CA3096407A1 - Rescuing voltage-gated sodium channel function in inhibitory neurons - Google Patents

Rescuing voltage-gated sodium channel function in inhibitory neurons Download PDF

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Publication number
CA3096407A1
CA3096407A1 CA3096407A CA3096407A CA3096407A1 CA 3096407 A1 CA3096407 A1 CA 3096407A1 CA 3096407 A CA3096407 A CA 3096407A CA 3096407 A CA3096407 A CA 3096407A CA 3096407 A1 CA3096407 A1 CA 3096407A1
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Prior art keywords
seq
expression construct
expression
vector
protein
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CA3096407A
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English (en)
French (fr)
Inventor
John K. Mich
Edward Sebastian LEIN
Jonathan Ting
Boaz P. LEVI
Erik HESS
Franck KALUME
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Seattle Childrens Hospital
Allen Institute
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Seattle Childrens Hospital
Allen Institute
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Publication of CA3096407A1 publication Critical patent/CA3096407A1/en
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    • C07K14/4702—Regulators; Modulating activity
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    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A—HUMAN NECESSITIES
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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
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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  • Biophysics (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Dermatology (AREA)
  • 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)
CA3096407A 2018-04-09 2019-04-09 Rescuing voltage-gated sodium channel function in inhibitory neurons Pending CA3096407A1 (en)

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

Publications (1)

Publication Number Publication Date
CA3096407A1 true CA3096407A1 (en) 2019-10-17

Family

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Family Applications (1)

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CA3096407A Pending CA3096407A1 (en) 2018-04-09 2019-04-09 Rescuing voltage-gated sodium channel function in inhibitory neurons

Country Status (6)

Country Link
US (2) US12121563B2 (https=)
EP (1) EP3781174A4 (https=)
JP (2) JP7472036B2 (https=)
AU (2) AU2019253700B2 (https=)
CA (1) CA3096407A1 (https=)
WO (1) WO2019199867A1 (https=)

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EP3933041B1 (en) 2015-12-14 2024-01-31 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant retardation
SG11202001590RA (en) 2017-08-25 2020-03-30 Stoke Therapeutics Inc Antisense oligomers for treatment of conditions and diseases
US12060558B2 (en) 2018-05-04 2024-08-13 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
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
CA3173609A1 (en) * 2020-03-27 2021-09-30 Allen Institute Artificial expression constructs for selectively modulating gene expression in non-neuronal brain cells
BR112022022889A2 (pt) 2020-05-11 2023-04-04 Stoke Therapeutics Inc Oligômeros antissentido de opa1 para tratamento de condições e doenças
EP4162041A4 (en) * 2020-06-04 2024-10-16 Allen Institute ARTIFICIAL EXPRESSION CONSTRUCTS TO SELECTIVELY MODULATE GENE EXPRESSION IN INHIBITORY NEOCORTICAL NEURONS
WO2022020706A1 (en) * 2020-07-24 2022-01-27 University Of Massachusetts Dna-binding domain transactivators and uses thereof
GB202013940D0 (en) 2020-09-04 2020-10-21 Synpromics Ltd Regulatory nucleic acid sequences
US20240012004A1 (en) * 2020-11-17 2024-01-11 Merck Sharp & Dohme Llc Increased voltage-gated sodium channel alpha protein subunit expression through viral 2a-mediated co-expression of nav beta subunits
US20250325704A1 (en) 2020-12-23 2025-10-23 Boehringer Ingelheim International Gmbh Viral capsid proteins with specificity to heart tissue cells
CA3207224A1 (en) * 2021-02-02 2022-08-11 Bryan GORE Artificial expression constructs for modulating gene expression in gabaergic neurons and astrocytes
TW202313972A (zh) * 2021-06-03 2023-04-01 荷蘭商美勒斯公司 新ｎｒｇ１融合物、融合接合處及檢測彼等之方法
JP2025520385A (ja) * 2022-06-13 2025-07-03 アレン インスティテュート 中枢神経系の非神経細胞において遺伝子発現を調節するための人工発現コンストラクト
AU2024214841A1 (en) * 2023-02-01 2025-08-28 Allen Institute Intein-mediated reconstitution of voltage-gated sodium channel function
WO2025019685A2 (en) * 2023-07-18 2025-01-23 Allen Institute Antisense oligonucleotides and artificial expression constructs for expressing rna in inhibitory neurons
WO2025038955A1 (en) * 2023-08-16 2025-02-20 Seattle Children's Hospital D/B/A Seattle Children's Research Institute Systems and methods for improving safety of split intein aav mediated gene therapy
WO2025092951A1 (en) * 2023-11-03 2025-05-08 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant adeno-associated viral vectors for treating fabry disease

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Also Published As

Publication number Publication date
US20250041384A1 (en) 2025-02-06
AU2019253700B2 (en) 2025-08-14
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
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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