IL281563B1 - Compositions and methods for restoring paternal ube3a gene expression in human Engelman syndrome - Google Patents

Compositions and methods for restoring paternal ube3a gene expression in human Engelman syndrome

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Publication number
IL281563B1
IL281563B1 IL281563A IL28156321A IL281563B1 IL 281563 B1 IL281563 B1 IL 281563B1 IL 281563 A IL281563 A IL 281563A IL 28156321 A IL28156321 A IL 28156321A IL 281563 B1 IL281563 B1 IL 281563B1
Authority
IL
Israel
Prior art keywords
tat
seq
shrna
nucleotide sequence
ube3a
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Application number
IL281563A
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English (en)
Hebrew (he)
Other versions
IL281563A (en
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Univ Connecticut
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Application filed by Univ Connecticut filed Critical Univ Connecticut
Publication of IL281563A publication Critical patent/IL281563A/en
Publication of IL281563B1 publication Critical patent/IL281563B1/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
    • C12N15/11—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
    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
    • C12N15/1137—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 against enzymes
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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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/121—Hammerhead
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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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/128—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes processing or releasing ribozyme
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15042—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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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
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
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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
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
IL281563A 2018-09-21 2021-03-16 Compositions and methods for restoring paternal ube3a gene expression in human Engelman syndrome IL281563B1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862734435P 2018-09-21 2018-09-21
PCT/US2019/052272 WO2020061528A1 (en) 2018-09-21 2019-09-20 Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome

Publications (2)

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IL281563A IL281563A (en) 2021-05-31
IL281563B1 true IL281563B1 (en) 2026-01-01

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EP (1) EP3833398A4 (enExample)
JP (1) JP7546295B2 (enExample)
KR (1) KR102782761B1 (enExample)
CN (2) CN119592561A (enExample)
AU (2) AU2019344006B2 (enExample)
CA (1) CA3113648A1 (enExample)
IL (1) IL281563B1 (enExample)
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WO2020061528A1 (en) 2018-09-21 2020-03-26 University Of Connecticut Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome
JP2023524247A (ja) * 2020-04-28 2023-06-09 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア アンジェルマン症候群の治療のための組成物及びその使用
US20240318198A1 (en) * 2021-02-03 2024-09-26 The Board Of Regents Of The University Of Texas System Gene therapy for angelman syndrome
CN119384504A (zh) * 2022-03-07 2025-01-28 康涅狄格大学 靶向SNORD115位置以恢复快乐木偶综合征中父系UBE3A基因表达的shRNA
KR20230152608A (ko) 2022-04-27 2023-11-03 중앙대학교 산학협력단 CRISPR-Cas12f1 시스템을 이용한 유전체의 단일 뉴클레오타이드 편집 방법 및 이의 용도

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WO2016086104A1 (en) * 2014-11-25 2016-06-02 Ionis Pharmaceuticals, Inc. Modulation of ube3a-ats expression

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AU2019344006B2 (en) 2025-11-27
KR20210065125A (ko) 2021-06-03
JP7546295B2 (ja) 2024-09-06
CN113164624B (zh) 2024-09-24
EP3833398A4 (en) 2021-12-22
WO2020061528A1 (en) 2020-03-26
MX2021003303A (es) 2021-06-23
KR102782761B1 (ko) 2025-03-14
US20210332368A1 (en) 2021-10-28
EP3833398A1 (en) 2021-06-16
AU2019344006A1 (en) 2021-03-25
CN119592561A (zh) 2025-03-11
JP2022501044A (ja) 2022-01-06
CA3113648A1 (en) 2020-03-26
US20250136994A1 (en) 2025-05-01
IL281563A (en) 2021-05-31
US12221609B2 (en) 2025-02-11
AU2026200604A1 (en) 2026-02-19
CN113164624A (zh) 2021-07-23

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