AU2019253700B2 - Rescuing voltage-gated sodium channel function in inhibitory neurons - Google Patents

Rescuing voltage-gated sodium channel function in inhibitory neurons

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Publication number
AU2019253700B2
AU2019253700B2 AU2019253700A AU2019253700A AU2019253700B2 AU 2019253700 B2 AU2019253700 B2 AU 2019253700B2 AU 2019253700 A AU2019253700 A AU 2019253700A AU 2019253700 A AU2019253700 A AU 2019253700A AU 2019253700 B2 AU2019253700 B2 AU 2019253700B2
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seq
vector
expression
scn1a
protein
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AU2019253700A2 (en
AU2019253700A1 (en
Inventor
Erik HESS
Franck KALUME
Edward Sebastian LEIN
Boaz P. LEVI
John K. Mich
Jonathan Ting
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Seattle Childrens Hospital
Allen Institute
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Seattle Childrens Hospital
Allen Institute
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    • A—HUMAN NECESSITIES
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    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C07K14/4705—Regulators; Modulating activity stimulating, promoting or activating activity
    • 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
    • A61K48/005—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
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2217/077—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out heterozygous knock out animals displaying phenotype
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • 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

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  • Biophysics (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
AU2019253700A 2018-04-09 2019-04-09 Rescuing voltage-gated sodium channel function in inhibitory neurons Active AU2019253700B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2025263840A AU2025263840A1 (en) 2018-04-09 2025-11-07 Rescuing voltage-gated sodium channel function in inhibitory neurons

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201862655043P 2018-04-09 2018-04-09
US62/655,043 2018-04-09
US201862742835P 2018-10-08 2018-10-08
US62/742,835 2018-10-08
US201962810281P 2019-02-25 2019-02-25
US62/810,281 2019-02-25
PCT/US2019/026638 WO2019199867A1 (en) 2018-04-09 2019-04-09 Rescuing voltage-gated sodium channel function in inhibitory neurons

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AU2019253700A1 AU2019253700A1 (en) 2020-11-26
AU2019253700A2 AU2019253700A2 (en) 2020-12-03
AU2019253700B2 true AU2019253700B2 (en) 2025-08-14

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EP (1) EP3781174A4 (https=)
JP (2) JP7472036B2 (https=)
AU (2) AU2019253700B2 (https=)
CA (1) CA3096407A1 (https=)
WO (1) WO2019199867A1 (https=)

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JP7695192B2 (ja) 2018-10-08 2025-06-18 アレン インスティテュート 介在ニューロンにおける遺伝子発現を選択的に調節するための人工発現構築物
JP7624728B2 (ja) 2019-02-25 2025-01-31 ユニバーシティ オブ マサチューセッツ Ｄｎａ結合ドメイントランスアクチベーター及びその使用
KR20210134003A (ko) 2019-02-27 2021-11-08 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 안티센스 올리고머
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US20250041384A1 (en) 2025-02-06
AU2019253700A2 (en) 2020-12-03
AU2025263840A1 (en) 2026-01-15
EP3781174A1 (en) 2021-02-24
EP3781174A4 (en) 2022-01-26
WO2019199867A1 (en) 2019-10-17
JP2021521152A (ja) 2021-08-26
JP7472036B2 (ja) 2024-04-22
CA3096407A1 (en) 2019-10-17
AU2019253700A1 (en) 2020-11-26
US20210015898A1 (en) 2021-01-21
US12121563B2 (en) 2024-10-22
JP2024095779A (ja) 2024-07-10
JP7793675B2 (ja) 2026-01-05

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