SG11201909203WA - Tissue selective transgene expression - Google Patents
Tissue selective transgene expressionInfo
- Publication number
- SG11201909203WA SG11201909203WA SG11201909203WA SG11201909203WA SG 11201909203W A SG11201909203W A SG 11201909203WA SG 11201909203W A SG11201909203W A SG 11201909203WA SG 11201909203W A SG11201909203W A SG 11201909203WA
- Authority
- SG
- Singapore
- Prior art keywords
- california
- suite
- international
- south san
- transgene
- Prior art date
Links
- 108700019146 Transgenes Proteins 0.000 title abstract 6
- 241000237502 Ostreidae Species 0.000 abstract 2
- 239000000203 mixture Substances 0.000 abstract 2
- 235000020636 oyster Nutrition 0.000 abstract 2
- 239000003814 drug Substances 0.000 abstract 1
- 230000008520 organization Effects 0.000 abstract 1
- 230000001105 regulatory effect Effects 0.000 abstract 1
Classifications
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
- A01K67/027—New or modified breeds of vertebrates
- A01K67/0275—Genetically modified vertebrates, e.g. transgenic
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- 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
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- 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
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
-
- 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
-
- 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
- C12N15/63—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
-
- 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
- C12N15/63—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
-
- 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
- C12N15/63—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
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
- A01K2217/052—Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/15—Animals comprising multiple alterations of the genome, by transgenesis or homologous recombination, e.g. obtained by cross-breeding
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
- A01K2267/0312—Animal model for Alzheimer's disease
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- 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
- A61P25/08—Antiepileptics; Anticonvulsants
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- 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
- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Virology (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Cell Biology (AREA)
- Immunology (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Pain & Pain Management (AREA)
- Environmental Sciences (AREA)
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201762480998P | 2017-04-03 | 2017-04-03 | |
PCT/US2018/025940 WO2018187363A1 (en) | 2017-04-03 | 2018-04-03 | Tissue selective transgene expression |
Publications (1)
Publication Number | Publication Date |
---|---|
SG11201909203WA true SG11201909203WA (en) | 2019-11-28 |
Family
ID=63713399
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG11201909203W SG11201909203WA (en) | 2017-04-03 | 2018-04-03 | Tissue selective transgene expression |
Country Status (16)
Country | Link |
---|---|
US (6) | US10287608B2 (ja) |
EP (1) | EP3607073A4 (ja) |
JP (2) | JP7246359B2 (ja) |
KR (1) | KR102604159B1 (ja) |
CN (2) | CN117821509A (ja) |
AU (1) | AU2018250161B2 (ja) |
BR (1) | BR112019020777A2 (ja) |
CA (1) | CA3058189A1 (ja) |
