CN116670769A - 调节tcf4基因表达和治疗皮特霍普金斯综合征的组合物和方法 - Google Patents

调节tcf4基因表达和治疗皮特霍普金斯综合征的组合物和方法 Download PDF

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Publication number
CN116670769A
CN116670769A CN202180079761.9A CN202180079761A CN116670769A CN 116670769 A CN116670769 A CN 116670769A CN 202180079761 A CN202180079761 A CN 202180079761A CN 116670769 A CN116670769 A CN 116670769A
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China
Prior art keywords
tcf4
nucleic acid
subject
acid construct
recombinant nucleic
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CN202180079761.9A
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Chinese (zh)
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A·R·穆奥特里
F·帕佩斯
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University of California San Diego UCSD
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University of California San Diego UCSD
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    • 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
    • A61K48/0066—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
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
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    • 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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    • C12N15/09—Recombinant DNA-technology
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/50—Physical structure
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    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination

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  • Wood Science & Technology (AREA)
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  • Pharmacology & Pharmacy (AREA)
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  • Gastroenterology & Hepatology (AREA)
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  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
CN202180079761.9A 2020-09-30 2021-09-30 调节tcf4基因表达和治疗皮特霍普金斯综合征的组合物和方法 Pending CN116670769A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063085878P 2020-09-30 2020-09-30
US63/085,878 2020-09-30
PCT/US2021/052987 WO2022072709A1 (en) 2020-09-30 2021-09-30 Compositions and methods for modulating tcf4 gene expression and treating pitt hopkins syndrome

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CN116670769A true CN116670769A (zh) 2023-08-29

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US (1) US20230372542A1 (https=)
EP (1) EP4222268A4 (https=)
JP (1) JP7786750B2 (https=)
KR (1) KR20230079190A (https=)
CN (1) CN116670769A (https=)
AU (1) AU2021351512A1 (https=)
CA (1) CA3194085A1 (https=)
IL (1) IL301610A (https=)
WO (1) WO2022072709A1 (https=)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117298153A (zh) * 2023-08-30 2023-12-29 济宁市第一人民医院 脑内皮细胞来源的细胞外囊泡在神经炎症中的应用
CN117476101A (zh) * 2023-11-22 2024-01-30 浙江大学 一种多组学单细胞测序数据区分恶性细胞的方法、系统、设备和介质

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025213093A1 (en) * 2024-04-04 2025-10-09 Lundquist Institute For Biomedical Innovation At Harbor-Ucla Medical Center Antisense oligonucleotides for treating pitt hopkin syndrome

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US20180305689A1 (en) * 2015-04-22 2018-10-25 Mina Therapeutics Limited Sarna compositions and methods of use

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117298153A (zh) * 2023-08-30 2023-12-29 济宁市第一人民医院 脑内皮细胞来源的细胞外囊泡在神经炎症中的应用
CN117298153B (zh) * 2023-08-30 2024-03-08 济宁市第一人民医院 脑内皮细胞来源的细胞外囊泡在神经炎症中的应用
CN117476101A (zh) * 2023-11-22 2024-01-30 浙江大学 一种多组学单细胞测序数据区分恶性细胞的方法、系统、设备和介质

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JP7786750B2 (ja) 2025-12-16
EP4222268A4 (en) 2025-05-14
WO2022072709A1 (en) 2022-04-07
US20230372542A1 (en) 2023-11-23
AU2021351512A9 (en) 2023-07-06
EP4222268A1 (en) 2023-08-09
KR20230079190A (ko) 2023-06-05
IL301610A (en) 2023-05-01
AU2021351512A1 (en) 2023-05-11
JP2023544702A (ja) 2023-10-25
CA3194085A1 (en) 2022-04-07

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