon and preparation method of medicine balloon
KR102152189B1 (en) 2020-02-11 2020-09-04 재단법인 아산사회복지재단 A recombinant vector for integration of T cell receptor into the targeted site in T cells and composition for integration of T cell receptor into the targeted site in T cells using the same
EP3872190A1 (en) 2020-02-26 2021-09-01 Antibodies-Online GmbH A method of using cut&run or cut&tag to validate crispr-cas targeting
JP2023522020A (en) * 2020-04-17 2023-05-26 ネバダ リサーチ アンド イノベイション コーポレイション CRISPR inhibition for facioscapulohumeral muscular dystrophy
AU2021267940A1 (en) 2020-05-08 2022-12-08 President And Fellows Of Harvard College Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
GB202007199D0 (en) * 2020-05-15 2020-07-01 Oxford Biomedica Ltd Viral vector production
EP4156913A1 (en) 2020-05-29 2023-04-05 KWS SAAT SE & Co. KGaA Plant haploid induction
GB2613486A (en) 2020-07-24 2023-06-07 Massachusetts Gen Hospital Enhanced virus-like particles and methods of use thereof for delivery to cells
CN112159809B (en) * 2020-09-22 2021-06-22 广州瑞风生物科技有限公司 gRNA targeting CTGF gene and its application
KR20230074534A (en) * 2020-09-24 2023-05-30 앨버트 아인슈타인 컬리지 오브 메디신 ATTB cell line, transgenic cell line derived therefrom, and method for producing the same
EP4001429A1 (en) 2020-11-16 2022-05-25 Antibodies-Online GmbH Analysis of crispr-cas binding and cleavage sites followed by high-throughput sequencing (abc-seq)
CN113057969A (en) * 2021-03-09 2021-07-02 吴志新 Clinical application experiment method of mesenchymal stem cells to infantile autism
CN113181376B (en) * 2021-03-17 2022-11-29 复旦大学附属眼耳鼻喉科医院 Application of Htra2 gene expression inhibitor in preventing acquired sensorineural deafness
CN113667734B (en) * 2021-07-16 2022-05-24 四川大学华西医院 Application of SHANK3 fragment sequence methylation detection reagent in preparation of schizophrenia diagnostic kit
CN118368974A (en) 2021-07-30 2024-07-19 科沃施种子欧洲股份两合公司 Plants with enhanced digestibility and marker haplotypes
CN117957317A (en) 2021-09-10 2024-04-30 安捷伦科技有限公司 Chemically modified guide RNAs for prime editing
MX2024003887A (en) 2021-10-14 2024-07-09 Arsenal Biosciences Inc Immune cells having co-expressed shrnas and logic gate systems.
AU2022399440A1 (en) * 2021-12-01 2024-06-20 Shape Therapeutics Inc. Functional aav capsids for intravitreal administration
WO2023143609A1 (en) * 2022-01-30 2023-08-03 Edigene Therapeutics (Beijing) Inc. Methods for nucleic acid editing to alter apoe4 function
CN115487315B (en) * 2022-04-20 2023-08-04 暨南大学 Drugs to treat Huntington's disease
CN115029346B (en) * 2022-04-22 2024-01-02 复旦大学附属眼耳鼻喉科医院 SgRNA, CRISPR/CasRx system for targeting knockdown Htra2 transcripts and application
WO2024042199A1 (en) 2022-08-26 2024-02-29 KWS SAAT SE & Co. KGaA Use of paired genes in hybrid breeding
WO2024112036A1 (en) * 2022-11-21 2024-05-30 고려대학교 산학협력단 Method for removing adapter dimer in high-efficiency adapter ligation reaction using cas9 protein
CN116515908B (en) * 2023-04-03 2024-04-26 苏州启辰生物科技有限公司 Method for establishing RPE65 gene editing model dog
WO2024236547A1 (en) 2023-05-18 2024-11-21 Inceptor Bio, Llc Modified phagocytic cells expressing chimeric antigen receptors comprising a herpes virus entry mediator (hvem) co-stimulatory domain and uses thereof
WO2024253957A1 (en) 2023-06-07 2024-12-12 Northwestern University Targeting autoimmunity-associated t cells through regulation of transcription factors
TW202515898A (en) * 2023-06-30 2025-04-16 日商武田藥品工業股份有限公司 Htt repressors and uses thereof