CL (2) | CL2019002805A1 (ja) |
CO (1) | CO2019011450A2 (ja) |
EA (1) | EA201992358A1 (ja) |
IL (1) | IL269767A (ja) |
MX (4) | MX2019011772A (ja) |
SG (1) | SG11201909203WA (ja) |
TW (3) | TW202315946A (ja) |
WO (1) | WO2018187363A1 (ja) |
Families Citing this family (17)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
SG11201702682PA (en) | 2014-10-03 | 2017-04-27 | Cold Spring Harbor Lab | Targeted augmentation of nuclear gene output |
SG11201802870RA (en) | 2015-10-09 | 2018-05-30 | Univ Southampton | Modulation of gene expression and screening for deregulated protein expression |
EP3933041B1 (en) | 2015-12-14 | 2024-01-31 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of autosomal dominant retardation |
US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
SG11201909203WA (en) | 2017-04-03 | 2019-11-28 | Encoded Therapeutics Inc | Tissue selective transgene expression |
KR20240035631A (ko) | 2017-08-25 | 2024-03-15 | 스톡 테라퓨틱스, 인크. | 병태 및 질환 치료용 안티센스 올리고머 |
CA3096407A1 (en) * | 2018-04-09 | 2019-10-17 | Allen Institute | Rescuing voltage-gated sodium channel function in inhibitory neurons |
CN113966399A (zh) | 2018-09-26 | 2022-01-21 | 加州理工学院 | 用于靶向基因疗法的腺相关病毒组合物 |
CN113966400A (zh) * | 2019-02-05 | 2022-01-21 | 博德研究所 | 用于使神经元细胞兴奋性正常化和治疗德拉韦综合征的中间神经元特异性治疗剂 |
CN113710693A (zh) * | 2019-02-25 | 2021-11-26 | 马萨诸塞大学 | Dna结合结构域反式激活因子及其用途 |
WO2020198017A1 (en) * | 2019-03-22 | 2020-10-01 | Encoded Therapeutics, Inc. | Multiplexing regulatory elements to identify cell-type specific regulatory elements |
SG11202113048SA (en) * | 2019-05-29 | 2021-12-30 | Encoded Therapeutics Inc | Compositions and methods for selective gene regulation |
US20230287410A1 (en) | 2020-05-11 | 2023-09-14 | Stoke Therapeutics, Inc. | Opa1 antisense oligomers for treatment of conditions and diseases |
EP4179098A1 (en) * | 2020-07-08 | 2023-05-17 | Baylor College Of Medicine | Gene therapy for stxbp1 encephalopathy |
KR20230037586A (ko) * | 2020-07-09 | 2023-03-16 | 가부시키가이샤 모달리스 | Mapt 유전자를 표적으로 하는 알츠하이머병의 치료 방법 |
AU2022378524A1 (en) * | 2021-10-28 | 2024-05-02 | UCB Biopharma SRL | Nucleic acid constructs, viral vectors and viral particles |
Family Cites Families (55)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US7405A (en) * | 1850-05-28 | John weidman | ||
JPH10501686A (ja) | 1994-04-13 | 1998-02-17 | ザ ロックフェラー ユニヴァーシティ | 神経系の細胞へのdnaのaav仲介送達 |
WO1998046273A2 (en) | 1997-04-17 | 1998-10-22 | Paola Leone | Delivery system for gene therapy to the brain |
CA2205076A1 (en) | 1997-05-14 | 1998-11-14 | Jim Hu | Episomal expression cassettes for gene therapy |
AU746454B2 (en) | 1998-03-02 | 2002-05-02 | Massachusetts Institute Of Technology | Poly zinc finger proteins with improved linkers |
US6303370B1 (en) | 1998-03-24 | 2001-10-16 | Mayo Foundation For Medical Education And Research | Tissue-specific regulatory elements |
CA2246005A1 (en) | 1998-10-01 | 2000-04-01 | Hsc Research And Development Limited Partnership | Hybrid genes for gene therapy in erythroid cells |
US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
US6649371B1 (en) | 1999-06-11 | 2003-11-18 | Neurosearch A/S | Potassium channel KCNQ5 and sequences encoding the same |
DE1252330T1 (de) * | 1999-11-26 | 2003-11-27 | Univ Montreal Mcgill | Loci der idiopathischen epilepsie, mutationen derselben und verfahren zu deren verwendung zur feststellung, prognose und behandlung von epilepsie |
