SA519401806B1 - توصيل داخل القراب لفيروس مرتبط غديا ناتج عودة الارتباط الجيني يرمز بروتين 2 ربط ميثيل -cpg - Google Patents

توصيل داخل القراب لفيروس مرتبط غديا ناتج عودة الارتباط الجيني يرمز بروتين 2 ربط ميثيل -cpg Download PDF

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SA519401806B1
SA519401806B1 SA519401806A SA519401806A SA519401806B1 SA 519401806 B1 SA519401806 B1 SA 519401806B1 SA 519401806 A SA519401806 A SA 519401806A SA 519401806 A SA519401806 A SA 519401806A SA 519401806 B1 SA519401806 B1 SA 519401806B1
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associated virus
mecp2
vector
aav
virus
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SA519401806A
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Arabic (ar)
English (en)
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كيه. كاسبر براين
فوست كيفين
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أوهايو ستيت إنوفيشين فاونديشين
ناشنوايد تشيلدرينز هوسبيتال إنك.
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Publication of SA519401806B1 publication Critical patent/SA519401806B1/ar

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61K48/0075—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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C07K14/4702—Regulators; Modulating activity
    • C—CHEMISTRY; METALLURGY
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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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
    • C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • C—CHEMISTRY; METALLURGY
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
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  • Biomedical Technology (AREA)
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  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Neurology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Dermatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychology (AREA)
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  • Marine Sciences & Fisheries (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
SA519401806A 2016-11-17 2019-05-17 توصيل داخل القراب لفيروس مرتبط غديا ناتج عودة الارتباط الجيني يرمز بروتين 2 ربط ميثيل -cpg SA519401806B1 (ar)

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US201662423618P 2016-11-17 2016-11-17

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SA519401806B1 true SA519401806B1 (ar) 2023-03-08

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US (3) US11583564B2 (enExample)
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Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL322146A (en) * 2016-11-17 2025-09-01 Nationwide Childrens Hospital Inc Intrathecal administration of adenovirus encoding methyl-CpG-binding protein 2
WO2019094253A1 (en) 2017-11-08 2019-05-16 Avexis Inc. Means and method for preparing viral vectors and uses of same
EP3801638A1 (en) 2018-06-08 2021-04-14 Novartis AG Cell-based assay for measuring drug product potency
CN113646436A (zh) * 2019-02-04 2021-11-12 全国儿童医院研究所 腺相关病毒对cln3多核苷酸的递送
AU2020366242A1 (en) * 2019-10-18 2022-05-26 Research Institute At Nationwide Children's Hospital Materials and methods for the treatment of disorders associated mutations in the IRF2BPL gene
EP4061831A1 (en) * 2019-11-22 2022-09-28 Research Institute at Nationwide Children's Hospital Materials and methods for treatment of disorders associated with the ighmbp2 gene
AU2022256458A1 (en) 2021-04-13 2023-11-16 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus encoding methyl-cpg binding protein 2 for treating pitt hopkins syndrome via intrathecal delivery
CN121175427A (zh) * 2023-05-15 2025-12-19 上海金珂博生物技术有限公司 一种Rett综合症的基因治疗