WO2025027136A1 (en) 2023-08-01 2025-02-06 ETH Zürich Single-cell crispr-screening of multiple gene perturbations in vivo
WO2025046513A1 (en) 2023-08-29 2025-03-06 Inceptor Bio, Llc Methods of manufacturing myeloid-derived cells from hematopoietic stem cells and compositions and uses thereof
WO2025196502A1 (en) 2024-03-20 2025-09-25 North Carolina Agricultural & Technical State University Choline kinase inhibitors as a therapeutic treatment for obesity
CN119745798A (en) * 2024-11-07 2025-04-04 河北中琨生物工程有限责任公司 Engineered umbilical cord stem cell exosomes carrying siCCR5, preparation method and application in the treatment of Alzheimer's disease

Family Cites Families (235)

* 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
DE3751873T2 (en) 1986-04-09 1997-02-13 Genzyme Corp Genetically transformed animals that secrete a desired protein in milk
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
US4873316A (en) 1987-06-23 1989-10-10 Biogen, Inc. Isolation of exogenous recombinant proteins from the milk of transgenic mammals
US5703055A (en) 1989-03-21 1997-12-30 Wisconsin Alumni Research Foundation Generation of antibodies through lipid mediated DNA delivery
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
WO1991017424A1 (en) 1990-05-03 1991-11-14 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
US7150982B2 (en) 1991-09-09 2006-12-19 Third Wave Technologies, Inc. RNA detection assays
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
US5593972A (en) 1993-01-26 1997-01-14 The Wistar Institute Genetic immunization
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US6007845A (en) 1994-07-22 1999-12-28 Massachusetts Institute Of Technology Nanoparticles and microparticles of non-linear hydrophilic-hydrophobic multiblock copolymers
US7745416B2 (en) 1995-04-11 2010-06-29 The Regents Of The University Of California Method for in vivo regulation of cardiac muscle contractility
JPH10510433A (en) 1995-06-06 1998-10-13 アイシス・ファーマシューティカルス・インコーポレーテッド Oligonucleotides with high chiral purity phosphorothioate linkages
US5985662A (en) 1995-07-13 1999-11-16 Isis Pharmaceuticals Inc. Antisense inhibition of hepatitis B virus replication
US5622856A (en) 1995-08-03 1997-04-22 Avigen High efficiency helper system for AAV vector production
US5855913A (en) 1997-01-16 1999-01-05 Massachusetts Instite Of Technology Particles incorporating surfactants for pulmonary drug delivery
US5985309A (en) 1996-05-24 1999-11-16 Massachusetts Institute Of Technology Preparation of particles for inhalation
US5846946A (en) 1996-06-14 1998-12-08 Pasteur Merieux Serums Et Vaccins Compositions and methods for administering Borrelia DNA
GB9907461D0 (en) 1999-03-31 1999-05-26 King S College London Neurite regeneration
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
GB9720465D0 (en) 1997-09-25 1997-11-26 Oxford Biomedica Ltd Dual-virus vectors
WO1999021977A1 (en) 1997-10-24 1999-05-06 Life Technologies, Inc. Recombinational cloning using nucleic acids having recombination sites
US6750059B1 (en) 1998-07-16 2004-06-15 Whatman, Inc. Archiving of vectors
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
GB0024550D0 (en) 2000-10-06 2000-11-22 Oxford Biomedica Ltd
US7033744B2 (en) 2001-03-16 2006-04-25 Naoya Kobayashi Method for proliferating a liver cell, a liver cell obtained thereby, and use thereof
WO2002080851A2 (en) 2001-04-05 2002-10-17 The Johns Hopkins University Chimeric vaccines
WO2003006477A1 (en) 2001-07-12 2003-01-23 University Of Massachusetts IN VIVO PRODUCTION OF SMALL INTERFERING RNAs THAT MEDIATE GENE SILENCING
