GENOME EDITING OF HUMAN NEURAL STEM CELLS USING NUCLEASES
Abstract The invention provides methods for generating a genetically modified human neural stem cells and pharmaceutical compositions comprising the genetically modified human neural stem cells. In particular, the methods involve the use of a DNA nuclease, such as TALEN, ZFN, or CRISPR/Cas mediated genome editing. Also provided are associated kits. The invention also provides methods for preventing or treating a neurodegenerative disease or a neurological injury in a human subject using the genetically modified human neural stem cells. 'A4 * CL Cur a...
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Patent| Erscheinungsjahr: |
2023 |
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| Erschienen: |
2023 |
| Enthalten in: |
Europäisches Patentamt - (2023) vom: 17. Aug. Zur Gesamtaufnahme - year:2023 |
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| Sprache: |
Englisch |
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| Beteiligte Personen: |
PORTEUS MATTHEW H [VerfasserIn] |
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| Links: |
Volltext [kostenfrei] |
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| Anmerkungen: |
Source: www.epo.org (no modifications made), First posted: 2023-08-17, Last update posted on www.tib.eu: 2024-01-01, Last updated: 2024-01-05 |
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| Patentnummer: |
AU2023208065 |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
EPA018174787 |
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| 520 | |a Abstract The invention provides methods for generating a genetically modified human neural stem cells and pharmaceutical compositions comprising the genetically modified human neural stem cells. In particular, the methods involve the use of a DNA nuclease, such as TALEN, ZFN, or CRISPR/Cas mediated genome editing. Also provided are associated kits. The invention also provides methods for preventing or treating a neurodegenerative disease or a neurological injury in a human subject using the genetically modified human neural stem cells. 'A4 * CL Cur a.. | ||
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| 650 | 4 | |a A61K: Preparations for medical, dental, or toilet purposes (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms a61j0003000000;chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles a61l; soap compositions c11d) | |
| 650 | 4 | |a C12N: Microorganisms or enzymes; compositions thereof; propagating, preserving, or maintaining microorganisms; mutation or genetic engineering; culture media (microbiological testing media c12q0001000000) | |
| 650 | 4 | |a A61P: Specific therapeutic activity of chemical compounds or medicinal preparations | |
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