MX374399B - Composiciones y sus usos para tratar y prevenir la enfermedad de huntington. - Google Patents

Composiciones y sus usos para tratar y prevenir la enfermedad de huntington.

Info

Publication number
MX374399B
MX374399B MX2018009444A MX2018009444A MX374399B MX 374399 B MX374399 B MX 374399B MX 2018009444 A MX2018009444 A MX 2018009444A MX 2018009444 A MX2018009444 A MX 2018009444A MX 374399 B MX374399 B MX 374399B
Authority
MX
Mexico
Prior art keywords
disease
compositions
treating
preventing huntington
huntington
Prior art date
Application number
MX2018009444A
Other languages
English (en)
Inventor
Edward J Rebar
H Steve Zhang
Jeffrey C Miller
Original Assignee
Sangamo Biosciences Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Sangamo Biosciences Inc filed Critical Sangamo Biosciences Inc
Publication of MX374399B publication Critical patent/MX374399B/es

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702Regulators; Modulating activity
    • C07K14/4703Inhibitors; Suppressors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/70Fusion polypeptide containing domain for protein-protein interaction
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/70Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/71Fusion polypeptide containing domain for protein-protein interaction containing domain for transcriptional activaation, e.g. VP16
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/80Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Psychology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Se describen composiciones y sus usos para tratar y prevenir la enfermedad de Huntington.
MX2018009444A 2012-02-29 2013-02-28 Composiciones y sus usos para tratar y prevenir la enfermedad de huntington. MX374399B (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261605028P 2012-02-29 2012-02-29
PCT/US2013/028348 WO2013130824A1 (en) 2012-02-29 2013-02-28 Methods and compositions for treating huntington's disease

Publications (1)

Publication Number Publication Date
MX374399B true MX374399B (es) 2025-03-06

Family

ID=49083290

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2018009444A MX374399B (es) 2012-02-29 2013-02-28 Composiciones y sus usos para tratar y prevenir la enfermedad de huntington.
MX2014010455A MX359327B (es) 2012-02-29 2013-02-28 Composiciones y sus usos para tratar y prevenir la enfermedad de huntington.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2014010455A MX359327B (es) 2012-02-29 2013-02-28 Composiciones y sus usos para tratar y prevenir la enfermedad de huntington.

Country Status (14)

Country Link
US (7) US8841260B2 (es)
EP (1) EP2820159B1 (es)
JP (3) JP6490426B2 (es)
KR (1) KR102084539B1 (es)
CN (2) CN108285491B (es)
AU (1) AU2013225950B2 (es)
BR (1) BR112014021104B1 (es)
CA (1) CA2865011C (es)
HK (1) HK1203566A1 (es)
MX (2) MX374399B (es)
NZ (1) NZ629427A (es)
RU (1) RU2639277C2 (es)
WO (1) WO2013130824A1 (es)
ZA (1) ZA201406209B (es)