WO2003014318A2 (en) 2001-08-08 2003-02-20 Genzyme Corporation Methods for treating diabetes and other blood sugar disorders
WO2003016338A1 (en) 2001-08-15 2003-02-27 Parker Hughes Institute Crystal structure of the btk kinase domain
WO2003045304A2 (en) 2001-09-26 2003-06-05 Mayo Foundation For Medical Education And Research Mutable vaccines
GB0125216D0 (en) 2001-10-19 2001-12-12 Univ Strathclyde Dendrimers for use in targeted delivery
CN1620508A (en) 2001-12-21 2005-05-25 牛津生物医学（英国）有限公司 Transgenic organism
US7206639B2 (en) 2002-03-15 2007-04-17 Sarcos Investments Lc Cochlear drug delivery system and method
WO2003087993A2 (en) 2002-04-09 2003-10-23 Beattie Kenneth L Oligonucleotide probes for genosensor chips
US20070020627A1 (en) 2002-06-11 2007-01-25 The Scripps Research Institute Artificial transcription factors
WO2004002453A1 (en) 2002-06-28 2004-01-08 Protiva Biotherapeutics Ltd. Method and apparatus for producing liposomes
GB0220467D0 (en) 2002-09-03 2002-10-09 Oxford Biomedica Ltd Composition
CA2499188A1 (en) 2002-09-27 2004-04-08 Cold Spring Harbor Laboratory Cell-based rna interference and related methods and compositions
WO2004046321A2 (en) 2002-11-15 2004-06-03 Trustees Of Boston University Cis/trans riboregulators
US20060178297A1 (en) 2003-01-28 2006-08-10 Troy Carol M Systems and methods for silencing expression of a gene in a cell and uses thereof
WO2005007814A2 (en) 2003-07-03 2005-01-27 The Regents Of The University Of California Genome mapping of functional dna elements and cellular proteins
EP2567693B1 (en) 2003-07-16 2015-10-21 Protiva Biotherapeutics Inc. Lipid encapsulated interfering RNA
DK2927318T3 (en) 2003-08-08 2020-08-03 Sangamo Therapeutics Inc Method and compositions for targeted cleavage and recombination
CA2551022C (en) 2003-09-15 2013-06-04 Protiva Biotherapeutics, Inc. Polyethyleneglycol-modified lipid compounds and uses thereof
GB0325379D0 (en) 2003-10-30 2003-12-03 Oxford Biomedica Ltd Vectors
US8673634B2 (en) 2003-11-13 2014-03-18 Massachusetts Eye & Ear Infirmary Method for the treatment of hearing loss
FR2862659B1 (en) 2003-11-21 2006-02-10 Pasteur Institut GENOME OF LEGIONELLA PNEUMOPHILA SOUCHE PARIS- DIAGNOSTIC AND EPIDEMIOLOGICAL APPLICATIONS
US8053232B2 (en) 2004-01-23 2011-11-08 Virxsys Corporation Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated RNA trans splicing
US20070244031A1 (en) 2004-01-27 2007-10-18 Quan Lu Methods and Compositions for Homozygous Gene Inactivation Using Collections of Pre-Defined Nucleotide Sequences Complementary Chromosomal Transcripts
WO2005072793A1 (en) 2004-01-29 2005-08-11 The Charles Stark Draper Laboratory, Inc. Implantable drug delivery apparatus
US20050220796A1 (en) 2004-03-31 2005-10-06 Dynan William S Compositions and methods for modulating DNA repair
ATE536418T1 (en) 2004-06-07 2011-12-15 Protiva Biotherapeutics Inc LIPID ENCAPSULATED INTERFERENCE RNA
US7745651B2 (en) 2004-06-07 2010-06-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
FR2872170B1 (en) 2004-06-25 2006-11-10 Centre Nat Rech Scient Cnrse NON-INTERACTIVE AND NON-REPLICATIVE LENTIVIRUS, PREPARATION AND USES
US20090214588A1 (en) 2004-07-16 2009-08-27 Nabel Gary J Vaccines against aids comprising cmv/r-nucleic acid constructs
GB0422877D0 (en) 2004-10-14 2004-11-17 Univ Glasgow Bioactive polymers
US20070081982A1 (en) 2005-04-28 2007-04-12 Elisabeth Evertsz Multiple RNAi expression cassettes for simultaneous delivery of RNAi agents related to heterozygotic expression patterns
US7892224B2 (en) 2005-06-01 2011-02-22 Brainlab Ag Inverse catheter planning