AU776576B2 (en) | 1999-12-06 | 2004-09-16 | Sangamo Biosciences, Inc. | Methods of using randomized libraries of zinc finger proteins for the identification of gene function |
WO2001094414A2 (en) | 2000-06-07 | 2001-12-13 | Ortho Mcneil Pharmaceutical, Inc. | THE HUMAN VOLTAGE GATED SODIUM CHANNEL β-1A SUBUNIT AND METHODS OF USE |
AUPR492201A0 (en) | 2001-05-10 | 2001-06-07 | Bionomics Limited | Novel mutation |
EP1402043A1 (en) | 2001-07-03 | 2004-03-31 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Methods of administering vectors to synaptically connected neurons |
US6998118B2 (en) | 2001-12-21 | 2006-02-14 | The Salk Institute For Biological Studies | Targeted retrograde gene delivery for neuronal protection |
CA2474751A1 (en) | 2002-02-25 | 2003-09-04 | Vanderbilt University | Expression system for human brain-specific voltage-gated sodium channel, type 1 |
US7361635B2 (en) | 2002-08-29 | 2008-04-22 | Sangamo Biosciences, Inc. | Simultaneous modulation of multiple genes |
ATE446371T1 (de) | 2003-01-28 | 2009-11-15 | Ludwig Aigner | Verwendung von regulatorischen sequenzen zur spezifischen, transienten expression in neuronal determinierten zellen |
US20040258666A1 (en) | 2003-05-01 | 2004-12-23 | Passini Marco A. | Gene therapy for neurometabolic disorders |
US7094600B2 (en) | 2003-06-26 | 2006-08-22 | The Research Foundation Of State University Of New York | Screen for sodium channel modulators |
JP2007532639A (ja) | 2004-04-14 | 2007-11-15 | エージェンシー フォー サイエンス,テクノロジー アンド リサーチ | 神経細胞への遺伝子送達方法 |
TWI293307B (en) | 2004-09-30 | 2008-02-11 | Ind Tech Res Inst | A liver-specific chimeric regulatory sequence and use thereof |
EP2021499A4 (en) * | 2005-12-16 | 2010-02-17 | Univ Leland Stanford Junior | FUNCTIONAL ARRAYS FOR THE IDENTIFICATION OF ELEMENTS FOR GENE PRESSION REGULATION WITH HIGH THROUGHPUT |
US20070161031A1 (en) | 2005-12-16 | 2007-07-12 | The Board Of Trustees Of The Leland Stanford Junior University | Functional arrays for high throughput characterization of gene expression regulatory elements |
WO2007084957A2 (en) | 2006-01-20 | 2007-07-26 | University Of California, San Francisco | Transplantation of neural cells |
EP2097538A4 (en) | 2006-12-07 | 2011-11-30 | Switchgear Genomics | TRANSCRIPTION REAGULATION ELEMENTS OF BIOLOGICAL PATHS, TOOLS AND METHODS |
EP2019143A1 (en) | 2007-07-23 | 2009-01-28 | Genethon | CNS gene delivery using peripheral administration of AAV vectors |
US20100297674A1 (en) | 2008-01-22 | 2010-11-25 | Chromocell Corporation | NOVEL CELL LINES EXPRESSING NaV AND METHODS USING THEM |
US20110165129A1 (en) | 2008-05-06 | 2011-07-07 | Arnold Kriegstein | Ameliorating Nervous Systems Disorders |
US9267151B2 (en) * | 2008-08-20 | 2016-02-23 | Brainco Biopharma, S.L. | STXBP1 overexpressing mouse and its uses in screening of treatments for neuropsychiatric illness |
WO2010037143A1 (en) | 2008-09-29 | 2010-04-01 | The University Of Montana | Vectors and methods of treating brain seizures |
US8815779B2 (en) | 2009-09-16 | 2014-08-26 | SwitchGear Genomics, Inc. | Transcription biomarkers of biological responses and methods |
US20110135611A1 (en) | 2009-12-03 | 2011-06-09 | The J. David Gladstone Institutes | Methods for treating apolipoprotein e4-associated disorders |
US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
CA2833908C (en) | 2010-04-23 | 2021-02-09 | University Of Massachusetts | Cns targeting aav vectors and methods of use thereof |
