CA3020181A1 - Genome editing of human neural stem cells using nucleases - Google Patents

Genome editing of human neural stem cells using nucleases Download PDF

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Publication number
CA3020181A1
CA3020181A1 CA3020181A CA3020181A CA3020181A1 CA 3020181 A1 CA3020181 A1 CA 3020181A1 CA 3020181 A CA3020181 A CA 3020181A CA 3020181 A CA3020181 A CA 3020181A CA 3020181 A1 CA3020181 A1 CA 3020181A1
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Prior art keywords
neural stem
dna
stem cells
transgene
stem cell
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Pending
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CA3020181A
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English (en)
French (fr)
Inventor
Matthew H. PORTEUS
Eric J. Kildebeck
Daniel P. DEVER
Joseph T. Clark
Ann Tsukamoto
Nobuko Uchida
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Leland Stanford Junior University
Bright Ocens Silicon Valley Inc
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Leland Stanford Junior University
Bright Ocens Silicon Valley Inc
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Publication of CA3020181A1 publication Critical patent/CA3020181A1/en
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    • 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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
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    • 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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/30Nerves; Brain; Eyes; Corneal cells; Cerebrospinal fluid; Neuronal stem cells; Neuronal precursor cells; Glial cells; Oligodendrocytes; Schwann cells; Astroglia; Astrocytes; Choroid plexus; Spinal cord tissue
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P25/00Drugs for disorders of the nervous system
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P25/00Drugs for disorders of the nervous system
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs 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
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    • 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
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications

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CA3020181A 2016-04-14 2017-04-13 Genome editing of human neural stem cells using nucleases Pending CA3020181A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662322652P 2016-04-14 2016-04-14
US62/322,652 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
CA3020181A1 true CA3020181A1 (en) 2017-10-19

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CA3020181A Pending CA3020181A1 (en) 2016-04-14 2017-04-13 Genome editing of human neural stem cells using nucleases

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

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US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
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WO2018031683A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
KR102622411B1 (ko) 2016-10-14 2024-01-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
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WO2017180926A1 (en) 2017-10-19
AU2023208065A1 (en) 2023-08-17
EP3443085B1 (en) 2022-09-14
JP2019511240A (ja) 2019-04-25
JP7197363B2 (ja) 2022-12-27
US20170298348A1 (en) 2017-10-19
AU2017250683A1 (en) 2018-11-01
EP3443085A4 (en) 2019-09-25
SG11201808920RA (en) 2018-11-29
US20230250423A1 (en) 2023-08-10
EP3443085A1 (en) 2019-02-20
CN109312308A (zh) 2019-02-05

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