SG11201808920RA - Genome editing of human neural stem cells using nucleases - Google Patents

Genome editing of human neural stem cells using nucleases

Info

Publication number
SG11201808920RA
SG11201808920RA SG11201808920RA SG11201808920RA SG11201808920RA SG 11201808920R A SG11201808920R A SG 11201808920RA SG 11201808920R A SG11201808920R A SG 11201808920RA SG 11201808920R A SG11201808920R A SG 11201808920RA SG 11201808920R A SG11201808920R A SG 11201808920RA
Authority
SG
Singapore
Prior art keywords
stanford
neural stem
international
stem cells
human neural
Prior art date
Application number
SG11201808920RA
Other languages
English (en)
Inventor
Matthew Porteus
Eric Kildebeck
Daniel Dever
Joseph Clark
Ann Tsukamoto
Nobuko Uchida
Original Assignee
Boco Silicon Valley Inc
Univ Leland Stanford Junior
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 Boco Silicon Valley Inc, Univ Leland Stanford Junior filed Critical Boco Silicon Valley Inc
Publication of SG11201808920RA publication Critical patent/SG11201808920RA/en

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/30—Nerves; Brain; Eyes; Corneal cells; Cerebrospinal fluid; Neuronal stem cells; Neuronal precursor cells; Glial cells; Oligodendrocytes; Schwann cells; Astroglia; Astrocytes; Choroid plexus; Spinal cord tissue
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Cell Biology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Developmental Biology & Embryology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Mycology (AREA)
  • Epidemiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Psychology (AREA)
  • Pain & Pain Management (AREA)
SG11201808920RA 2016-04-14 2017-04-13 Genome editing of human neural stem cells using nucleases SG11201808920RA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662322652P 2016-04-14 2016-04-14
PCT/US2017/027504 WO2017180926A1 (en) 2016-04-14 2017-04-13 Genome editing of human neural stem cells using nucleases

Publications (1)

Publication Number Publication Date
SG11201808920RA true SG11201808920RA (en) 2018-11-29

Family

ID=60037918

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11201808920RA SG11201808920RA (en) 2016-04-14 2017-04-13 Genome editing of human neural stem cells using nucleases

Country Status (8)

Country Link
US (2) US20170298348A1 (zh)
EP (1) EP3443085B1 (zh)
JP (1) JP7197363B2 (zh)
CN (1) CN109312308A (zh)
AU (2) AU2017250683A1 (zh)
CA (1) CA3020181A1 (zh)
SG (1) SG11201808920RA (zh)
WO (1) WO2017180926A1 (zh)

Families Citing this family (29)

* 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
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
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US20150166982A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting pi3k point mutations
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
IL294014B1 (en) 2015-10-23 2024-03-01 Harvard College Nucleobase editors and their uses
AU2017306676B2 (en) 2016-08-03 2024-02-22 President And Fellows Of Harvard College Adenosine nucleobase editors and uses thereof
CA3033327A1 (en) 2016-08-09 2018-02-15 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
CN110214180A (zh) 2016-10-14 2019-09-06 哈佛大学的校长及成员们 核碱基编辑器的aav递送
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
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
KR20190130613A (ko) 2017-03-23 2019-11-22 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
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
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
US11795443B2 (en) 2017-10-16 2023-10-24 The Broad Institute, Inc. Uses of adenosine base editors
EP3942040A1 (en) 2019-03-19 2022-01-26 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
CN110075319A (zh) * 2019-04-29 2019-08-02 白涛 一种抗遗传病生物导弹
EP4103177A4 (en) * 2020-02-11 2024-03-27 University of Virginia Patent Foundation COMPOSITIONS AND METHODS FOR THE TREATMENT AND/OR PREVENTION OF EYE DISEASES
MX2022014008A (es) 2020-05-08 2023-02-09 Broad Inst Inc Métodos y composiciones para la edición simultánea de ambas cadenas de una secuencia de nucleótidos de doble cadena objetivo.
KR20230019156A (ko) * 2020-06-02 2023-02-07 카탈렌트 파마 솔루션즈, 엘엘씨 유전자 삽입을 위한 다수의 도크들이 있는 세포주
CN111925998A (zh) * 2020-06-09 2020-11-13 广州再生医学与健康广东省实验室 模拟SARS-CoV-2感染的系统及其制备方法与应用
WO2022163770A1 (ja) * 2021-01-28 2022-08-04 国立研究開発法人理化学研究所 ゲノム編集ツールの評価方法
WO2023102712A1 (zh) * 2021-12-07 2023-06-15 深圳市先康达生命科学有限公司 一种基因生物制剂及其制备方法和应用