AU2006272634B2 (en) 2005-07-26 2013-01-24 Sangamo Therapeutics, Inc. Targeted integration and expression of exogenous nucleic acid sequences
DK2341149T3 (en) 2005-08-26 2017-02-27 Dupont Nutrition Biosci Aps Use of CRISPR-associated genes (Cas)
WO2007030743A2 (en) 2005-09-08 2007-03-15 Massachusetts Eye & Ear Infirmary Cochlear implants containing biological cells and uses thereof
US7838658B2 (en) 2005-10-20 2010-11-23 Ian Maclachlan siRNA silencing of filovirus gene expression
JP5043672B2 (en) 2005-10-28 2012-10-10 田辺三菱製薬株式会社 Novel cell membrane penetrating peptide
JP5336853B2 (en) 2005-11-02 2013-11-06 プロチバ バイオセラピューティクス インコーポレイティッド Modified siRNA molecules and methods of use thereof
GB0526211D0 (en) 2005-12-22 2006-02-01 Oxford Biomedica Ltd Viral vectors
EP1989307B1 (en) 2006-02-08 2012-08-08 Quark Pharmaceuticals, Inc. NOVEL TANDEM siRNAS
WO2007093836A1 (en) 2006-02-13 2007-08-23 Cellectis Meganuclease variants cleaving a dna target sequence from a xp gene and uses thereof
EP1994182B1 (en) 2006-03-15 2019-05-29 Siemens Healthcare Diagnostics Inc. Degenerate nucleobase analogs
CA2651839C (en) 2006-05-11 2016-02-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the pcsk9 gene
US8748567B2 (en) 2006-05-22 2014-06-10 Children's Medical Center Corporation Method for delivery across the blood brain barrier
US7915399B2 (en) 2006-06-09 2011-03-29 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
WO2008010009A1 (en) 2006-07-18 2008-01-24 Cellectis Meganuclease variants cleaving a dna target sequence from a rag gene and uses thereof
JP2008078613A (en) 2006-08-24 2008-04-03 Rohm Co Ltd Nitride semiconductor manufacturing method and nitride semiconductor device
JP5453097B2 (en) 2006-11-14 2014-03-26 セレクティス Meganuclease variants that cleave DNA target sequences from HPRT genes and uses thereof
WO2008093152A1 (en) 2007-02-01 2008-08-07 Cellectis Obligate heterodimer meganucleases and uses thereof
SI2126130T1 (en) 2007-03-02 2015-10-30 Dupont Nutrition Biosciences Aps Cultures with improved phage resistance
PE20090064A1 (en) 2007-03-26 2009-03-02 Novartis Ag DOUBLE-CHAIN RIBONUCLEIC ACID TO INHIBIT THE EXPRESSION OF THE HUMAN E6AP GENE AND THE PHARMACEUTICAL COMPOSITION THAT INCLUDES IT
WO2009013559A1 (en) 2007-07-23 2009-01-29 Cellectis Meganuclease variants cleaving a dna target sequence from the human hemoglobin beta gene and uses thereof
WO2009019528A1 (en) 2007-08-03 2009-02-12 Cellectis Meganuclease variants cleaving a dna target sequence from the human interleukin-2 receptor gamma chain gene and uses thereof
CN101801445B (en) 2007-08-14 2013-04-17 弗雷德哈钦森癌症研究中心 Needle Array Assembly for Delivery of Therapeutic Drugs
WO2009088786A1 (en) 2007-12-31 2009-07-16 Nanocor Therapeutics, Inc. Rna interference for the treatment of heart failure
CN101990433B (en) 2008-02-07 2014-11-05 马萨诸塞眼科耳科诊所 Compounds that enhance atoh-1 expression
WO2009105690A2 (en) 2008-02-21 2009-08-27 Targeted Genetics Corporation Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea
NZ588583A (en) 2008-04-15 2012-08-31 Protiva Biotherapeutics Inc Novel lipid formulations for nucleic acid delivery
EP2283029A1 (en) 2008-06-04 2011-02-16 Medical Research Council Peptides
JP5524189B2 (en) 2008-06-06 2014-06-18 クォーク ファーマシューティカルズ インコーポレーティッド Compositions and methods for the treatment of otic disorders
BRPI0910198A2 (en) 2008-06-30 2016-01-12 Silenseed Ltd A composition for administration from a local site of a nucleotide-based agent and method for treating a subject suffering from a disease requiring local treatment with a nucleotide-based agent.
