AU2019276645B2 - Compositions and methods for treatment of dominant retinitis pigmentosa - Google Patents

Compositions and methods for treatment of dominant retinitis pigmentosa Download PDF

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AU2019276645B2
AU2019276645B2 AU2019276645A AU2019276645A AU2019276645B2 AU 2019276645 B2 AU2019276645 B2 AU 2019276645B2 AU 2019276645 A AU2019276645 A AU 2019276645A AU 2019276645 A AU2019276645 A AU 2019276645A AU 2019276645 B2 AU2019276645 B2 AU 2019276645B2
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rho
vector
seq
sequence
subject
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AU2019276645A1 (en
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Gustavo D. Aguirre
William BELTRAN
Artur CIDECIYAN
William W. Hauswirth
Samuel Jacobson
Alfred S. Lewin
Michael T. Massengill
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University of Pennsylvania Penn
University of Florida Research Foundation Inc
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University of Florida Research Foundation Inc
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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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    • 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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N15/1138—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 receptors or cell surface proteins
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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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    • C12N15/86—Viral vectors
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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AU2019276645A 2018-06-01 2019-06-03 Compositions and methods for treatment of dominant retinitis pigmentosa Active AU2019276645B2 (en)

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US201862679585P 2018-06-01 2018-06-01
US62/679,585 2018-06-01
US201962809539P 2019-02-22 2019-02-22
US62/809,539 2019-02-22
PCT/US2019/035159 WO2019232517A1 (en) 2018-06-01 2019-06-03 Compositions and methods for treatment of dominant retinitis pigmentosa

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AU2019276645A1 AU2019276645A1 (en) 2020-11-26
AU2019276645B2 true AU2019276645B2 (en) 2025-06-26

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US (2) US12203074B2 (https=)
EP (1) EP3802836A4 (https=)
JP (1) JP7563694B2 (https=)
CN (2) CN112204145B (https=)
AU (1) AU2019276645B2 (https=)
CA (1) CA3102095A1 (https=)
IL (1) IL279091B1 (https=)
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WO2021212686A1 (zh) * 2020-04-21 2021-10-28 北京中因科技有限公司 RHO-adRP基于基因编辑的方法和组合物
CN114521214A (zh) * 2020-07-24 2022-05-20 尔知渃米斯股份有限公司 视紫红质转录体特异性反式剪接核酶及其用途
JP7334346B2 (ja) * 2020-07-24 2023-08-28 アールズィーノミクス・インコーポレイテッド ロドプシン転写体に特異的なトランススプライシングリボザイム及びその用途
US20220098615A1 (en) * 2020-09-30 2022-03-31 NGGT, Inc. Dual functional expression vectors and methods of use thereof
WO2024211266A1 (en) * 2023-04-07 2024-10-10 University Of Houston System Downregulation of rhodopsin as a therapeutic strategy for peripherin-2-associated inherited retinal disorders

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IL279091B1 (en) 2026-01-01
IL279091A (en) 2021-01-31
JP2021525531A (ja) 2021-09-27
US20250215437A1 (en) 2025-07-03
WO2019232517A1 (en) 2019-12-05
US12203074B2 (en) 2025-01-21
US20210207147A1 (en) 2021-07-08
AU2019276645A1 (en) 2020-11-26
JP7563694B2 (ja) 2024-10-08
EP3802836A4 (en) 2022-10-19
EP3802836A1 (en) 2021-04-14
CN118480544A (zh) 2024-08-13
CN112204145A (zh) 2021-01-08
CN112204145B (zh) 2024-03-26
CA3102095A1 (en) 2019-12-05

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