Families Citing this family (188)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2734621B1 (en) 2011-07-22 2019-09-04 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
EP3604555B1 (en) 2011-10-14 2024-12-25 President and Fellows of Harvard College Sequencing by structure assembly
WO2014163886A1 (en) 2013-03-12 2014-10-09 President And Fellows Of Harvard College Method of generating a three-dimensional nucleic acid containing matrix
ES2991004T3 (es) 2011-12-22 2024-12-02 Harvard College Métodos para la detección de analitos
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
NZ629427A (en) 2012-02-29 2016-04-29 Sangamo Biosciences Inc Methods and compositions for treating huntington’s disease
PL4289948T3 (pl) 2012-05-25 2025-06-02 The Regents Of The University Of California Sposoby i kompozycje do modyfikacji kierowanego na rna docelowego dna i do nakierowanej na rna modulacji transkrypcji
CN105142669B (zh) 2012-12-06 2018-07-03 西格马-奥尔德里奇有限责任公司 基于crispr的基因组修饰和调控
WO2014093701A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
EP3434776A1 (en) 2012-12-12 2019-01-30 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
CN105658796B (zh) 2012-12-12 2021-10-26 布罗德研究所有限公司 用于序列操纵的crispr-cas组分系统、方法以及组合物
WO2014093622A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
EP4282970A3 (en) 2012-12-17 2024-01-17 President and Fellows of Harvard College Rna-guided human genome engineering
KR20170108172A (ko) 2013-03-14 2017-09-26 카리부 바이오사이언시스 인코포레이티드 핵산-표적화 핵산의 조성물 및 방법
US10295547B2 (en) 2013-03-14 2019-05-21 University Of Florida Research Foundation, Incorporated Use and treatment of di-amino acid repeat-containing proteins associated with ALS
US11332719B2 (en) * 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
US10760064B2 (en) 2013-03-15 2020-09-01 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
IL289396B2 (en) 2013-03-15 2023-12-01 The General Hospital Coporation Using truncated guide rnas (tru-grnas) to increase specificity for rna-guided genome editing
JP2016522679A (ja) 2013-04-04 2016-08-04 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 CRISPR/Cas系を用いたゲノム編集の治療的使用
MY197877A (en) 2013-06-04 2023-07-22 Harvard College Rna-guided transcriptional regulation
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
SG11201510327TA (en) 2013-06-17 2016-01-28 Broad Inst Inc Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
EP3597755A1 (en) 2013-06-17 2020-01-22 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
EP3011033B1 (en) 2013-06-17 2020-02-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
KR20240172759A (ko) 2013-06-17 2024-12-10 더 브로드 인스티튜트, 인코퍼레이티드 간의 표적화 및 치료를 위한 CRISPR­Cas 시스템, 벡터 및 조성물의 전달 및 용도
CA2915837A1 (en) 2013-06-17 2014-12-24 The Broad Institute, Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
EP3674411A1 (en) 2013-06-17 2020-07-01 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
US10011850B2 (en) 2013-06-21 2018-07-03 The General Hospital Corporation Using RNA-guided FokI Nucleases (RFNs) to increase specificity for RNA-Guided Genome Editing
EP3019595A4 (en) 2013-07-09 2016-11-30 THERAPEUTIC USES OF A GENERIC CHANGE WITH CRISPR / CAS SYSTEMS
AU2014287397B2 (en) 2013-07-10 2019-10-10 President And Fellows Of Harvard College Orthogonal Cas9 proteins for RNA-guided gene regulation and editing
US11306328B2 (en) 2013-07-26 2022-04-19 President And Fellows Of Harvard College Genome engineering
US9163284B2 (en) 2013-08-09 2015-10-20 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
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
AU2014338910B2 (en) * 2013-10-25 2020-12-10 Cellectis Design of rare-cutting endonucleases for efficient and specific targeting DNA sequences comprising highly repetitive motives
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 (ja) 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関連方法および支配gRNAのある組成物
NZ719477A (en) 2013-11-11 2022-05-27 Sangamo Therapeutics Inc Methods and compositions for treating huntington’s disease