WO2010004594A1 (en) 2008-07-08 2010-01-14 S.I.F.I. Societa' Industria Farmaceutica Italiana S.P.A. Ophthalmic compositions for treating pathologies of the posterior segment of the eye
WO2010011961A2 (en) 2008-07-25 2010-01-28 University Of Georgia Research Foundation, Inc. Prokaryotic rnai-like system and methods of use
WO2010014218A2 (en) 2008-07-29 2010-02-04 Academia Sinica Puf-a and related compounds for treatment of retinopathies and sight-threatening ophthalmologic disorders
WO2010026443A1 (en) 2008-09-08 2010-03-11 Cellectis Meganuclease variants cleaving a dna target sequence from a glutamine synthetase gene and uses thereof
WO2010030963A2 (en) 2008-09-15 2010-03-18 Children's Medical Center Corporation Modulation of bcl11a for treatment of hemoglobinopathies
US20100076057A1 (en) 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
AU2009311697B2 (en) 2008-10-29 2014-12-18 Sangamo Therapeutics, Inc. Methods and compositions for inactivating glutamine synthetase gene expression
US9404098B2 (en) 2008-11-06 2016-08-02 University Of Georgia Research Foundation, Inc. Method for cleaving a target RNA using a Cas6 polypeptide
CN104910025B (en) 2008-11-07 2019-07-16 麻省理工学院 Alkamine lipid and its purposes
US20110016540A1 (en) 2008-12-04 2011-01-20 Sigma-Aldrich Co. Genome editing of genes associated with trinucleotide repeat expansion disorders in animals
US20110023139A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in cardiovascular disease
US20110023153A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in alzheimer's disease
US20110023146A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in secretase-associated disorders
US20120159653A1 (en) 2008-12-04 2012-06-21 Sigma-Aldrich Co. Genomic editing of genes involved in macular degeneration
SG172760A1 (en) 2008-12-04 2011-08-29 Sangamo Biosciences Inc Genome editing in rats using zinc-finger nucleases
US20110023144A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in amyotrophyic lateral sclerosis disease
US20110023145A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in autism spectrum disorders
CA2746514C (en) 2008-12-10 2018-11-27 Alnylam Pharmaceuticals, Inc. Gnaq targeted dsrna compositions and methods for inhibiting expression
WO2010075424A2 (en) 2008-12-22 2010-07-01 The Regents Of University Of California Compositions and methods for downregulating prokaryotic genes
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
US20110239315A1 (en) 2009-01-12 2011-09-29 Ulla Bonas Modular dna-binding domains and methods of use
EP2408921B1 (en) 2009-03-20 2017-04-19 Sangamo BioSciences, Inc. Modification of cxcr4 using engineered zinc finger proteins
RU2556376C2 (en) 2009-04-07 2015-07-10 ДАУ АГРОСАЙЕНСИЗ ЭлЭлСи Nanoparticle mediated delivery of sequence specific nucleases
EP2427577A4 (en) 2009-05-04 2013-10-23 Hutchinson Fred Cancer Res PSEUDOTYPED COCAL VIRUS ENVELOPE RETROVIRAL VECTORS (VESICULOVIRUS)
WO2011036510A1 (en) 2009-09-24 2011-03-31 Cellectis Meganuclease variants cleaving the genome of the herpes simplex virus and uses thereof
WO2010143917A2 (en) 2009-06-11 2010-12-16 Toolgen Incorporation Targeted genomic rearrangements using site-specific nucleases
US8236943B2 (en) 2009-07-01 2012-08-07 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein B
CA2767127A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
AU2010275432A1 (en) 2009-07-24 2012-02-02 Sigma-Aldrich Co. Llc. Method for genome editing
CA2769262C (en) 2009-07-28 2019-04-30 Sangamo Biosciences, Inc. Methods and compositions for treating trinucleotide repeat disorders
US8927807B2 (en) 2009-09-03 2015-01-06 The Regents Of The University Of California Nitrate-responsive promoter
US8889394B2 (en) 2009-09-07 2014-11-18 Empire Technology Development Llc Multiple domain proteins
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