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7217565B2 (en) 1999-02-12 2007-05-15 Stemcells California, Inc. Enriched central nervous system stem cell and progenitor cell populations, and methods for identifying, isolating and enriching for such populations
US7037719B1 (en) 1999-02-12 2006-05-02 Stemcells California, Inc. Enriched central nervous system stem cell and progenitor cell populations, and methods for identifying, isolating and enriching for such populations
US7105150B2 (en) 1999-02-12 2006-09-12 Stemcells California, Inc. In vivo screening methods using enriched neural stem cells
EP1540348A4 (en) 2002-08-27 2006-10-11 Stemcells California Inc ENRICHED CELLS OF STEM CELLS AND PROGENITORS OF THE CENTRAL NERVOUS SYSTEM, AND METHODS OF IDENTIFYING, ISOLATING AND ENRICHING SUCH POPULATIONS
WO2013158309A2 (en) * 2012-04-18 2013-10-24 The Board Of Trustees Of The Leland Stanford Junior University Non-disruptive gene targeting
PT2800811T (pt) 2012-05-25 2017-08-17 Univ California Métodos e composições para modificação de adn alvo dirigida por arn e para modulação dirigida por arn de transcrição
ES2769310T3 (es) * 2012-12-06 2020-06-25 Sigma Aldrich Co Llc Modificación y regulación del genoma basada en CRISPR
EP4279588A3 (en) 2012-12-12 2024-01-17 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
CA2926078C (en) 2013-10-17 2021-11-16 Sangamo Biosciences, Inc. Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells
CA2930590C (en) * 2013-11-15 2021-02-16 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Engineering neural stem cells using homologous recombination
WO2015188065A1 (en) * 2014-06-05 2015-12-10 Sangamo Biosciences, Inc. Methods and compositions for nuclease design

Also Published As

Publication number Publication date
CA3020181A1 (en) 2017-10-19
EP3443085B1 (en) 2022-09-14
JP7197363B2 (ja) 2022-12-27
US20230250423A1 (en) 2023-08-10
US20170298348A1 (en) 2017-10-19
AU2023208065A1 (en) 2023-08-17
WO2017180926A1 (en) 2017-10-19
EP3443085A4 (en) 2019-09-25
EP3443085A1 (en) 2019-02-20
AU2017250683A1 (en) 2018-11-01
CN109312308A (zh) 2019-02-05
JP2019511240A (ja) 2019-04-25

Similar Documents

Publication Publication Date Title
SG11201808920RA (en) Genome editing of human neural stem cells using nucleases
SG11201811292RA (en) Compositions and methods for the depletion of cells
SG11201807187XA (en) Binding members to pd-l1
SG11201900138TA (en) Programmed death 1 ligand 1 (pd-l1) binding proteins and methods of use thereof
SG11201811719RA (en) Conjugates for targeted cell surface editing
SG11201901996UA (en) Formulations of ( r)-2-amino-3-phenylpropyl carbamate
SG11201901757YA (en) Pd-1 homing endonuclease variants, compositions, and methods of use
SG11201909949XA (en) Targeted immunotolerance
SG11201908719QA (en) Biomarkers and car t cell therapies with enhanced efficacy
SG11201811431VA (en) Multispecific antibodies against cd40 and cd137
SG11201809627QA (en) Anti pd-1 and anti-lag3 antibodies for cancer treatment
SG11201809710RA (en) Genetically engineered cells and methods of making the same
SG11201811432WA (en) Rna for cancer therapy
SG11201804375WA (en) Compositions and methods for internalizing enzymes
SG11201807252QA (en) Anti-lag-3 antibodies
SG11201909203WA (en) Tissue selective transgene expression
SG11201808909WA (en) Ex vivo bite-activated t cells
SG11201408405WA (en) Engineered three-dimensional connective tissue constructs and methods of making the same
SG11201805680SA (en) Methods and compositions for the treatment of neurologic disease
SG11201811251RA (en) Multi-chamber deposition equipment for solid free form fabrication
SG11201809751XA (en) Egfr inhibitor compounds
SG11201807593TA (en) Compositions and methods to program therapeutic cells using targeted nucleic acid nanocarriers
SG11201807041PA (en) Improved preparations of adult liver progenitor cells
SG11201807827VA (en) Process for the preparation of pegylated drug-linkers and intermediates thereof
SG11201806400XA (en) Replicative transposon system