US10787684B2 (en) 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
US9074199B1 (en) 2013-11-19 2015-07-07 President And Fellows Of Harvard College Mutant Cas9 proteins
EP2878667A1 (en) * 2013-11-29 2015-06-03 Institut Pasteur TAL effector means useful for partial or full deletion of DNA tandem repeats
EP3080259B1 (en) 2013-12-12 2023-02-01 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
JP2017501149A (ja) 2013-12-12 2017-01-12 ザ・ブロード・インスティテュート・インコーポレイテッド 粒子送達構成成分を用いた障害及び疾患の標的化のためのcrispr−cas系及び組成物の送達、使用及び治療適用
EP3080260B1 (en) 2013-12-12 2019-03-06 The Broad Institute, Inc. Crispr-cas systems and methods for altering expression of gene products, structural information and inducible modular cas enzymes
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
EP3080271B1 (en) 2013-12-12 2020-02-12 The Broad Institute, Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
WO2015089354A1 (en) * 2013-12-12 2015-06-18 The Broad Institute Inc. Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
CN105899658B (zh) 2013-12-12 2020-02-18 布罗德研究所有限公司 针对hbv和病毒性疾病以及障碍的crispr-cas系统和组合物的递送、用途和治疗应用
EP3653229A1 (en) 2013-12-12 2020-05-20 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing
EP3105325B2 (en) 2014-02-13 2024-10-23 Takara Bio USA, Inc. Methods of depleting a target molecule from an initial collection of nucleic acids, and compositions and kits for practicing the same
EP3957735A1 (en) 2014-03-05 2022-02-23 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
ES2745769T3 (es) 2014-03-10 2020-03-03 Editas Medicine Inc Procedimientos y composiciones relacionados con CRISPR/CAS para tratar la amaurosis congénita de Leber 10 (LCA10)
ES2879373T3 (es) * 2014-03-18 2021-11-22 Sangamo Therapeutics Inc Métodos y composiciones para la regulación de la expresión de proteínas de dedo de zinc
WO2015148863A2 (en) 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
US12460231B2 (en) 2014-04-02 2025-11-04 Editas Medicine, Inc. Crispr/CAS-related methods and compositions for treating primary open angle glaucoma
MX378789B (es) * 2014-05-08 2025-03-11 Sangamo Biosciences Inc Métodos y composiciones para tratar la enfermedad de huntington.
AU2015259191B2 (en) 2014-05-13 2019-03-21 Sangamo Therapeutics, Inc. Methods and compositions for prevention or treatment of a disease
EP3152319A4 (en) 2014-06-05 2017-12-27 Sangamo BioSciences, Inc. Methods and compositions for nuclease design
WO2016007839A1 (en) 2014-07-11 2016-01-14 President And Fellows Of Harvard College Methods for high-throughput labelling and detection of biological features in situ using microscopy
WO2016019144A2 (en) 2014-07-30 2016-02-04 Sangamo Biosciences, Inc. Gene correction of scid-related genes in hematopoietic stem and progenitor cells
CA2956224A1 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
EP2982758A1 (en) 2014-08-04 2016-02-10 Centre Hospitalier Universitaire Vaudois (CHUV) Genome editing for the treatment of huntington's disease
AU2015308910B2 (en) 2014-08-27 2017-12-07 Caribou Biosciences, Inc. Methods for increasing Cas9-mediated engineering efficiency
KR102455807B1 (ko) 2014-10-01 2022-10-18 더 제너럴 하스피탈 코포레이션 뉴클레아제-유도 상동성-지정 복구의 효율 증가 방법
GB201418965D0 (es) 2014-10-24 2014-12-10 Ospedale San Raffaele And Fond Telethon
US12180263B2 (en) 2014-11-06 2024-12-31 President And Fellows Of Harvard College Cells lacking B2M surface expression and methods for allogeneic administration of such cells
MX2017006217A (es) 2014-11-14 2018-05-02 Voyager Therapeutics Inc Polinucleotidos moduladores.
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
EP3889260A1 (en) 2014-12-12 2021-10-06 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
CA2970370A1 (en) 2014-12-24 2016-06-30 Massachusetts Institute Of Technology Crispr having or associated with destabilization domains
SG11201706444TA (en) 2015-02-10 2017-09-28 Genzyme Corp VARIANT RNAi
EP4019975A1 (en) 2015-04-24 2022-06-29 Editas Medicine, Inc. Evaluation of cas9 molecule/guide rna molecule complexes