US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
LT2566972T (lt) | 2010-05-03 | 2020-03-10 | Sangamo Therapeutics, Inc. | Kompozicijos, skirtos cinko pirštu modulių susiejimui |
PL2585596T3 (pl) | 2010-06-23 | 2021-06-28 | Curna, Inc. | Leczenie chorób związanych z podjednostką alfa kanału sodowego bramkowanego napięciem (SCNA) poprzez hamowanie naturalnego transkryptu antysensownego SCNA |
JP5704361B2 (ja) | 2010-10-27 | 2015-04-22 | 学校法人自治医科大学 | 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン |
EP2635686B1 (en) | 2010-11-05 | 2018-03-28 | The Regents of The University of California | Neuronal specific targeting of caveolin expression to restore synaptic signaling and improve cognitive function in the neurodegenerative brain and motor function in spinal cord |
WO2012065143A1 (en) * | 2010-11-12 | 2012-05-18 | The General Hospital Corporation | Polycomb-associated non-coding rnas |
EP2655621B1 (en) * | 2010-12-20 | 2018-05-23 | The General Hospital Corporation | Polycomb-associated non-coding rnas |
WO2012112578A2 (en) | 2011-02-14 | 2012-08-23 | The Children's Hospital Of Philadelphia | Improved aav8 vector with enhanced functional activity and methods of use thereof |
WO2013123503A1 (en) | 2012-02-17 | 2013-08-22 | The Children's Hospital Of Philadelphia | Aav vector compositions and methods for gene transfer to cells, organs and tissues |
US9284575B2 (en) | 2012-03-06 | 2016-03-15 | Duke University | Synthetic regulation of gene expression |
WO2013155222A2 (en) | 2012-04-10 | 2013-10-17 | The Regents Of The University Of California | Brain-specific enhancers for cell-based therapy |
KR102527259B1 (ko) | 2013-03-15 | 2023-04-27 | 더 칠드런스 호스피탈 오브 필라델피아 | 스터퍼/필러 폴리누클레오티드 서열을 포함하는 벡터 및 사용 방법 |
BR112015028605A8 (pt) | 2013-05-15 | 2019-12-24 | Univ Minnesota | uso de uma composição e uso de um imunossupressor e uma composição |
JP6594891B2 (ja) | 2014-03-18 | 2019-10-23 | サンガモ セラピューティクス, インコーポレイテッド | ジンクフィンガータンパク質発現を調節するための方法および組成物 |
EP3151846A4 (en) * | 2014-06-05 | 2017-12-27 | Sangamo BioSciences, Inc. | Methods and compositions for nuclease design |
US11020443B2 (en) | 2015-04-23 | 2021-06-01 | University Of Massachusetts | Modulation of AAV vector transgene expression |
CN104846015B (zh) | 2015-05-27 | 2018-03-27 | 深圳先进技术研究院 | 特异性兴奋伏隔核中的gaba能神经元的组合物及其在改善精神分裂症异样行为中的应用 |
WO2017075335A1 (en) | 2015-10-28 | 2017-05-04 | Voyager Therapeutics, Inc. | Regulatable expression using adeno-associated virus (aav) |
MX2018004755A (es) | 2015-10-29 | 2018-12-19 | Voyager Therapeutics Inc | Entrega de polinucleotidos dirigidos al sistema nervioso central. |
SG11201909203WA (en) | 2017-04-03 | 2019-11-28 | Encoded Therapeutics Inc | Tissue selective transgene expression |
-
2018
- 2018-04-03 SG SG11201909203W patent/SG11201909203WA/en unknown
- 2018-04-03 CA CA3058189A patent/CA3058189A1/en active Pending
- 2018-04-03 KR KR1020197032432A patent/KR102604159B1/ko active IP Right Grant
- 2018-04-03 EA EA201992358A patent/EA201992358A1/ru unknown
- 2018-04-03 JP JP2020502552A patent/JP7246359B2/ja active Active
- 2018-04-03 EP EP18781685.5A patent/EP3607073A4/en active Pending
- 2018-04-03 TW TW111150117A patent/TW202315946A/zh unknown
- 2018-04-03 MX MX2019011772A patent/MX2019011772A/es unknown
- 2018-04-03 TW TW107111969A patent/TWI808079B/zh active
- 2018-04-03 CN CN202410011187.9A patent/CN117821509A/zh active Pending
- 2018-04-03 BR BR112019020777A patent/BR112019020777A2/pt unknown
