Abstract
Abstract: Gene editing using the CRISPR/Cas9 system provides new opportunities to treat human diseases. Approaches aimed at increasing the efficiency of genome editing are therefore important to develop. To increase the level of editing of the CXCR4 locus, which is a target for gene therapy of HIV infection, the Cas9 protein was modified by introducing additional NLS signals and ribonucleoprotein complexes of Cas9 and guide RNA were stabilized with poly-L-glutamic acid. The approach allowed a 1.8-fold increase in the level of CXCR4 knockout in the CEM/R5 T cell line and a 2-fold increase in the level of knock-in of the HIV-1 fusion peptide inhibitor MT-C34 in primary CD4+ T lymphocytes.
| Original language | English (US) |
|---|---|
| Pages (from-to) | S28-S32 |
| Journal | Doklady Biological Sciences |
| Volume | 513 |
| Issue number | Suppl 1 |
| DOIs | |
| State | Published - Dec 2023 |
Bibliographical note
Publisher Copyright:© Pleiades Publishing, Ltd. 2023. ISSN 0012-4966, Doklady Biological Sciences, 2023, Vol. 513, Suppl. 1, pp. S28–S32. Pleiades Publishing, Ltd., 2023. ISSN 0012-4966, Doklady Biological Sciences, 2024. Pleiades Publishing, Ltd., 2024.
UN SDGs
This output contributes to the following UN Sustainable Development Goals (SDGs)
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SDG 3 Good Health and Well-being
Keywords
- CRISPR/Cas9
- CXCR4
- HIV
- NLS
- PGA
- RNP
- genome editing
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