US10179918B2 (en) 2015-05-07 2019-01-15 Sangamo Therapeutics, Inc. Methods and compositions for increasing transgene activity
CN107921148A (zh) 2015-05-08 2018-04-17 哈佛学院校长同事会 通用供体干细胞和相关方法
US10509045B2 (en) 2015-05-29 2019-12-17 University Of Florida Research Foundation, Incorporated Methods for diagnosing Huntington's disease
WO2016205759A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
MX392008B (es) 2015-06-18 2025-03-21 Broad Inst Inc Mutaciones de la enzima crispr que reducen los efectos fuera del blanco
WO2016205749A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
US9580727B1 (en) 2015-08-07 2017-02-28 Caribou Biosciences, Inc. Compositions and methods of engineered CRISPR-Cas9 systems using split-nexus Cas9-associated polynucleotides
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US10435441B2 (en) * 2015-09-23 2019-10-08 Sangamo Therapeutics, Inc. HTT repressors and uses thereof
WO2017070598A1 (en) 2015-10-23 2017-04-27 Caribou Biosciences, Inc. Engineered crispr class 2 cross-type nucleic-acid targeting nucleic acids
EP4434589A3 (en) 2015-10-23 2025-05-14 President and Fellows of Harvard College Evolved cas9 proteins for gene editing
EP3371329B1 (en) 2015-11-03 2026-03-25 President and Fellows of Harvard College Method and apparatus for volumetric imaging of a three-dimensional nucleic acid containing matrix
CN116763942A (zh) 2016-02-18 2023-09-19 宾州研究基金会 脑内生成gaba能神经元
WO2017165862A1 (en) 2016-03-25 2017-09-28 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
EP3440100A4 (en) 2016-04-04 2020-01-22 University of Florida Research Foundation, Incorporated HANDLING EIF3 TO MODULATE A NON-ATG TRANSLATION ASSOCIATED WITH A REPETITION (RAN)
CA3210120C (en) 2016-04-25 2024-04-09 President And Fellows Of Harvard College Hybridization chain reaction methods for in situ molecular detection
BR112018073472A2 (pt) 2016-05-18 2019-08-27 Voyager Therapeutics, Inc. composições e métodos de tratamento da doença de huntington
AU2017267665C1 (en) 2016-05-18 2023-10-05 Voyager Therapeutics, Inc. Modulatory polynucleotides
AU2017295720B2 (en) 2016-07-13 2021-07-22 Vertex Pharmaceuticals Incorporated Methods, compositions and kits for increasing genome editing efficiency
CA3032822A1 (en) 2016-08-02 2018-02-08 Editas Medicine, Inc. Compositions and methods for treating cep290 associated disease
JP7231935B2 (ja) 2016-08-03 2023-03-08 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 アデノシン核酸塩基編集因子およびそれらの使用
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
GB2569252A (en) 2016-08-31 2019-06-12 Harvard College Methods of combining the detection of biomolecules into a single assay using fluorescent in situ sequencing
WO2018049009A2 (en) * 2016-09-07 2018-03-15 Sangamo Therapeutics, Inc. Modulation of liver genes
AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of Harvard College AAV delivery of nucleobase editors
KR102674612B1 (ko) 2016-12-01 2024-06-14 상가모 테라퓨틱스, 인코포레이티드 Tau 조절제 및 그것의 전달을 위한 방법 및 조성물
US9816093B1 (en) 2016-12-06 2017-11-14 Caribou Biosciences, Inc. Engineered nucleic acid-targeting nucleic acids
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
US11268093B2 (en) 2017-01-03 2022-03-08 Rula Zain-Luqman Therapeutic method for huntington's disease
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
CN110662556A (zh) 2017-03-09 2020-01-07 哈佛大学的校长及成员们 癌症疫苗
JP2020510439A (ja) 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 シトシンからグアニンへの塩基編集因子
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
WO2018176009A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
AU2018255293B2 (en) 2017-04-17 2024-03-07 University Of Florida Research Foundation, Incorporated Regulation of RAN translation by PKR and eIF2a-P pathways
US12058986B2 (en) 2017-04-20 2024-08-13 Egenesis, Inc. Method for generating a genetically modified pig with inactivated porcine endogenous retrovirus (PERV) elements
