CN117500933A - 能够实现精确的hdr介导的体内基因编辑的基于树枝状聚合物的全合一脂质纳米颗粒 - Google Patents

能够实现精确的hdr介导的体内基因编辑的基于树枝状聚合物的全合一脂质纳米颗粒 Download PDF

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CN117500933A
CN117500933A CN202280042709.0A CN202280042709A CN117500933A CN 117500933 A CN117500933 A CN 117500933A CN 202280042709 A CN202280042709 A CN 202280042709A CN 117500933 A CN117500933 A CN 117500933A
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D·J·西格沃特
L·法比亚克
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University of Texas System
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University of Texas System
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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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
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    • 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
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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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)
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
CN202280042709.0A 2021-04-22 2022-04-22 能够实现精确的hdr介导的体内基因编辑的基于树枝状聚合物的全合一脂质纳米颗粒 Pending CN117500933A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163178453P 2021-04-22 2021-04-22
US63/178,453 2021-04-22
PCT/US2022/026001 WO2022226344A1 (en) 2021-04-22 2022-04-22 All-in-one dendrimer-based lipid nanoparticles enable precise hdr-mediated gene editing in vivo

Publications (1)

Publication Number Publication Date
CN117500933A true CN117500933A (zh) 2024-02-02

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CN202280042709.0A Pending CN117500933A (zh) 2021-04-22 2022-04-22 能够实现精确的hdr介导的体内基因编辑的基于树枝状聚合物的全合一脂质纳米颗粒

Country Status (8)

Country Link
US (1) US20240207442A1 (https=)
EP (1) EP4326886A4 (https=)
JP (1) JP2024516164A (https=)
CN (1) CN117500933A (https=)
AU (1) AU2022262422A1 (https=)
CA (1) CA3215509A1 (https=)
IL (1) IL307874A (https=)
WO (1) WO2022226344A1 (https=)

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JP2024511463A (ja) 2021-03-22 2024-03-13 リコード セラピューティクス，インク． 細胞への標的送達のための組成物および方法
US20250382640A1 (en) * 2022-06-29 2025-12-18 Juno Therapeutics, Inc. Lipid nanoparticles for delivery of nucleic acids
US12364773B2 (en) 2023-12-01 2025-07-22 Recode Therapeutics, Inc. Lipid nanoparticle compositions and uses thereof
CN120754284A (zh) * 2025-07-30 2025-10-10 中国医学科学院基础医学研究所 一种用于肝脏靶向联合递送CRISPR-Cas9系统和单链DNA修复模板的脂质纳米颗粒

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CA3215509A1 (en) 2022-10-27
AU2022262422A1 (en) 2023-11-02
JP2024516164A (ja) 2024-04-12
EP4326886A1 (en) 2024-02-28
IL307874A (en) 2023-12-01
EP4326886A4 (en) 2025-04-30
AU2022262422A9 (en) 2023-11-16
US20240207442A1 (en) 2024-06-27
WO2022226344A1 (en) 2022-10-27

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