JP7786750B2 - Ｔｃｆ４遺伝子発現を調節するため、およびピット・ホプキンス症候群を治療するための組成物および方法 - Google Patents

Ｔｃｆ４遺伝子発現を調節するため、およびピット・ホプキンス症候群を治療するための組成物および方法

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JP7786750B2
JP7786750B2 JP2023519419A JP2023519419A JP7786750B2 JP 7786750 B2 JP7786750 B2 JP 7786750B2 JP 2023519419 A JP2023519419 A JP 2023519419A JP 2023519419 A JP2023519419 A JP 2023519419A JP 7786750 B2 JP7786750 B2 JP 7786750B2
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tcf4
pths
expression
cells
nucleic acid
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JP2023544702A5 (https=
JP2023544702A (ja
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ムオトリ、アリソン、アール．
ペーパース、ファビオ
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University of California San Diego UCSD
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
    • A61K48/0066—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
    • A61K48/0008—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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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
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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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JP2023519419A 2020-09-30 2021-09-30 Ｔｃｆ４遺伝子発現を調節するため、およびピット・ホプキンス症候群を治療するための組成物および方法 Active JP7786750B2 (ja)

Applications Claiming Priority (3)

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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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JP2023544702A JP2023544702A (ja) 2023-10-25
JP2023544702A5 JP2023544702A5 (https=) 2024-08-13
JP7786750B2 true JP7786750B2 (ja) 2025-12-16

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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=)

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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
CN117476101B (zh) * 2023-11-22 2025-10-10 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
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

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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

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
Datebase: GenBank, [online], ACCESSION: NM_001083962, DEFINITION: Homo sapiens transcription factor 4 (TCF4), transcript variant 1, mRNA，2019年，[取得日 2025年7月17日], 取得先<https://www.ncbi.nlm.nih.gov/nuccore/NM_001083962.1/>
Genetic Normalization Strategy for the Treatment of Pitt-Hopkins Syndrome，Carolina Digital Repository, [online]，2020年04月25日，[取得日 2025年7月17日], 取得先<https://cdr.lib.unc.edu/downloads/0g354m478?locale=en>，DOI: https//doi.org/10.17615/dg8j-dh82, 文献公知日記載先<https://cdr.lib.unc.edu/concern/honors_theses/c534fv28p>
Human Molecular genetics，2012年，Vol.21, No.13，pp.2873-2888
invitrogen，User Manual: pcDNA3.1(+) and pcDNA3.1(-) Mammalian Expression Vector, [online]，2010年，Catalog nos. V790-20 and V795-20，[取得日 2025年7月17日], 取得先<https://assets.thermofisher.com/TFS-Assets/LSG/manuals/pcdna3_1_man.pdf>

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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
CN116670769A (zh) 2023-08-29
JP2023544702A (ja) 2023-10-25
CA3194085A1 (en) 2022-04-07

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