CN111108198A (zh) 2017-05-05 2020-05-05 沃雅戈治疗公司 治疗亨廷顿病的组合物和方法
WO2018209158A2 (en) 2017-05-10 2018-11-15 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
AU2018291041B2 (en) 2017-06-30 2021-08-05 Inscripta, Inc. Automated cell processing methods, modules, instruments, and systems
US11732274B2 (en) 2017-07-28 2023-08-22 President And Fellows Of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE)
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
MY205041A (en) * 2017-09-22 2024-09-29 Genzyme Corp Variant rnai
US12504380B2 (en) 2017-09-25 2025-12-23 University Of Florida Research Foundation, Incorporated Immunoassays for detection of ran proteins
CA3076214A1 (en) 2017-09-26 2019-04-04 University Of Florida Research Foundation, Incorporated Use of metformin and analogs thereof to reduce ran protein levels in the treatment of neurological disorders
US10435713B2 (en) 2017-09-30 2019-10-08 Inscripta, Inc. Flow through electroporation instrumentation
KR20200121782A (ko) 2017-10-16 2020-10-26 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
EA202091709A1 (ru) 2018-01-17 2020-11-10 Вертекс Фармасьютикалз Инкорпорейтед Ингибиторы днк-пк
ES2940786T3 (es) 2018-01-17 2023-05-11 Vertex Pharma Compuestos de quinoxalinona, composiciones, métodos y kits para aumentar la eficiencia de edición del genoma
BR112020014315A2 (pt) 2018-01-17 2020-12-08 Vertex Pharmaceuticals Incorporated Inibidores de dna-pk
WO2019147743A1 (en) 2018-01-26 2019-08-01 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
EP3765614A1 (en) 2018-03-14 2021-01-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
WO2019204457A1 (en) 2018-04-18 2019-10-24 Sangamo Therapeutics, Inc. Zinc finger protein compositions for modulation of huntingtin (htt)
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
CN116144653A (zh) * 2018-06-21 2023-05-23 陕西杆粒生物科技有限公司 一种抗凋亡的杆状病毒表达载体
US12264181B2 (en) 2018-06-27 2025-04-01 Altius Institute For Biomedical Sciences Nucleic acid binding domains and methods of use thereof
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
MX2021001395A (es) 2018-08-03 2021-08-11 Genzyme Corp Arni variante contra alfa-sinucleína.
WO2020041380A1 (en) 2018-08-20 2020-02-27 The Broad Institute, Inc. Methods and compositions for optochemical control of crispr-cas9
US20200109406A1 (en) * 2018-10-02 2020-04-09 Sangamo Therapeutics, Inc. Engineered genetic modulators
EP3860618B1 (en) 2018-10-02 2026-02-04 Sangamo Therapeutics, Inc. Compositions for treating tauopathy
JP7555123B2 (ja) 2018-10-09 2024-09-24 ザ ユニヴァーシティ オブ ブリティッシュ コロンビア 有機溶媒不含かつ劣化剤不含のトランスフェクション・コンピテント・ベシクルを含む組成物及びシステム並びにそれらに関連する方法
WO2020076976A1 (en) 2018-10-10 2020-04-16 Readcoor, Inc. Three-dimensional spatial molecular indexing
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
CN113544266A (zh) 2018-12-17 2021-10-22 博德研究所 Crispr相关转座酶系统和其使用方法
US12264323B2 (en) 2018-12-17 2025-04-01 The Broad Institute, Inc. CRISPR CPF1 direct repeat variants
CN113301909B (zh) * 2019-01-15 2025-01-28 桑格摩生物治疗股份有限公司 Htt阻遏物及其应用
WO2020154500A1 (en) 2019-01-23 2020-07-30 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
MX2021011426A (es) 2019-03-19 2022-03-11 Broad Inst Inc Metodos y composiciones para editar secuencias de nucleótidos.
CN113950526B (zh) * 2019-04-09 2025-03-21 加利福尼亚大学董事会 通过靶向体内表观遗传阻遏实现的持久镇痛
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
PE20212332A1 (es) 2019-04-23 2021-12-14 Sangamo Therapeutics Inc Moduladores de la expresioon del gen de marco de lectura abierto 72 del cromosoma 9 y usos de los mismos
BR112021026381A2 (pt) * 2019-06-28 2022-02-08 Penn State Res Found Métodos e materiais para tratamento da doença de huntington
JP2022543112A (ja) 2019-08-01 2022-10-07 サナ バイオテクノロジー,インコーポレイテッド Dux4発現細胞およびそれらの使用
KR20220068222A (ko) 2019-08-23 2022-05-25 사나 바이오테크놀로지, 인크. Cd24 발현 세포 및 이의 용도
AU2020348916B2 (en) 2019-09-20 2025-12-18 University Of Florida Research Foundation, Incorporated Detection of antibodies against RAN proteins from serum and tissue lysates
