MX2021005997A - Vectores virales recombinantes y acidos nucleicos para producirlos. - Google Patents

Vectores virales recombinantes y acidos nucleicos para producirlos.

Info

Publication number
MX2021005997A
MX2021005997A MX2021005997A MX2021005997A MX2021005997A MX 2021005997 A MX2021005997 A MX 2021005997A MX 2021005997 A MX2021005997 A MX 2021005997A MX 2021005997 A MX2021005997 A MX 2021005997A MX 2021005997 A MX2021005997 A MX 2021005997A
Authority
MX
Mexico
Prior art keywords
nucleic acids
producing
same
viral vectors
recombinant viral
Prior art date
Application number
MX2021005997A
Other languages
English (en)
Spanish (es)
Inventor
Thomas Darby
David Dismuke
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 filed Critical
Publication of MX2021005997A publication Critical patent/MX2021005997A/es

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0085Brain, e.g. brain implants; Spinal cord
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurosurgery (AREA)
  • Psychology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
MX2021005997A 2018-11-21 2019-11-21 Vectores virales recombinantes y acidos nucleicos para producirlos. MX2021005997A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862770202P 2018-11-21 2018-11-21
PCT/US2019/062531 WO2020106916A1 (en) 2018-11-21 2019-11-21 Recombinant viral vectors and nucleic acids for producing the same

Publications (1)

Publication Number Publication Date
MX2021005997A true MX2021005997A (es) 2021-08-11

Family

ID=70773595

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021005997A MX2021005997A (es) 2018-11-21 2019-11-21 Vectores virales recombinantes y acidos nucleicos para producirlos.

Country Status (20)

Country Link
US (1) US20210324418A1 (https=)
EP (1) EP3883954A4 (https=)
JP (2) JP7704998B2 (https=)
KR (1) KR20210103469A (https=)
CN (1) CN113302201A (https=)
AR (1) AR117145A1 (https=)
AU (1) AU2019385506A1 (https=)
BR (1) BR112021009733A2 (https=)
CA (1) CA3120289A1 (https=)
CL (1) CL2021001327A1 (https=)
CO (1) CO2021008120A2 (https=)
EA (1) EA202191418A1 (https=)
EC (1) ECSP21044840A (https=)
IL (1) IL283344A (https=)
MX (1) MX2021005997A (https=)
PE (1) PE20211419A1 (https=)
PH (1) PH12021551155A1 (https=)
SG (1) SG11202105326WA (https=)
TW (1) TW202039533A (https=)
WO (1) WO2020106916A1 (https=)

Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107686842A (zh) * 2016-08-03 2018-02-13 南京大学 一种靶多核苷酸编辑方法及其应用
EP3740580A4 (en) 2018-01-19 2021-10-20 Duke University GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS
AU2019247748A1 (en) 2018-04-03 2020-10-08 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
EP3774852A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
JP2022551739A (ja) 2019-10-17 2022-12-13 ストライドバイオ,インコーポレイテッド ニーマン・ピック病c型の治療のためのアデノ随伴ウイルスベクター
US12611436B2 (en) 2019-10-17 2026-04-28 Sarepta Therapeutics, Inc. AAV transfer cassette
US12173304B2 (en) * 2020-07-08 2024-12-24 Neuracle Genetics Inc. Intron fragments
WO2022026410A2 (en) * 2020-07-27 2022-02-03 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
AU2021328475A1 (en) 2020-08-19 2023-03-16 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome
EP4222270A4 (en) * 2020-09-29 2025-02-26 Neuexcell Therapeutics Inc. NEUROD1-VECTOR
JP2023543360A (ja) * 2020-09-29 2023-10-13 ニューエクセル・セラピューティクス・インコーポレイテッド Dlx2ベクター
WO2022072309A1 (en) * 2020-09-29 2022-04-07 NeuExcell Therapeutics Inc. Ascl1 vector
WO2022072325A1 (en) * 2020-09-29 2022-04-07 NeuExcell Therapeutics Inc. Neurod1 combination vector
WO2022087321A1 (en) * 2020-10-21 2022-04-28 Duke University Dual aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy
CA3203975A1 (en) 2020-12-03 2022-06-09 Battelle Memorial Institute Polymer nanoparticle and dna nanostructure compositions and methods for non-viral delivery
WO2022147575A1 (en) * 2021-01-04 2022-07-07 University Of Florida Research Foundation, Incorporated Methods and compositions for treatment of friedreich's ataxia
CA3216359A1 (en) 2021-04-07 2022-10-13 Battelle Memorial Institute Rapid design, build, test, and learn technologies for identifying and using non-viral carriers
IL311499A (en) * 2021-09-17 2024-05-01 Astellas Gene Therapies Inc Frataxin gene therapy
CN119213133B (zh) 2022-05-16 2026-01-06 上海天泽云泰生物医药有限公司 用于治疗i型戊二酸尿症的重组aav载体
US20250361525A1 (en) * 2022-06-08 2025-11-27 Scribe Therapeutics Inc. Aav vectors for gene editing
EP4598590A1 (en) * 2022-10-08 2025-08-13 Lingyi Biotech Co., Ltd. Construct for enhancing gene expression
WO2024163678A2 (en) * 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
AU2024353375A1 (en) 2023-09-29 2026-04-09 Battelle Memorial Institute Polymer nanoparticle compositions for in vivo expression of polypeptides
WO2025122954A1 (en) 2023-12-08 2025-06-12 Battelle Memorial Institute Use of dna origami nanostructures for molecular information based data storage systems
CN117701638B (zh) * 2023-12-18 2024-10-18 苏州新芽基因生物技术有限公司 一种抑制残留aav载体质粒和质粒骨架转录活性的方法
WO2026019230A1 (ko) * 2024-07-15 2026-01-22 삼성바이오에피스 주식회사 스터퍼를 포함하는 재조합 aav 생산을 위한 벡터 시스템

Family Cites Families (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002081632A2 (en) * 2001-04-04 2002-10-17 The Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services Nucleic acids for transgene expression
US20090221620A1 (en) * 2008-02-20 2009-09-03 Celera Corporation Gentic polymorphisms associated with stroke, methods of detection and uses thereof
AU2014255665B2 (en) * 2013-04-18 2018-08-02 Fondazione Telethon Effective delivery of large genes by dual AAV vectors
US20160256571A1 (en) * 2013-09-30 2016-09-08 Sanofi Invention
US10947596B2 (en) 2014-05-06 2021-03-16 Dana-Farber Cancer Institute, Inc. Compositions and methods for identification, assessment, prevention, and treatment of cancer using NFS1 biomarkers and modulators
US11697825B2 (en) * 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
MX2017009336A (es) * 2015-01-16 2017-11-15 Voyager Therapeutics Inc Polinucleótidos dirigidos al sistema nervioso central.
CR20170406A (es) * 2015-02-10 2017-11-14 Genzyme Corp Arni variante
US11040113B2 (en) * 2015-03-23 2021-06-22 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical composition for the treatment and the prevention of neurological phenotype associated with Friedreich ataxia
WO2016164642A1 (en) * 2015-04-08 2016-10-13 The United States Of America, As Represented By The Secretary Of Health And Human Services Viral gene therapy as treatment for cholesterol storage disease or disorder
BR112017024514A2 (pt) * 2015-05-16 2018-07-24 Genzyme Corporation edição genética de mutações intrônicas profundas
MX390848B (es) * 2015-06-23 2025-03-21 Childrens Hospital Philadelphia Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos.
WO2017023708A1 (en) * 2015-07-31 2017-02-09 California Institute Of Technology Activity-dependent expression of nucleic acids
SG11201802408RA (en) * 2015-11-05 2018-05-30 Bamboo Therapeutics Inc Modified friedreich ataxia genes and vectors for gene therapy
MX2018007330A (es) * 2015-12-15 2019-03-14 Genzyme Corp Vectores virales adeno-asociados para tratar mucolipidosis de tipo ii.
EP4484443A3 (en) * 2016-06-29 2025-03-26 CRISPR Therapeutics AG Materials and methods for treatment of friedreich ataxia and other related disorders
CA3045335A1 (en) * 2016-12-01 2018-06-07 Universite Laval Crispr-based treatment of friedreich ataxia
WO2018204764A1 (en) * 2017-05-05 2018-11-08 Camp4 Therapeutics Corporation Identification and targeted modulation of gene signaling networks
AU2018338728B2 (en) * 2017-09-29 2025-01-02 Centre National De La Recherche Scientifique (Cnrs) Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery

Also Published As

Publication number Publication date
CO2021008120A2 (es) 2021-08-09
AU2019385506A1 (en) 2021-06-03
SG11202105326WA (en) 2021-06-29
JP2025102783A (ja) 2025-07-08
PE20211419A1 (es) 2021-08-03
EP3883954A4 (en) 2022-08-10
JP7704998B2 (ja) 2025-07-09
PH12021551155A1 (en) 2021-11-03
BR112021009733A2 (pt) 2022-01-04
CN113302201A (zh) 2021-08-24
EP3883954A1 (en) 2021-09-29
EA202191418A1 (ru) 2021-08-05
CL2021001327A1 (es) 2021-12-31
US20210324418A1 (en) 2021-10-21
WO2020106916A1 (en) 2020-05-28
AR117145A1 (es) 2021-07-14
IL283344A (en) 2021-07-29
TW202039533A (zh) 2020-11-01
JP2022508182A (ja) 2022-01-19
ECSP21044840A (es) 2021-09-30
CA3120289A1 (en) 2020-05-28
KR20210103469A (ko) 2021-08-23

Similar Documents

Publication Publication Date Title
PH12021551155A1 (en) Recombinant viral vectors and nucleic acids for producing the same
MX2022004353A (es) Casete de transferencia de aav.
MX2021006646A (es) Vector viral adenoasociado recombinante para el suministro de genes.
MX2022004352A (es) Vectores virales adenoasociados para el tratamiento de la enfermedad de niemann-pick tipo c.
PH12020551938A1 (en) Liver targeting adeno-associated viral vectors
CL2018000170A1 (es) Vectores de aav para la terapia génica de la retina y el snc (divisional de solicitud 201602713)
MX2018006840A (es) Focalizacion de peptidos para dirigir virus adenoasociados.
WO2017189964A3 (en) Compositions for the treatment of disease
PH12019500068A1 (en) Novel adeno-associated virus capsid proteins
MY190926A (en) Cpg reduced factor viii variants, compositions and methods and uses for treatment of hemostasis disorders
EP4269579A3 (en) Gene therapy constructs for treating wilson disease
MX2018005286A (es) Constructo genetico.
PH12018502361A1 (en) Gdf15 fusion proteins and uses thereof
ZA201908445B (en) Recombinant adenoviruses carrying transgenes
MX375365B (es) Metodos mejorados para purificacion de vecotres de aav recombinantes
LT3911354T (lt) Aav mediacijos genų terapija, atstatanti otoferlino geną
MX2018007234A (es) Vectores virales adenoasociados utiles en el tratamiento de atrofia muscular espinal.
WO2020093018A8 (en) A codon optimized otoferlin aav dual vector gene therapy
EP4345165A3 (en) Gene therapy for retinitis pigmentosa
WO2020214809A3 (en) Gene therapies for stargardt disease (abca4)
ZA202003416B (en) Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies
EP4055174A4 (en) TRANSGENIC CASSETTES, AAV VECTORS AND AAV VECTORS FOR EXPRESSING HUMAN CODON-OPTIMIZED SLCA1
WO2018213618A8 (en) GENE THERAPY AGAINST TUBEROUS SCLEROSIS
EP3864160A4 (en) KIR 7.1 GENE THERAPY VECTORS AND METHODS OF USING THE SAME
HK1237282A1 (en) High isomerohydrolase activity mutants of mammalian rpe65