CN102725412B (en) 2009-11-27 2017-09-22 巴斯夫植物科学有限公司 Optimized endonucleases and uses thereof
SG181601A1 (en) 2009-12-10 2012-07-30 Univ Minnesota Tal effector-mediated dna modification
CA2784547A1 (en) 2009-12-23 2011-06-30 Max-Planck-Gesellschaft Zur Foerderung Der Wissenschaften E.V. Influenza targets
JP2013518602A (en) 2010-02-09 2013-05-23 サンガモ バイオサイエンシーズ， インコーポレイテッド Targeted genome modification by partially single-stranded donor molecules
US10087431B2 (en) 2010-03-10 2018-10-02 The Regents Of The University Of California Methods of generating nucleic acid fragments
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
EP3078753B1 (en) 2010-05-10 2018-04-18 The Regents of The University of California Methods using endoribonuclease compositions
EP2569435A1 (en) 2010-05-12 2013-03-20 Cellectis Meganuclease variants cleaving a dna target sequence from the rhodopsin gene and uses thereof
US8372951B2 (en) 2010-05-14 2013-02-12 National Tsing Hua University Cell penetrating peptides for intracellular delivery
EP2571512B1 (en) 2010-05-17 2017-08-23 Sangamo BioSciences, Inc. Novel dna-binding proteins and uses thereof
JP5965392B2 (en) 2010-05-28 2016-08-03 オックスフォード バイオメディカ （ユーケー） リミテッド Delivery of lentiviral vectors to the brain
KR101971741B1 (en) 2010-07-23 2019-08-14 시그마-알드리치 컴퍼니., 엘엘씨 Genome editing using targeting endonucleases and single-stranded nucleic acids
EP2601611B1 (en) 2010-08-02 2020-12-09 Integrated Dna Technologies, Inc. Methods for predicting stability and melting temperatures of nucleic acid duplexes
EP2609135A4 (en) 2010-08-26 2015-05-20 Massachusetts Inst Technology POLY (BETA-AMINO ALCOHOLS), THEIR PREPARATION AND USES THEREOF
WO2012031205A2 (en) 2010-09-03 2012-03-08 The Brigham And Women's Hospital, Inc. Lipid-polymer hybrid particles
BR112013008881B1 (en) * 2010-10-12 2021-12-07 The Children's Hospital Of Philadelphia NON-NATURALLY OCCURRING PROTEIN, ZINC FINGER NUCLEASE, AND COMPOSITION
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
US20120204282A1 (en) 2011-02-04 2012-08-09 Sangamo Biosciences, Inc. Methods and compositions for treating occular disorders
EP2691443B1 (en) 2011-03-28 2021-02-17 Massachusetts Institute of Technology Conjugated lipomers and uses thereof
DE12722942T1 (en) 2011-03-31 2021-09-30 Modernatx, Inc. RELEASE AND FORMULATION OF MANIPULATED NUCLEIC ACIDS
PT3693025T (en) * 2011-04-22 2022-01-20 Univ California Adeno-associated virus virions with variant capsid and methods of use thereof
CA2834375C (en) 2011-04-27 2020-07-14 Amyris, Inc. Methods for genomic modification
US20120295960A1 (en) 2011-05-20 2012-11-22 Oxford Biomedica (Uk) Ltd. Treatment regimen for parkinson's disease
US20140113376A1 (en) 2011-06-01 2014-04-24 Rotem Sorek Compositions and methods for downregulating prokaryotic genes
KR102061557B1 (en) * 2011-09-21 2020-01-03 상가모 테라퓨틱스, 인코포레이티드 Methods and compositions for refulation of transgene expression
JP2014530603A (en) 2011-10-06 2014-11-20 サンガモ バイオサイエンシーズ， インコーポレイテッド Methods and compositions for controlling HIV infection
JP6188703B2 (en) 2011-10-27 2017-08-30 サンガモ セラピューティクス， インコーポレイテッド Methods and compositions for modifying the HPRT locus
EP2591770B1 (en) 2011-11-14 2016-03-16 Silenseed Ltd Compositions for siRNA delivery and methods of manufacturing and using same
CA2855653A1 (en) 2011-11-18 2013-05-23 Universite Laval Methods and products for increasing frataxin levels and uses thereof
HK1202434A1 (en) 2011-11-22 2015-10-02 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
US8450107B1 (en) 2011-11-30 2013-05-28 The Broad Institute Inc. Nucleotide-specific recognition sequences for designer TAL effectors
CA3018046A1 (en) 2011-12-16 2013-06-20 Moderna Therapeutics, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