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
AU2020368539A1 (en) 2019-10-16 2022-04-28 Massachusetts Institute Of Technology Engineered muscle targeting compositions
CA3159620A1 (en) 2019-11-01 2021-05-06 Sangamo Therapeutics, Inc. Compositions and methods for genome engineering
CN119499394A (zh) * 2019-11-01 2025-02-25 阿尔尼拉姆医药品有限公司 亨廷顿(HTT)iRNA药剂组合物及其使用方法
WO2021087358A1 (en) 2019-11-01 2021-05-06 Sangamo Therapeutics, Inc. Gin recombinase variants
WO2021155063A1 (en) 2020-01-29 2021-08-05 Readcoor, Llc Compositions and methods for analyte detection
US20230293593A1 (en) 2020-03-25 2023-09-21 Sana Biotechnology, Inc. Hypoimmunogenic neural cells for the treatment of neurological disorders and conditions
WO2021226558A1 (en) 2020-05-08 2021-11-11 The Broad Institute, Inc. Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
US20230190871A1 (en) 2020-05-20 2023-06-22 Sana Biotechnology, Inc. Methods and compositions for treatment of viral infections
IL300516A (en) 2020-08-13 2023-04-01 Sana Biotechnology Inc Methods for treating susceptible patients with hypoimmunogenic cells, and related methods and compounds
US20220049303A1 (en) 2020-08-17 2022-02-17 Readcoor, Llc Methods and systems for spatial mapping of genetic variants
RU2748997C1 (ru) * 2020-12-11 2021-06-02 Федеральное государственное автономное образовательное учреждение высшего образования "Российский национальный исследовательский медицинский университет имени Н.И. Пирогова" Министерства здравоохранения Российской Федерации (ФГАОУ ВО РНИМУ им. Н.И. Пирогова Минздрава России) Универсальный эпигеномный репрессор транскрипции целевых генов в клетках человека
JP2024501971A (ja) 2020-12-31 2024-01-17 サナ バイオテクノロジー,インコーポレイテッド Car-t活性を調節するための方法及び組成物
US20240226164A1 (en) 2021-05-27 2024-07-11 Sana Biotechnology, Inc. Hypoimmunogenic cells comprising engineered hla-e or hla-g
AU2022309875A1 (en) 2021-07-14 2024-01-25 Sana Biotechnology, Inc. Altered expression of y chromosome-linked antigens in hypoimmunogenic cells
KR20240073006A (ko) 2021-08-11 2024-05-24 사나 바이오테크놀로지, 인크. 동종이계 세포 요법을 위한 유전자 변형된 1차 세포
AU2022325231A1 (en) 2021-08-11 2024-02-08 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy to reduce complement-mediated inflammatory reactions
WO2023019226A1 (en) 2021-08-11 2023-02-16 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy
AU2022325955A1 (en) 2021-08-11 2024-02-08 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy to reduce instant blood mediated inflammatory reactions
WO2023019203A1 (en) 2021-08-11 2023-02-16 Sana Biotechnology, Inc. Inducible systems for altering gene expression in hypoimmunogenic cells
EP4419117A1 (en) 2021-10-22 2024-08-28 Sana Biotechnology, Inc. Methods of engineering allogeneic t cells with a transgene in a tcr locus and associated compositions and methods
CN119072319A (zh) 2021-12-23 2024-12-03 萨那生物技术股份有限公司 用于治疗自身免疫性疾病的嵌合抗原受体(car)t细胞及相关方法
US20250302953A1 (en) 2022-02-14 2025-10-02 Sana Biotechnology, Inc. Methods of treating patients exhibiting a prior failed therapy with hypoimmunogenic cells
AU2023220128A1 (en) 2022-02-17 2024-08-22 Sana Biotechnology, Inc. Engineered cd47 proteins and uses thereof
WO2023173123A1 (en) 2022-03-11 2023-09-14 Sana Biotechnology, Inc. Genetically modified cells and compositions and uses thereof
CN115786395A (zh) * 2022-08-19 2023-03-14 南京启真基因工程有限公司 构建htt基因突变的亨廷顿舞蹈症模型猪核移植供体细胞的基因编辑系统及其应用
WO2024151541A1 (en) 2023-01-09 2024-07-18 Sana Biotechnology, Inc. Type-1 diabetes autoimmune mouse
EP4704867A1 (en) 2023-05-03 2026-03-11 Sana Biotechnology, Inc. Methods of dosing and administration of engineered islet cells
WO2024243236A2 (en) 2023-05-22 2024-11-28 Sana Biotechnology, Inc. Methods of delivery of islet cells and related methods
WO2025004001A1 (en) 2023-06-30 2025-01-02 Takeda Pharmaceutical Company Limited Htt repressors and uses thereof
WO2025106739A1 (en) * 2023-11-14 2025-05-22 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of htt expression

