IL280636A - Alternative splicing regulation of gene expression and therapeutic methods - Google Patents

Alternative splicing regulation of gene expression and therapeutic methods

Info

Publication number
IL280636A
IL280636A IL280636A IL28063621A IL280636A IL 280636 A IL280636 A IL 280636A IL 280636 A IL280636 A IL 280636A IL 28063621 A IL28063621 A IL 28063621A IL 280636 A IL280636 A IL 280636A
Authority
IL
Israel
Prior art keywords
gene expression
therapeutic methods
alternative splicing
splicing control
control
Prior art date
Application number
IL280636A
Other languages
English (en)
Hebrew (he)
Inventor
L Davidson Beverly
Mas Monteys Alejandro
Al HUNDLEY Ammiel
T Ranum Paul
Original Assignee
Childrens Hospital Philadelphia
L Davidson Beverly
Mas Monteys Alejandro
Al HUNDLEY Ammiel
T Ranum Paul
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 Childrens Hospital Philadelphia, L Davidson Beverly, Mas Monteys Alejandro, Al HUNDLEY Ammiel, T Ranum Paul filed Critical Childrens Hospital Philadelphia
Publication of IL280636A publication Critical patent/IL280636A/en

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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/50—Pyridazines; Hydrogenated pyridazines
    • A61K31/501—Pyridazines; Hydrogenated pyridazines not condensed and containing further heterocyclic rings
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/357—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having two or more oxygen atoms in the same ring, e.g. crown ethers, guanadrel
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • A—HUMAN NECESSITIES
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
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    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
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    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
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    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
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    • 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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    • 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
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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
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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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    • 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 [RNase]; Deoxyribonucleases [DNase]
    • A—HUMAN NECESSITIES
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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
    • C12N15/11—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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • 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
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
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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

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  • Health & Medical Sciences (AREA)
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  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Mycology (AREA)
  • Psychology (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
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IL280636A 2018-08-07 2021-02-04 Alternative splicing regulation of gene expression and therapeutic methods IL280636A (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862715756P 2018-08-07 2018-08-07
US201962837701P 2019-04-23 2019-04-23
US201962838223P 2019-04-24 2019-04-24
US201962872417P 2019-07-10 2019-07-10
PCT/US2019/045401 WO2020033473A1 (en) 2018-08-07 2019-08-07 Alternative splicing regulation of gene expression and therapeutic methods

Publications (1)

Publication Number Publication Date
IL280636A true IL280636A (en) 2021-03-25

Family

ID=69413338

Family Applications (1)

Application Number Title Priority Date Filing Date
IL280636A IL280636A (en) 2018-08-07 2021-02-04 Alternative splicing regulation of gene expression and therapeutic methods

Country Status (9)

Country Link
US (1) US20210355509A1 (https=)
EP (1) EP3833357A4 (https=)
JP (2) JP7615021B2 (https=)
KR (1) KR20210042123A (https=)
CN (1) CN112805009A (https=)
AU (1) AU2019317573A1 (https=)
CA (1) CA3108293A1 (https=)
IL (1) IL280636A (https=)
WO (1) WO2020033473A1 (https=)

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JP7013753B2 (ja) * 2017-09-15 2022-02-01 株式会社三洋物産 遊技機
JP7013754B2 (ja) * 2017-09-15 2022-02-01 株式会社三洋物産 遊技機
BR112022002794A2 (pt) * 2019-08-15 2022-08-09 Childrens Hospital Philadelphia Terapia combinada de transgene e mirna derivado de íntron para tratamento de sca1
TW202131920A (zh) * 2019-11-01 2021-09-01 瑞士商諾華公司 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途
EP4103717A4 (en) * 2020-02-12 2024-02-28 The Children's Hospital of Philadelphia COMPOSITIONS AND METHODS FOR INDUCIBLE ALTERNATIVE SPLICING REGULATION OF GENE EXPRESSION
JP2020151570A (ja) * 2020-06-24 2020-09-24 株式会社三洋物産 遊技機
EP4294459A4 (en) * 2021-02-19 2025-06-18 University Of Florida Research Foundation, Incorporated Methods and compositions to confer regulation to gene therapy cargoes by heterologous use of alternative splicing cassettes
JP2021079226A (ja) * 2021-03-01 2021-05-27 株式会社三洋物産 遊技機
JP2021079225A (ja) * 2021-03-01 2021-05-27 株式会社三洋物産 遊技機
JP2021087836A (ja) * 2021-03-01 2021-06-10 株式会社三洋物産 遊技機
JP2021087833A (ja) * 2021-03-01 2021-06-10 株式会社三洋物産 遊技機
US20250346893A1 (en) * 2021-10-22 2025-11-13 Fred Hutchinson Cancer Center Synthetic introns for targeted gene expression
EP4526455A1 (en) 2022-05-18 2025-03-26 The Children's Hospital of Philadelphia Compositions and methods for inducible alternative splicing regulation of gene expression
KR20250039324A (ko) * 2022-05-24 2025-03-20 케이트 테라퓨틱스, 인크. Xlmtm 치료를 위한 조성물
KR102470669B1 (ko) 2022-06-03 2022-11-25 충남대학교 산학협력단 스플라이싱-스위치 올리고뉴클레오티드를 유효성분으로 포함하는 폴리프로모 1 유전자의 스플라이싱 조절용 조성물
JP7556432B2 (ja) * 2022-08-22 2024-09-26 株式会社三洋物産 遊技機
JP7556434B2 (ja) * 2022-08-22 2024-09-26 株式会社三洋物産 遊技機
JP7556433B2 (ja) * 2022-08-22 2024-09-26 株式会社三洋物産 遊技機
CN118339290A (zh) * 2022-09-23 2024-07-12 北京基驭医疗科技有限公司 包含基于小分子药物的可变剪接调节元件的核酸分子
CN115747217B (zh) * 2022-10-26 2023-11-28 江苏省人民医院（南京医科大学第一附属医院） 长链非编码rna pdxdc1-as1及其应用
EP4701634A1 (en) * 2023-04-28 2026-03-04 Kriya Therapeutics, Inc. Riboswitches for regulating gene expression and therapeutic methods of using the same
WO2025170984A1 (en) * 2024-02-05 2025-08-14 The Children's Hospital Of Philadelphia Cell-type-specific control of gene expression in auditory hair cells

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