AU2019236288B2 - Methods and compositions for inducible expression of neurotrophic factors - Google Patents
Methods and compositions for inducible expression of neurotrophic factors Download PDFInfo
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- AU2019236288B2 AU2019236288B2 AU2019236288A AU2019236288A AU2019236288B2 AU 2019236288 B2 AU2019236288 B2 AU 2019236288B2 AU 2019236288 A AU2019236288 A AU 2019236288A AU 2019236288 A AU2019236288 A AU 2019236288A AU 2019236288 B2 AU2019236288 B2 AU 2019236288B2
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- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/65—Tetracyclines
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- 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
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- A61K38/00—Medicinal preparations containing peptides
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- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/18—Growth factors; Growth regulators
- A61K38/185—Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
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- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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- 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
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- 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
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- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/475—Growth factors; Growth regulators
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- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/475—Growth factors; Growth regulators
- C07K14/4756—Neuregulins, i.e. p185erbB2 ligands, glial growth factor, heregulin, ARIA, neu differentiation factor
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- 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
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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
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- C12N5/0602—Vertebrate cells
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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
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- C12N5/0619—Neurons
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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0618—Cells of the nervous system
- C12N5/0623—Stem cells
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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/10—Cells modified by introduction of foreign genetic material
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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- C12N2506/00—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells
- C12N2506/45—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells from artificially induced pluripotent stem cells
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
- C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
- C12N2830/003—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor tet inducible
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/20—Vector systems having a special element relevant for transcription transcription of more than one cistron
- C12N2830/205—Vector systems having a special element relevant for transcription transcription of more than one cistron bidirectional
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- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Cell Biology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
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- Proteomics, Peptides & Aminoacids (AREA)
- Biophysics (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Developmental Biology & Embryology (AREA)
- Psychology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
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Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862644332P | 2018-03-16 | 2018-03-16 | |
| US62/644,332 | 2018-03-16 | ||
| US201862773752P | 2018-11-30 | 2018-11-30 | |
| US62/773,752 | 2018-11-30 | ||