Family Cites Families (103)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US789538A (en) 1904-11-11 1905-05-09 Colin E Ham Dumb-bell.
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
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
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
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
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
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (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
US5789538A (en) 1995-02-03 1998-08-04 Massachusetts Institute Of Technology Zinc finger proteins with high affinity new DNA binding specificities
US5928638A (en) 1996-06-17 1999-07-27 Systemix, Inc. Methods for gene transfer
US5925523A (en) 1996-08-23 1999-07-20 President & Fellows Of Harvard College Intraction trap assay, reagents and uses thereof
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
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
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
US6534261B1 (en) * 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
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
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
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
US20070060606A1 (en) 1999-10-07 2007-03-15 Robertson Harold A Compounds and methods for modulating phosphodiesterase 10A
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
DE60126483T2 (de) 2000-04-28 2007-12-06 Sangamo BioSciences, Inc., Richmond Gezielte Modifikation der Chromatinstruktur
WO2001083732A2 (en) 2000-04-28 2001-11-08 Sangamo Biosciences, Inc. Databases of regulatory sequences; methods of making and using same
AU2001263155A1 (en) 2000-05-16 2001-11-26 Massachusetts Institute Of Technology Methods and compositions for interaction trap assays
EP1303608A2 (en) * 2000-07-21 2003-04-23 Syngenta Participations AG Zinc finger domain recognition code and uses thereof
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
US6919204B2 (en) 2000-09-29 2005-07-19 Sangamo Biosciences, Inc. Modulation of gene expression using localization domains
WO2002044376A2 (en) 2000-11-28 2002-06-06 Sangamo Biosciences, Inc. Modulation of gene expression using insulator binding proteins
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
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
US8206965B2 (en) 2002-03-15 2012-06-26 Cellectis S.A. Hybrid and single chain meganucleases and use thereof
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
WO2004067753A2 (en) 2003-01-28 2004-08-12 Cellectis Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application thereof.
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
KR100535326B1 (ko) 2004-01-20 2005-12-09 한국생명공학연구원 줄기 세포로부터 자연살해 세포로의 분화 조절용 유전자를유효성분으로 포함하는 분화 조절제
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
US7358085B2 (en) * 2005-02-28 2008-04-15 Sangamo Biosciences, Inc. Anti-angiogenic methods and compositions
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
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
CN101365801B (zh) * 2005-10-28 2013-03-27 阿尔尼拉姆医药品有限公司 抑制亨廷顿基因表达的组合物和方法
EP2018424A2 (en) 2006-05-19 2009-01-28 Sangamo BioSciences, Inc. Methods and compositions for inactivation of dihydrofolate reductase
DE602007005634D1 (de) 2006-05-25 2010-05-12 Sangamo Biosciences Inc Variante foki-spaltungshälften-domänen
EP2019839B1 (en) 2006-05-25 2011-12-07 Sangamo BioSciences, Inc. Methods and compositions for gene inactivation
US9187758B2 (en) 2006-12-14 2015-11-17 Sangamo Biosciences, Inc. Optimized non-canonical zinc finger proteins
SI2412812T1 (sl) * 2006-12-14 2015-12-31 Dow Agrosciences Llc Optimizirani nekanonični proteini s cinkovim prstom
DE602007005629D1 (de) 2007-06-18 2010-05-12 Commissariat Energie Atomique Reversibles siRNA-Silencing eines mutierten und endogenen Huntington-Wildtypgens und dessen Anwendung zur Behandlung von Morbus Huntington
DK2167523T3 (da) 2007-06-19 2014-09-08 Univ Louisiana State Syntese og anvendelse af anti-reverse phosphorothioatanaloger af messenger-rna-cappen
CN101883863B (zh) 2007-09-27 2018-01-23 桑格摩生物科学股份有限公司 生物活性核酸酶的快速体内鉴定
KR101666228B1 (ko) 2007-09-28 2016-10-13 인트렉손 코포레이션 생물치료학적 분자를 발현시키기 위한 치료학적 유전자-스위치 작제물 및 생물반응기, 및 이의 용도
DE102007056956B4 (de) 2007-11-27 2009-10-29 Moosbauer, Peter, Dipl.-Ing.(FH) Schaltung zur Regelung der Stromversorgung eines Verbrauchers und Verfahren zum Betrieb einer Schaltung
JP5763530B2 (ja) 2008-06-10 2015-08-12 サンガモ バイオサイエンシーズ, インコーポレイテッド Bax−およびBak−欠損細胞株の生成のための方法および組成物
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
US9234016B2 (en) * 2009-07-28 2016-01-12 Sangamo Biosciences, Inc. Engineered zinc finger proteins for treating trinucleotide repeat disorders
DK2534173T3 (da) 2010-02-08 2019-10-14 Sangamo Therapeutics Inc Manipulerede spaltnings-halvdomæner
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
EP2441774B1 (en) * 2010-10-15 2018-12-12 Fundació Centre de Regulació Genòmica Peptides having zinc finger domains and uses thereof
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
US20130137173A1 (en) * 2011-11-30 2013-05-30 Feng Zhang Nucleotide-specific recognition sequences for designer tal effectors
NZ629427A (en) 2012-02-29 2016-04-29 Sangamo Biosciences Inc Methods and compositions for treating huntington’s disease

