CA3041668A1 - 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
CA3041668A1
CA3041668A1 CA3041668A CA3041668A CA3041668A1 CA 3041668 A1 CA3041668 A1 CA 3041668A1 CA 3041668 A CA3041668 A CA 3041668A CA 3041668 A CA3041668 A CA 3041668A CA 3041668 A1 CA3041668 A1 CA 3041668A1
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cell
gene
dna
il2rg
nuclease
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CA3041668A
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French (fr)
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Anthony Conway
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Sangamo Therapeutics Inc
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Sangamo Therapeutics Inc
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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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    • A61K35/545—Embryonic stem cells; Pluripotent stem cells; Induced pluripotent stem cells; Uncharacterised stem cells
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    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • A—HUMAN NECESSITIES
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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
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    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16611—Simplexvirus, e.g. human herpesvirus 1, 2
    • C12N2710/16632—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16611—Simplexvirus, e.g. human herpesvirus 1, 2
    • C12N2710/16641—Use of virus, viral particle or viral elements as a vector
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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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  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Developmental Biology & Embryology (AREA)
  • Reproductive Health (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Transplantation (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Mycology (AREA)
  • Gynecology & Obstetrics (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CA3041668A 2016-10-31 2017-10-31 Gene correction of scid-related genes in hematopoietic stem and progenitor cells Pending CA3041668A1 (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)

Publication Number Publication Date
CA3041668A1 true CA3041668A1 (en) 2018-05-03

Family

ID=62025525

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CA3041668A Pending CA3041668A1 (en) 2016-10-31 2017-10-31 Gene correction of scid-related genes in hematopoietic stem and progenitor cells

Country Status (6)

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

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
CN115279421A (zh) * 2020-01-10 2022-11-01 坚固生物科技公司 用于联合疗法的病毒载体

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