EA202192815A1 - Опосредуемая aav-вектором делеция крупной мутационной "горячей точки" для лечения мышечной дистрофии дюшенна - Google Patents

Опосредуемая aav-вектором делеция крупной мутационной "горячей точки" для лечения мышечной дистрофии дюшенна

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Publication number
EA202192815A1
EA202192815A1 EA202192815A EA202192815A EA202192815A1 EA 202192815 A1 EA202192815 A1 EA 202192815A1 EA 202192815 A EA202192815 A EA 202192815A EA 202192815 A EA202192815 A EA 202192815A EA 202192815 A1 EA202192815 A1 EA 202192815A1
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EA
Eurasian Patent Office
Prior art keywords
hotspoint
aav
deletion
vector
treatment
Prior art date
Application number
EA202192815A
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English (en)
Inventor
Чарльз А. Герсбах
Карен Булаклак
Жаклин Н. Робинсон-Хамм
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Дьюк Юниверсити
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Publication of EA202192815A1 publication Critical patent/EA202192815A1/ru

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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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    • 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
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
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    • C07K14/4708—Duchenne dystrophy
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    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)

Abstract

В изобретении раскрываются терапевтические мишени для коррекции гена дистрофина человека посредством редактирования гена и способы их применения.
EA202192815A 2019-04-14 2020-04-14 Опосредуемая aav-вектором делеция крупной мутационной "горячей точки" для лечения мышечной дистрофии дюшенна EA202192815A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962833760P 2019-04-14 2019-04-14
PCT/US2020/028148 WO2020214609A1 (en) 2019-04-14 2020-04-14 Aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy

Publications (1)

Publication Number Publication Date
EA202192815A1 true EA202192815A1 (ru) 2022-01-24

Family

ID=72837591

Family Applications (1)

Application Number Title Priority Date Filing Date
EA202192815A EA202192815A1 (ru) 2019-04-14 2020-04-14 Опосредуемая aav-вектором делеция крупной мутационной "горячей точки" для лечения мышечной дистрофии дюшенна

Country Status (17)

Country Link
US (1) US20220184229A1 (ru)
EP (1) EP3931331A4 (ru)
JP (1) JP2022529631A (ru)
KR (1) KR20210151916A (ru)
CN (1) CN113785063A (ru)
AR (1) AR118670A1 (ru)
AU (1) AU2020257898A1 (ru)
BR (1) BR112021020515A2 (ru)
CA (1) CA3137248A1 (ru)
CL (1) CL2021002680A1 (ru)
CO (1) CO2021013692A2 (ru)
EA (1) EA202192815A1 (ru)
IL (1) IL287163A (ru)
MX (1) MX2021012511A (ru)
SG (1) SG11202111172QA (ru)
TW (1) TW202104594A (ru)
WO (1) WO2020214609A1 (ru)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3597741A1 (en) 2012-04-27 2020-01-22 Duke University Genetic correction of mutated genes
ES2929110T3 (es) 2015-08-25 2022-11-24 Univ Duke Composiciones y métodos para mejorar la especificidad en ingeniería genética usando endonucleasas guiadas por ARN
US11970710B2 (en) 2015-10-13 2024-04-30 Duke University Genome engineering with Type I CRISPR systems in eukaryotic cells
WO2021127057A1 (en) * 2019-12-16 2021-06-24 President And Fellows Of Harvard College Methods and compositions for correction of dmd mutations
EP4232152A1 (en) * 2020-10-21 2023-08-30 Duke University Dual aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy
AU2022344251A1 (en) * 2021-09-08 2024-03-28 Flagship Pioneering Innovations Vi, Llc Serpina-modulating compositions and methods

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20230136697A (ko) * 2013-06-05 2023-09-26 듀크 유니버시티 Rna-가이드 유전자 편집 및 유전자 조절
EP3384055A4 (en) * 2015-11-30 2019-04-17 Duke University THERAPEUTIC TARGETS FOR CORRECTING HUMAN DYSTROPHINE BY GENE EDITING AND METHOD OF USE
CA3048963A1 (en) * 2016-02-11 2017-08-17 The Regents Of The University Of California Methods and compositions for modifying a mutant dystrophin gene in a cell's genome
WO2019067786A1 (en) * 2017-09-29 2019-04-04 The Regents Of The University Of California MULTI-ARM POLYROTAXANE PLATFORM FOR PROTECTED ADMINISTRATION OF NUCLEIC ACIDS

Also Published As

Publication number Publication date
US20220184229A1 (en) 2022-06-16
CL2021002680A1 (es) 2022-07-15
JP2022529631A (ja) 2022-06-23
BR112021020515A2 (pt) 2022-01-04
MX2021012511A (es) 2022-01-06
EP3931331A1 (en) 2022-01-05
WO2020214609A1 (en) 2020-10-22
CO2021013692A2 (es) 2022-01-17
EP3931331A4 (en) 2022-11-30
AR118670A1 (es) 2021-10-20
CA3137248A1 (en) 2020-10-22
IL287163A (en) 2021-12-01
AU2020257898A1 (en) 2021-11-04
SG11202111172QA (en) 2021-11-29
KR20210151916A (ko) 2021-12-14
CN113785063A (zh) 2021-12-10
TW202104594A (zh) 2021-02-01

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