- 2018-04-03 WO PCT/US2018/025940 patent/WO2018187363A1/en active Application Filing
- 2018-04-03 TW TW111150116A patent/TW202315945A/zh unknown
- 2018-04-03 CN CN201880036950.6A patent/CN110730823B/zh active Active
- 2018-04-03 AU AU2018250161A patent/AU2018250161B2/en active Active
- 2018-10-05 US US16/153,420 patent/US10287608B2/en active Active
- 2018-10-05 US US16/153,443 patent/US20190024121A1/en not_active Abandoned
- 2018-10-05 US US16/153,401 patent/US10287607B2/en active Active
- 2018-10-05 US US16/153,433 patent/US10519465B2/en active Active
-
2019
- 2019-09-30 MX MX2022015604A patent/MX2022015604A/es unknown
- 2019-09-30 MX MX2022015605A patent/MX2022015605A/es unknown
- 2019-09-30 MX MX2022015603A patent/MX2022015603A/es unknown
- 2019-10-01 CL CL2019002805A patent/CL2019002805A1/es unknown
- 2019-10-02 IL IL26976719A patent/IL269767A/en unknown
- 2019-10-16 CO CO2019011450A patent/CO2019011450A2/es unknown
- 2019-10-31 US US16/670,996 patent/US20200165628A1/en not_active Abandoned
-
2022
- 2022-11-16 US US18/055,968 patent/US20230203531A1/en active Pending
-
2023
- 2023-01-30 CL CL2023000294A patent/CL2023000294A1/es unknown
- 2023-03-14 JP JP2023039486A patent/JP7477675B2/ja active Active
Also Published As
Similar Documents
Publication | Publication Date | Title |
---|---|---|
SG11201909203WA (en) | Tissue selective transgene expression | |
SG11201901364VA (en) | Engineered target specific nucleases | |
SG11201805320XA (en) | Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration | |
SG11201901531TA (en) | Regulation of gene expression using engineered nucleases | |
SG11201804070XA (en) | Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a | |
SG11201805162VA (en) | Plasmid constructs for heterologous protein expression and methods of use | |
SG11201907653QA (en) | Compositions and methods for enhanced gene expression | |
SG11201908489XA (en) | De novo synthesized combinatorial nucleic acid libraries | |
SG11201809344QA (en) | Process for synthesizing 2-hydroxy-6-((2-(1-isopropyl-1h-pyrazol-5-yl)-pyridin-3-yl)methoxy)benzaldehyde | |
SG11201909432SA (en) | Compounds for increasing genome editing efficiency | |
SG11201909949XA (en) | Targeted immunotolerance | |
SG11201811048UA (en) | Single-cell transcript sequencing | |
SG11201808622SA (en) | Chimeric receptors to flt3 and methods of use thereof | |
SG11201811232XA (en) | Treatment of amd using aav2 variant with aflibercept | |
SG11201903167QA (en) | Compounds and methods for reducing atxn3 expression | |
SG11201807881VA (en) | Compositions and methods for making antibodies based on use of an expression-enhancing locus | |
SG11201901206SA (en) | Trimer stabilizing hiv envelope protein mutations | |
SG11201900038QA (en) | Compositions and methods comprising improvements of crispr guide rnas using the h1 promoter | |
SG11201804375WA (en) | Compositions and methods for internalizing enzymes | |
SG11201809144XA (en) | Materials and methods for treatment of hemoglobinopathies | |
SG11201807134RA (en) | Transposon system and methods of use | |
SG11201901959YA (en) | Modified stem cell memory t cells, methods of making and methods of using same | |
SG11201810757YA (en) | Non-human animals expressing exogenous terminal deoxynucleotidyltransferase | |
SG11201810525XA (en) | Anti-gitr antibodies and uses thereof | |
SG11201905508VA (en) | Hsd17b13 variants and uses thereof |