Also Published As

Publication number Publication date
CN104204225A (zh) 2014-12-10
EP2820159B1 (en) 2019-10-23
AU2013225950A1 (en) 2014-09-18
ZA201406209B (en) 2016-01-27
EP2820159A4 (en) 2015-08-26
CA2865011A1 (en) 2013-09-06
JP2015509958A (ja) 2015-04-02
US10471123B2 (en) 2019-11-12
CN108285491B (zh) 2021-08-10
KR102084539B1 (ko) 2020-03-04
US9499597B2 (en) 2016-11-22
US20190183970A1 (en) 2019-06-20
US10857203B2 (en) 2020-12-08
US10265377B2 (en) 2019-04-23
AU2013225950B2 (en) 2018-02-15
RU2639277C2 (ru) 2017-12-20
JP6490426B2 (ja) 2019-03-27
NZ629427A (en) 2016-04-29
US20200030408A1 (en) 2020-01-30
KR20140131973A (ko) 2014-11-14
EP2820159A1 (en) 2015-01-07
JP2017143835A (ja) 2017-08-24
MX2014010455A (es) 2015-02-10
US20130253040A1 (en) 2013-09-26
JP2019022531A (ja) 2019-02-14
HK1203566A1 (zh) 2015-10-30
BR112014021104A2 (pt) 2017-07-04
US8841260B2 (en) 2014-09-23
BR112014021104B1 (pt) 2023-03-28
CN108285491A (zh) 2018-07-17
RU2014138989A (ru) 2016-04-20
US11723952B2 (en) 2023-08-15
US20170035839A1 (en) 2017-02-09
US20210060124A1 (en) 2021-03-04
MX359327B (es) 2018-09-25
CA2865011C (en) 2021-06-15
US20140336133A1 (en) 2014-11-13
WO2013130824A1 (en) 2013-09-06
US20230414710A1 (en) 2023-12-28

Similar Documents

Publication Publication Date Title
MX374399B (es) Composiciones y sus usos para tratar y prevenir la enfermedad de huntington.
MX2015003140A (es) Formulaciones de enzalutamida.
BR112016026378A2 (pt) formulações e métodos para tratamento com queratina
MX2017013562A (es) Reguladores de microbioma y usos relacionados de los mismos.
EP3547968A4 (en) PROSTHESIS OBTAINED BY TISSUE ENGINEERING
MX358514B (es) Compuestos para tratar la atrofia muscular espinal.
TR201813877T4 (tr) Spi̇nal müsküler atrofi̇ni̇n tedavi̇ edi̇lmesi̇ne yöneli̇k bi̇leşi̇kler.
UY34484A (es) Benzotienilo-pirrolotriazinas disustituidas y sus usos
PL3145522T3 (pl) Kombinacja, kompozycja i sposób podawania kombinacji lub kompozycji zwierzętom
UY34750A (es) ?compuestos para el tratamiento del hiv, composiciones,métodos de preparación, intermediarios y métodos terapéuticos?.
AR091959A1 (es) Agentes de sosten sinteticos y agentes de sosten monodispersos y metodos para la elaboracion de los mismos
CR20120202A (es) Métodos y composiciones para tratar cáncer
UY34210A (es) Composiciones, métodos y sistemas para la micropropagación de plantas
UY35427A (es) Compuestos y sus usos en la modulación de la hemoglobina
MX379016B (es) Composiciones y metodos para tratar enfermedad de gaucher.
DK3010506T3 (da) Pridopidin i behandling af huntingtons sygdom
UY35425A (es) Compuestos y sus usos en la modulación de la hemoglobina
UY35426A (es) Compuestos y sus usos en la modulación de la hemoglobina
UY34165A (es) ?compuestos aldh-2 mitocondriales humanos para el tratamiento de la adicción?.
MX359769B (es) Metodos para descelularizar huesos.
UY34725A (es) Nuevos derivados de tienopirimidina, procesos para su preparación y sus usos terapéuticos.
BR112015006512A2 (pt) composição para tratar tecidos biológicos danificados
MX2015003701A (es) Composiciones para tratamiento.
BR112014030159A2 (pt) agente para tratamento de floculação.
BR112014031512A2 (pt) composição cosmética e método para o tratamento cosmético.