BR112014020625A2 (en) 2012-02-24 2017-07-04 Hutchinson Fred Cancer Res polynucleotide, polypeptide, composition, cell, and stem cell edited by genome
CN108285491B (en) 2012-02-29 2021-08-10 桑格摩生物科学股份有限公司 Methods and compositions for treating huntington's disease
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
AU2013204327B2 (en) 2012-04-20 2016-09-01 Aviagen Cell transfection method
FI3597749T3 (en) * 2012-05-25 2023-10-09 Univ California METHODS AND COMPOSITIONS FOR RNA-DIRECTED MODIFICATION OF TARGET DNA AND RNA-DIRECTED MODULATION OF TRANSCRIPTION
US9623071B2 (en) 2012-06-01 2017-04-18 Drexel University Modulation of hepatitis B virus CCCDNA transcription
JP2015527889A (en) 2012-07-25 2015-09-24 ザ ブロード インスティテュート， インコーポレイテッド Inducible DNA binding protein and genomic disruption tools and their applications
US8614194B1 (en) 2012-07-25 2013-12-24 Kaohsiung Medical University Anionic cell penetrating peptide and its use for intracellular delivery
JP6401704B2 (en) 2012-10-10 2018-10-10 サンガモ セラピューティクス， インコーポレイテッド Compounds that modify T cells and uses thereof
EP4397760A3 (en) 2012-10-23 2024-10-09 Toolgen Incorporated Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
ES2714154T3 (en) 2012-12-06 2019-05-27 Sigma Aldrich Co Llc Modification and regulation of the genome based on CRISPR
WO2014093479A1 (en) 2012-12-11 2014-06-19 Montana State University Crispr (clustered regularly interspaced short palindromic repeats) rna-guided control of gene regulation
ES2576126T3 (en) 2012-12-12 2016-07-05 The Broad Institute, Inc. Modification by genetic technology and optimization of improved enzyme systems, methods and compositions for sequence manipulation
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
WO2014093655A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
EP3705490B1 (en) 2012-12-12 2024-03-06 The Broad Institute, Inc. Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
EP4279588A3 (en) 2012-12-12 2024-01-17 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
WO2014093709A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
JP2016505256A (en) 2012-12-12 2016-02-25 ザ・ブロード・インスティテュート・インコーポレイテッ CRISPR-Cas component system, method and composition for sequence manipulation
IL293526A (en) 2012-12-12 2022-08-01 Harvard College Providing, engineering and optimizing systems, methods and compositions for sequence manipulation and therapeutic applications
IL239326B2 (en) 2012-12-17 2025-02-01 Harvard College Rna-guided human genome engineering
CA2898184A1 (en) 2013-01-16 2014-07-24 Emory University Cas9-nucleic acid complexes and uses related thereto
US11135273B2 (en) 2013-02-07 2021-10-05 The Rockefeller University Sequence specific antimicrobials
US10660943B2 (en) 2013-02-07 2020-05-26 The Rockefeller University Sequence specific antimicrobials
US9163837B2 (en) 2013-02-27 2015-10-20 Siemens Aktiengesellschaft Flow conditioner in a combustor of a gas turbine engine
BR112015022061B8 (en) 2013-03-14 2023-12-26 Caribou Biosciences Inc Genetically engineered single-stranded guide nucleic acid targeting nucleic acid, polynucleotide, method for cleaving a target nucleic acid and for ligating a target nucleic acid, composition and kit
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
CN113563476A (en) 2013-03-15 2021-10-29 通用医疗公司 RNA-guided targeting of genetic and epigenetic regulatory proteins to specific genomic loci
WO2014154269A1 (en) 2013-03-27 2014-10-02 Wilco Ag Method of inline inspecting and/or testing devices and apparatus to perform such method
EP2981612B1 (en) 2013-04-04 2019-07-03 Trustees of Dartmouth College Compositions and methods for in vivo excision of hiv-1 proviral dna
CA2910489A1 (en) 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition
AU2014273082B2 (en) 2013-05-29 2018-11-08 Cellectis A method for producing precise DNA cleavage using Cas9 nickase activity