Family Cites Families (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5837484A (en) 1993-11-09 1998-11-17 Medical College Of Ohio Stable cell lines capable of expressing the adeno-associated virus replication gene
AU688428B2 (en) 1993-11-09 1998-03-12 Johns Hopkins University, The Generation of high titers of recombinant AAV vectors
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
RU2273645C9 (ru) 1994-08-17 2006-11-27 Дзе Рокефеллер Юниверсити Полипептид ожирения (ов)(варианты), его аналог (варианты) и слитый белок (варианты), изолированная молекула нуклеиновой кислоты, молекула днк, рекомбинантный вектор клонирования, рекомбинантный вектор экспрессии, фармацевтическая композиция, моноклональное и поликлональное антитело
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
WO1996017947A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
JPH11511326A (ja) 1995-08-30 1999-10-05 ジエンザイム コーポレイション アデノウィルスおよびａａｖの精製
AU715543B2 (en) 1995-09-08 2000-02-03 Genzyme Corporation Improved AAV vectors for gene therapy
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
DE69738351T2 (de) 1996-09-06 2008-11-13 The Trustees Of The University Of Pennsylvania Verfaheren zur durch rekombinante adeno-assoziierte virus-gerichtete gentherapie
CA2995542A1 (en) 1997-09-05 1999-03-11 Genzyme Corporation Methods for generating high titer helper-free preparations of recombinant aav vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
GB9916702D0 (en) * 1999-07-16 1999-09-15 Cantab Pharma Res Virus vectors and preparations and their use
CA2406743A1 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
EP1463805B1 (en) 2001-12-17 2014-10-22 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
CA2554380C (en) * 2004-02-17 2015-05-19 The Hospital For Sick Children Mecp2e1 gene
HUE046709T2 (hu) * 2006-10-03 2020-03-30 Genzyme Corp Génterápia amiotrófiás laterálszklerózis és más gerincvelõi rendellenességek kezelésére
BRPI0906490A2 (pt) 2008-01-15 2015-07-14 Abbott Lab Vetores de expressão de mamífero melhorados e uso dos mesmos
US20090246768A1 (en) * 2008-02-15 2009-10-01 Sawalha Amr H Predicting and Diagnosing Patients With Autoimmune Disease
US11219696B2 (en) * 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9
US9415121B2 (en) * 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
JP2015092462A (ja) 2013-09-30 2015-05-14 Tdk株式会社 正極及びそれを用いたリチウムイオン二次電池
JP6202701B2 (ja) 2014-03-21 2017-09-27 株式会社日立国際電気 基板処理装置、半導体装置の製造方法及びプログラム
JP6197169B2 (ja) 2014-09-29 2017-09-20 東芝メモリ株式会社 半導体装置の製造方法
JP6754361B2 (ja) * 2014-12-16 2020-09-09 ボード オブ リージェンツ オブ ザ ユニバーシティ オブ ネブラスカ 若年型バッテン病のための遺伝子療法
IL322146A (en) * 2016-11-17 2025-09-01 Nationwide Childrens Hospital Inc Intrathecal administration of adenovirus encoding methyl-CpG-binding protein 2
AU2018281145A1 (en) * 2017-06-06 2020-01-02 University Of Massachusetts Self-regulating AAV vectors for safe expression of MeCP2 in rett syndrome

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WO2018094251A1 (en) 2018-05-24
IL266678B1 (en) 2025-08-01
EP3541429A4 (en) 2020-08-12
IL266678B2 (en) 2025-12-01
JP2024045289A (ja) 2024-04-02
KR102641846B1 (ko) 2024-02-27
AU2026200191A1 (en) 2026-01-29
BR112019009834A2 (pt) 2019-10-08
CN118490845A (zh) 2024-08-16
MA46863A (fr) 2019-09-25
CA3044291A1 (en) 2018-05-24
BR112019009834B1 (pt) 2022-05-17
AU2017362491A1 (en) 2019-06-06
US12357663B2 (en) 2025-07-15
KR20190126047A (ko) 2019-11-08
US20250312400A1 (en) 2025-10-09
US11583564B2 (en) 2023-02-21
JP2020503265A (ja) 2020-01-30
EP3541429A1 (en) 2019-09-25
US20230321164A1 (en) 2023-10-12
MX2019005874A (es) 2020-02-07
CN110325219A (zh) 2019-10-11
SG10202105090WA (en) 2021-06-29
JP2022137210A (ja) 2022-09-21
AU2017362491B2 (en) 2023-02-02
IL322146A (en) 2025-09-01
JP7106534B2 (ja) 2022-07-26
US20200179467A1 (en) 2020-06-11
RU2019118300A (ru) 2020-12-17
CL2019001354A1 (es) 2019-09-13
AU2023202598A1 (en) 2023-07-20
IL266678A (en) 2019-07-31
RU2019118300A3 (enExample) 2021-01-11
CN110325219B (zh) 2024-03-26

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