GENE EDITING OF MONOGENIC DISORDERS IN HUMAN HEMATOPOIETIC STEM CELLS -- CORRECTION OF X-LINKED HYPER-IGM SYNDROME (XHIM)
In certain embodiments methods of treating X-Linked Hyper-IgM Syndrome (XHIM) in a mammal am provided where the methods comprise: i) providing differentiated T cells and/or stem/progenitor cells from the mammal; ii) performing a targeted insertion of a corrective CD40L cDNA at the CD40LG gene locus in said cells to provide a corrected CD40LG gene wherein said targeted insertion places said corrective CD40L cDNA downstream and operably linked to the endogenous CD40LG enhancer/promoter, and iii) introducing said cells into said mammal where said corrected CD40LG gene is expressed in a physiologically regulated manner..
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Patent| Erscheinungsjahr: |
2023 |
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| Erschienen: |
2023 |
| Enthalten in: |
Europäisches Patentamt - (2023) vom: 25. Mai Zur Gesamtaufnahme - year:2023 |
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| Sprache: |
Englisch |
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| Beteiligte Personen: |
KUO CAROLINE Y [VerfasserIn] |
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| Links: |
Volltext [kostenfrei] |
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| Anmerkungen: |
Source: www.epo.org (no modifications made), First posted: 2023-05-25, Last update posted on www.tib.eu: 2023-06-30, Last updated: 2023-07-08 |
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| Patentnummer: |
US2023158110 |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
EPA017418569 |
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| 245 | 1 | 0 | |a GENE EDITING OF MONOGENIC DISORDERS IN HUMAN HEMATOPOIETIC STEM CELLS -- CORRECTION OF X-LINKED HYPER-IGM SYNDROME (XHIM) |
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| 520 | |a In certain embodiments methods of treating X-Linked Hyper-IgM Syndrome (XHIM) in a mammal am provided where the methods comprise: i) providing differentiated T cells and/or stem/progenitor cells from the mammal; ii) performing a targeted insertion of a corrective CD40L cDNA at the CD40LG gene locus in said cells to provide a corrected CD40LG gene wherein said targeted insertion places said corrective CD40L cDNA downstream and operably linked to the endogenous CD40LG enhancer/promoter, and iii) introducing said cells into said mammal where said corrected CD40LG gene is expressed in a physiologically regulated manner. | ||
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