IL323349A - טיפול גנטי - Google Patents

טיפול גנטי

Info

Publication number
IL323349A
IL323349A IL323349A IL32334925A IL323349A IL 323349 A IL323349 A IL 323349A IL 323349 A IL323349 A IL 323349A IL 32334925 A IL32334925 A IL 32334925A IL 323349 A IL323349 A IL 323349A
Authority
IL
Israel
Prior art keywords
gene therapy
therapy
gene
Prior art date
Application number
IL323349A
Other languages
English (en)
Original Assignee
Ospedale San Raffaele Srl
Consiglio Nazionale Ricerche
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 Ospedale San Raffaele Srl, Consiglio Nazionale Ricerche filed Critical Ospedale San Raffaele Srl
Publication of IL323349A publication Critical patent/IL323349A/he

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • 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
    • A61K48/005Medicinal 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
    • 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
    • A61K48/0075Medicinal 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
    • 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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • 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
    • 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
    • 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
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/50Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
IL323349A 2023-03-22 2025-09-14 טיפול גנטי IL323349A (he)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP23163561 2023-03-22
PCT/EP2024/057868 WO2024194491A1 (en) 2023-03-22 2024-03-22 Gene therapy

Publications (1)

Publication Number Publication Date
IL323349A true IL323349A (he) 2025-11-01

Family

ID=85771989

Family Applications (1)

Application Number Title Priority Date Filing Date
IL323349A IL323349A (he) 2023-03-22 2025-09-14 טיפול גנטי

Country Status (4)

Country Link
AU (1) AU2024241563A1 (he)
IL (1) IL323349A (he)
MX (1) MX2025010896A (he)
WO (1) WO2024194491A1 (he)

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2153654T3 (es) 1996-10-17 2001-03-01 Oxford Biomedica Ltd Vectores retrovirales.
GB9803351D0 (en) 1998-02-17 1998-04-15 Oxford Biomedica Ltd Anti-viral vectors
GB0009760D0 (en) 2000-04-19 2000-06-07 Oxford Biomedica Ltd Method
EP2213738B1 (en) * 2002-11-14 2012-10-10 Dharmacon, Inc. siRNA molecules targeting Bcl-2
EP3561062A1 (en) 2013-09-13 2019-10-30 California Institute of Technology Selective recovery
CN108699565B (zh) 2015-12-11 2023-08-08 加州理工学院 用于定向腺相关病毒(aav)的靶向肽
SG11201911737PA (en) * 2017-06-06 2020-01-30 Univ Massachusetts Self-regulating aav vectors for safe expression of mecp2 in rett syndrome
CA3110289A1 (en) * 2018-08-30 2020-03-05 The University Of North Carolina At Chapel Hill Feedback enabled synthetic genes, target seed match cassettes, and their uses
GB201905301D0 (en) * 2019-04-15 2019-05-29 Ospedale San Raffaele Srl Gene therapy
CN119907694A (zh) * 2022-07-08 2025-04-29 圣拉斐尔医院有限责任公司 转基因盒

Also Published As

Publication number Publication date
AU2024241563A1 (en) 2025-10-09
WO2024194491A1 (en) 2024-09-26
MX2025010896A (es) 2025-12-01

Similar Documents

Publication Publication Date Title
GB202003618D0 (en) Gene Therapy
GB202114972D0 (en) Gene therapy
IL309742A (he) קלטות ביטוי אופטימליות לריפוי גני
CA221425S (en) Therapy eye-mask
GB201905301D0 (en) Gene therapy
GB202206346D0 (en) Gene therapy
GB202010894D0 (en) Gene therapy
GB202003109D0 (en) Gene therapy
GB202002202D0 (en) Gene therapy
GB202002639D0 (en) Therapy
IL323349A (he) טיפול גנטי
GB202316264D0 (en) Gene therapy
GB202315668D0 (en) Gene therapy
CA3264521A1 (en) GENE THERAPY
GB202214445D0 (en) Gene therapy
GB202212092D0 (en) Gene therapy
IL306119A (he) טיפול גנטי לטיפול בבטא-המוגלובינופתיות
IL310018A (he) ריפוי גנים kcnv2
GB202114973D0 (en) Gene therapy
GB202003536D0 (en) Gene therapy
GB202003120D0 (en) Gene therapy
GB202001930D0 (en) Gene therapy
GB202506402D0 (en) Gene therapy
SG11202110844UA (en) Mixed-cell gene therapy
CA3265741A1 (en) GENE THERAPY TREATMENT