MA44681A - Administration de b-sarcoglycane et de microarn-29 à l'aide d'un vecteur de virus adéno-associé, et traitement de la dystrophie musculaire - Google Patents

Administration de b-sarcoglycane et de microarn-29 à l'aide d'un vecteur de virus adéno-associé, et traitement de la dystrophie musculaire

Info

Publication number
MA44681A
MA44681A MA044681A MA44681A MA44681A MA 44681 A MA44681 A MA 44681A MA 044681 A MA044681 A MA 044681A MA 44681 A MA44681 A MA 44681A MA 44681 A MA44681 A MA 44681A
Authority
MA
Morocco
Prior art keywords
sarcoglycane
microarn
adeno
administration
treatment
Prior art date
Application number
MA044681A
Other languages
English (en)
French (fr)
Inventor
Jerry R Mendell
Louise Rodino-Klapac
Original Assignee
Res Inst Nationwide Childrens Hospital
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Res Inst Nationwide Childrens Hospital filed Critical Res Inst Nationwide Childrens Hospital
Publication of MA44681A publication Critical patent/MA44681A/fr

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    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • 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
    • C07K14/4716—Muscle proteins, e.g. myosin, actin
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • C—CHEMISTRY; METALLURGY
    • 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
    • C—CHEMISTRY; METALLURGY
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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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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    • 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
    • C12N15/86—Viral vectors
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
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    • C12N5/0656—Adult fibroblasts
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0681—Cells of the genital tract; Non-germinal cells from gonads
    • C12N5/0682—Cells of the female genital tract, e.g. endometrium; Non-germinal cells from ovaries, e.g. ovarian follicle cells
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    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0684—Cells of the urinary tract or kidneys
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
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    • C12N5/0693—Tumour cells; Cancer cells
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    • C12N2510/00—Genetically modified cells
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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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    • 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
    • C12N2750/14145—Special targeting system for viral vectors
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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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  • General Health & Medical Sciences (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Cell Biology (AREA)
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  • Neurosurgery (AREA)
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MA044681A 2016-04-15 2017-04-14 Administration de b-sarcoglycane et de microarn-29 à l'aide d'un vecteur de virus adéno-associé, et traitement de la dystrophie musculaire MA44681A (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662323333P 2016-04-15 2016-04-15
US201662433548P 2016-12-13 2016-12-13

Publications (1)

Publication Number Publication Date
MA44681A true MA44681A (fr) 2019-02-20

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MA044681A MA44681A (fr) 2016-04-15 2017-04-14 Administration de b-sarcoglycane et de microarn-29 à l'aide d'un vecteur de virus adéno-associé, et traitement de la dystrophie musculaire

Country Status (28)

Country Link
US (3) US11358993B2 (enExample)
EP (2) EP3442600B1 (enExample)
JP (2) JP7079733B2 (enExample)
KR (1) KR102502091B1 (enExample)
CN (2) CN117384964A (enExample)
AU (2) AU2017250756B2 (enExample)
BR (1) BR112018071167A2 (enExample)
CA (1) CA3021057A1 (enExample)
CO (1) CO2018012082A2 (enExample)
DK (1) DK3442600T5 (enExample)
ES (1) ES2979174T3 (enExample)
FI (1) FI3442600T3 (enExample)
HR (1) HRP20240580T1 (enExample)
HU (1) HUE066442T2 (enExample)
IL (1) IL262262B2 (enExample)
LT (1) LT3442600T (enExample)
MA (1) MA44681A (enExample)
MX (2) MX391208B (enExample)
MY (1) MY195439A (enExample)
PL (1) PL3442600T3 (enExample)
PT (1) PT3442600T (enExample)
RS (1) RS65490B1 (enExample)
SA (1) SA518400245B1 (enExample)
SG (1) SG11201809029RA (enExample)
SI (1) SI3442600T1 (enExample)
SM (1) SMT202400169T1 (enExample)
WO (1) WO2017180976A1 (enExample)
ZA (1) ZA201806863B (enExample)

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WO2019012336A2 (en) 2017-03-17 2019-01-17 Newcastle University ADENO-ASSOCIATED VIRAL VECTOR DELIVERY OF A MICRO-DYSTROPHIN FRAGMENT FOR TREATING MUSCLE DYSTROPHY
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US12391928B2 (en) * 2018-06-29 2025-08-19 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2A
EP3598978B1 (en) 2018-07-26 2024-05-29 EXOFIX S.r.l. Fibroadipogenic progenitor-derived exosomes for regeneration of dystrophic muscles
US20220119489A1 (en) * 2019-01-25 2022-04-21 Children's Medical Center Corporation Compositions and methods for treating muscular dystrophy and related disorders
CA3146364A1 (en) * 2019-02-02 2020-08-06 University Of Pittsburgh-Of The Commonwealth System Of Higher Education One-step gene therapy for duchenne muscular dystrophy via gene replacement and anti-inflammation
CN113766935A (zh) 2019-02-26 2021-12-07 全国儿童医院研究所 β-肌聚糖的腺相关病毒载体递送和肌营养不良症的治疗
SI4017871T1 (sl) * 2019-08-21 2024-06-28 Research Institute At Nationwide Children's Hospital Dostava alfa-sarkoglikana z adeno povezanim virusnim vektorjem in zdravljenje mišične distrofije
US20230040544A1 (en) * 2019-12-16 2023-02-09 Research Institute At Nationwide Children's Hospital Compositions and methods for restoring and maintaining the dystrophin-associated protein complex (dapc)
WO2021255245A2 (en) * 2020-06-19 2021-12-23 Genethon Gene therapy expression system allowing an adequate expression in the muscles and in the heart of sgcg
EP4219726A1 (en) 2021-10-15 2023-08-02 Research Institute at Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
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