| PCT/US2019/022595 WO2019178550A1 (en) | 2018-03-16 | 2019-03-15 | Methods and compositions for inducible expression of neurotrophic factors |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| AU2019236288A1 AU2019236288A1 (en) | 2020-09-17 |
| AU2019236288B2 true AU2019236288B2 (en) | 2025-06-05 |
Family
ID=67907321
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2019236288A Active AU2019236288B2 (en) | 2018-03-16 | 2019-03-15 | Methods and compositions for inducible expression of neurotrophic factors |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US12258571B2 (https=) |
| EP (1) | EP3765058A4 (https=) |
| JP (2) | JP7535944B2 (https=) |
| KR (1) | KR102843346B1 (https=) |
| CN (1) | CN111867617A (https=) |
| AU (1) | AU2019236288B2 (https=) |
| WO (1) | WO2019178550A1 (https=) |
Families Citing this family (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20190018000A1 (en) | 2016-01-12 | 2019-01-17 | Cedars-Sinai Medical Center | A method of non destructive monitoring of biological processes in microfluidic tissue culture systems |
| CA3012042A1 (en) | 2016-01-26 | 2017-08-03 | Cedars-Sinai Medical Center | Systems and methods for in vivo dual recombinase-mediated cassette exchange (drmce) and disease models thereof |
| WO2018140647A1 (en) | 2017-01-25 | 2018-08-02 | Cedars-Sinai Medical Center | In vitro induction of mammary-like differentiation from human pluripotent stem cells |
| US11767513B2 (en) | 2017-03-14 | 2023-09-26 | Cedars-Sinai Medical Center | Neuromuscular junction |
| US11414648B2 (en) | 2017-03-24 | 2022-08-16 | Cedars-Sinai Medical Center | Methods and compositions for production of fallopian tube epithelium |
| US12258571B2 (en) | 2018-03-16 | 2025-03-25 | Cedars-Sinai Medical Center | Methods and compositions for inducible expression of neurotrophic factors |
| EP4700387A2 (en) | 2018-03-23 | 2026-02-25 | Cedars-Sinai Medical Center | Methods of use of islet cells |
| EP3775161A4 (en) | 2018-04-06 | 2022-04-06 | Cedars-Sinai Medical Center | NEURODEGENERATIVE DISEASE MODELS DERIVED FROM HUMAN PLURIPOTENTIC STEM CELLS ON A MICROFLUIDIC CHIP |
| US11981918B2 (en) | 2018-04-06 | 2024-05-14 | Cedars-Sinai Medical Center | Differentiation technique to generate dopaminergic neurons from induced pluripotent stem cells |
| EP3787613B1 (en) | 2018-04-30 | 2026-03-11 | Cedars-Sinai Medical Center | Pkc activation for use in treating early onset sporadic parkinson's disease |
| WO2020084168A1 (en) * | 2018-10-26 | 2020-04-30 | Icm (Institut Du Cerveau Et De La Moelle Épinière) | Tetracycline compound for treating drug-induced dyskinesia |
| AU2020370288B2 (en) * | 2019-10-22 | 2026-02-12 | Cedars-Sinai Medical Center | Cortical neural progenitor cells from iPSCs |
| US20230047254A1 (en) * | 2019-12-29 | 2023-02-16 | Gloriana Therapeutics | Mammalian Cells Secreting GDNF and Their Therapeutic Use |
| KR20220061403A (ko) * | 2020-11-06 | 2022-05-13 | (주) 에스바이오메딕스 | 다능성 줄기세포 유래 신경전구세포를 포함하는 퇴행성 뇌질환 치료용 약학적 조성물 |
| CN121420065A (zh) * | 2023-06-29 | 2026-01-27 | 香港大学 | 用于在肝脏或肝癌中的可控转录的方法 |
| GB202312699D0 (en) * | 2023-08-18 | 2023-10-04 | Cambridge Entpr Ltd | Controllable expression in mammalian cells |
| WO2025091023A1 (en) * | 2023-10-27 | 2025-05-01 | Northwestern University | Engineered cell transfected by all-in-one vector for therapeutic agents production and delivery and applications thereof |
Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20030228295A1 (en) * | 2002-04-25 | 2003-12-11 | Svendsen Clive N. | Use of human neural stem cells secreting GDNF for treatment of parkinson's and other neurodegenerative diseases |
Family Cites Families (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7575924B2 (en) | 2000-11-13 | 2009-08-18 | Research Development Foundation | Methods and compositions relating to improved lentiviral vectors and their applications |
| US20030084468A1 (en) | 2001-09-05 | 2003-05-01 | Economides Aris N. | Methods of expressing transgenes |
| GB2404382B (en) | 2003-07-28 | 2008-01-30 | Oxitec Ltd | Pest control |
| US20120107284A1 (en) * | 2006-06-30 | 2012-05-03 | Elena Kozlova | Stem cells for transplantation and methods for production thereof |