EP4159243A1 (en) 2013-06-04 2023-04-05 President and Fellows of Harvard College Rna-guided transcriptional regulation
US9267135B2 (en) 2013-06-04 2016-02-23 President And Fellows Of Harvard College RNA-guided transcriptional regulation
AU2014274840B2 (en) * 2013-06-05 2020-03-12 Duke University RNA-guided gene editing and gene regulation
KR20160056869A (en) 2013-06-17 2016-05-20 더 브로드 인스티튜트, 인코퍼레이티드 Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
RU2716420C2 (en) 2013-06-17 2020-03-11 Те Брод Инститьют Инк. Delivery and use of systems of crispr-cas, vectors and compositions for targeted action and therapy in liver
JP6738729B2 (en) * 2013-06-17 2020-08-12 ザ・ブロード・インスティテュート・インコーポレイテッド Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling postmitotic cell diseases and disorders
EP3011030B1 (en) * 2013-06-17 2023-11-08 The Broad Institute, Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
WO2014204727A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
EP3011029B1 (en) * 2013-06-17 2019-12-11 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
CN103343120B (en) 2013-07-04 2015-03-04 中国科学院遗传与发育生物学研究所 Wheat genome site-specific modification method
PT3019619T (en) 2013-07-11 2021-11-11 Modernatx Inc COMPOSITIONS COMPRISING SYNTHETIC POLYNUCLEOTIDES ENCODING SYNTHETIC CRISPR AND SGARN-RELATED PROTEINS AND METHODS OF USE
CN103388006B (en) 2013-07-26 2015-10-28 华东师范大学 A kind of construction process of site-directed point mutation
EA037850B1 (en) 2013-08-29 2021-05-27 Тэмпл Юниверсити Оф Зе Коммонвэлс Систем Оф Хайе Эдьюкейшн Methods and compositions for rna-guided treatment of hiv infection
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
WO2015048577A2 (en) 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions
US10822606B2 (en) 2013-09-27 2020-11-03 The Regents Of The University Of California Optimized small guide RNAs and methods of use
WO2015065964A1 (en) 2013-10-28 2015-05-07 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof
JP2016536021A (en) 2013-11-07 2016-11-24 エディタス・メディシン，インコーポレイテッド CRISPR-related methods and compositions with governing gRNA
WO2015071474A2 (en) 2013-11-18 2015-05-21 Crispr Therapeutics Ag Crispr-cas system materials and methods
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
JP2017503485A (en) 2013-12-12 2017-02-02 ザ・ブロード・インスティテュート・インコーポレイテッド CRISPR-CAS system and method for altering gene product expression, structural information, and inducible modular CAS enzyme
KR20160089526A (en) 2013-12-12 2016-07-27 더 브로드 인스티튜트, 인코퍼레이티드 Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
AU2014362245A1 (en) 2013-12-12 2016-06-16 Massachusetts Institute Of Technology Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders
CN103668472B (en) * 2013-12-31 2014-12-24 北京大学 Method for constructing eukaryon gene knockout library by using CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats)/Cas9 system
WO2015113063A1 (en) 2014-01-27 2015-07-30 Georgia Tech Research Corporation Methods and systems for identifying crispr/cas off-target sites
JP6323228B2 (en) 2014-07-18 2018-05-16 富士電機株式会社 Power converter
US9932566B2 (en) 2014-08-07 2018-04-03 Agilent Technologies, Inc. CIS-blocked guide RNA
DK3180426T3 (en) 2014-08-17 2020-03-30 Broad Inst Inc RETURNING BY USING CAS9 NICKASES
EP3265559B1 (en) 2015-03-03 2021-01-06 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
KR102138209B1 (en) 2015-05-06 2020-07-28 스니프르 테크놀로지스 리미티드 Microbial population change and microbial population modification
JP7107683B2 (en) 2015-06-18 2022-07-27 ザ・ブロード・インスティテュート・インコーポレイテッド CRISPR enzyme mutations that reduce off-target effects
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
JP6186470B2 (en) 2016-04-20 2017-08-23 パイオニア株式会社 Acoustic device, volume control method, volume control program, and recording medium

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