AU2017347928A1 - Gene correction of scid-related genes in hematopoietic stem and progenitor cells - Google Patents

Gene correction of scid-related genes in hematopoietic stem and progenitor cells Download PDF

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Publication number
AU2017347928A1
AU2017347928A1 AU2017347928A AU2017347928A AU2017347928A1 AU 2017347928 A1 AU2017347928 A1 AU 2017347928A1 AU 2017347928 A AU2017347928 A AU 2017347928A AU 2017347928 A AU2017347928 A AU 2017347928A AU 2017347928 A1 AU2017347928 A1 AU 2017347928A1
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Prior art keywords
cell
gene
dna
il2rg
nuclease
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AU2017347928A
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Anthony Conway
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Sangamo Therapeutics Inc
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Sangamo Therapeutics Inc
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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
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K2035/124—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood cells
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    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16111—Cytomegalovirus, e.g. human herpesvirus 5
    • C12N2710/16171—Demonstrated in vivo effect
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    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16611—Simplexvirus, e.g. human herpesvirus 1, 2
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    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16611—Simplexvirus, e.g. human herpesvirus 1, 2
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    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16611—Simplexvirus, e.g. human herpesvirus 1, 2
    • C12N2710/16671—Demonstrated in vivo effect

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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Developmental Biology & Embryology (AREA)
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  • Virology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Transplantation (AREA)
  • Gastroenterology & Hepatology (AREA)
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  • Gynecology & Obstetrics (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
AU2017347928A 2016-10-31 2017-10-31 Gene correction of scid-related genes in hematopoietic stem and progenitor cells Abandoned AU2017347928A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662415056P 2016-10-31 2016-10-31
US62/415,056 2016-10-31
PCT/US2017/059197 WO2018081775A1 (en) 2016-10-31 2017-10-31 Gene correction of scid-related genes in hematopoietic stem and progenitor cells

Publications (1)

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AU2017347928A1 true AU2017347928A1 (en) 2019-05-09

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AU2017347928A Abandoned AU2017347928A1 (en) 2016-10-31 2017-10-31 Gene correction of scid-related genes in hematopoietic stem and progenitor cells

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Country Link
US (1) US11020492B2 (https=)
EP (1) EP3532106A4 (https=)
JP (2) JP7108608B2 (https=)
AU (1) AU2017347928A1 (https=)
CA (1) CA3041668A1 (https=)
WO (1) WO2018081775A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
KR102828669B1 (ko) * 2019-11-13 2025-07-02 더 리전트 오브 더 유니버시티 오브 캘리포니아 재조합 활성화 유전자 1 (rag1) 중증 복합 면역결핍증 (scid) 을 치료하기 위한 조혈 줄기 세포에서의 렌티바이러스 벡터
EP4087617A4 (en) * 2020-01-10 2024-05-01 Solid Biosciences Inc. VIRAL VECTOR FOR COMBINATION THERAPY

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