| US20100077495A1 (en) | 2006-12-21 | 2010-03-25 | Davis David P | Compositions and methods for the expression of nucleic acids |
| US8592211B2 (en) * | 2009-03-20 | 2013-11-26 | The Rockefeller University | Enhanced PiggyBac transposon and methods for transposon mutagenesis |
| US20120107938A1 (en) | 2009-06-29 | 2012-05-03 | University Of Basel | Methods and kits for high efficiency engineering of conditional mouse alleles |
| WO2013155222A2 (en) * | 2012-04-10 | 2013-10-17 | The Regents Of The University Of California | Brain-specific enhancers for cell-based therapy |
| CN102816793B (zh) * | 2012-08-22 | 2014-09-10 | 陕西师范大学 | 单载体双向启动的Tet-on诱导表达系统及其构建方法和应用 |
| JP2016508520A (ja) | 2013-02-15 | 2016-03-22 | インターナショナル ステム セル コーポレイション | 神経変性疾患の治療のための、ヒト多能性幹細胞に由来する神経細胞の使用 |
| US10519421B2 (en) | 2013-03-21 | 2019-12-31 | Kyoto University | Induction of motor neurons from pluripotent stem cells |
| WO2014204724A1 (en) | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation |
| WO2015143342A1 (en) | 2014-03-21 | 2015-09-24 | Cellular Dynamics International, Inc. | Production of midbrain dopaminergic neurons and methods for the use thereof |
| CA2943296C (en) * | 2014-03-21 | 2022-10-18 | The Sydney Children's Hospitals Network (Randwick And Westmead) (Incorporating The Royal Alexandra Hospital For Children) | Stable gene transfer to proliferating cells |
| EP3122370A1 (en) | 2014-03-28 | 2017-02-01 | Buck Institute for Research on Aging | Methods and compositions for modulating the immune system |
| AU2015279552B2 (en) | 2014-06-27 | 2021-01-21 | Angiocrine Bioscience, Inc. | Neural cells expressing adenovirus E4ORF1, and methods of making and using the same |
| JP6928604B2 (ja) * | 2015-11-04 | 2021-09-01 | フェイト セラピューティクス,インコーポレイテッド | 万能性細胞のゲノム改変 |
| CA3012042A1 (en) | 2016-01-26 | 2017-08-03 | Cedars-Sinai Medical Center | Systems and methods for in vivo dual recombinase-mediated cassette exchange (drmce) and disease models thereof |
| US10221395B2 (en) | 2016-06-16 | 2019-03-05 | Cedars-Sinai Medical Center | Efficient method for reprogramming blood to induced pluripotent stem cells |
| US12258571B2 (en) | 2018-03-16 | 2025-03-25 | Cedars-Sinai Medical Center | Methods and compositions for inducible expression of neurotrophic factors |
| KR20220024527A (ko) | 2019-06-17 | 2022-03-03 | 세다르스-신나이 메디칼 센터 | 생체내 이중 재조합효소-매개된 카세트 교환 (dRMCE)을 위한 시스템 및 방법 그리고 이들의 질환 모델 |
-
2019
- 2019-03-15 US US16/979,640 patent/US12258571B2/en active Active
- 2019-03-15 JP JP2020549702A patent/JP7535944B2/ja active Active
- 2019-03-15 EP EP19768063.0A patent/EP3765058A4/en active Pending
- 2019-03-15 KR KR1020207029729A patent/KR102843346B1/ko active Active
- 2019-03-15 AU AU2019236288A patent/AU2019236288B2/en active Active
- 2019-03-15 CN CN201980019683.6A patent/CN111867617A/zh active Pending
- 2019-03-15 WO PCT/US2019/022595 patent/WO2019178550A1/en not_active Ceased
-
2024
- 2024-03-25 JP JP2024047652A patent/JP2024073654A/ja active Pending
Patent Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20030228295A1 (en) * | 2002-04-25 | 2003-12-11 | Svendsen Clive N. | Use of human neural stem cells secreting GDNF for treatment of parkinson's and other neurodegenerative diseases |
Non-Patent Citations (1)
| Title |
|---|
| Sareen, et al., "Human Neural Progenitor Cells Generated from Induced Pluripotent Stem Cells can Survive, Migrate, and Integrate in the Rodent Spinal Cord", J. COMP. NEUROL., 2014, vol. 522, No. 12, pp 2707 - 27280 * |
Also Published As
| Publication number | Publication date |
|---|---|
| JP2024073654A (ja) | 2024-05-29 |
| KR102843346B1 (ko) | 2025-08-06 |
| JP7535944B2 (ja) | 2024-08-19 |
| US20210024955A1 (en) | 2021-01-28 |
| EP3765058A4 (en) | 2021-12-22 |
| US12258571B2 (en) | 2025-03-25 |
| KR20200132957A (ko) | 2020-11-25 |
| CA3092284A1 (en) | 2019-09-19 |
| JP2021518365A (ja) | 2021-08-02 |
| CN111867617A (zh) | 2020-10-30 |
| EP3765058A1 (en) | 2021-01-20 |
| AU2019236288A1 (en) | 2020-09-17 |
| WO2019178550A1 (en) | 2019-09-19 |
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