LVIP02-LC2-2-BE1
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(only basic data is shown)
General
Cell Line |
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| hPSCreg name | LVPEIi005-A-1 |
| Cite as: | LVPEIi005-A-1 |
| Alternative name(s) |
LVIP02-LC2-2-BE1
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| Cell line type | Human induced pluripotent stem cell (hiPSC) |
| Similar lines | No similar lines found. |
| Last update | 21st August 2024 |
| Notes | This patient specific induced pluripotent stem cell (iPSC) line is a sub-clone of the parental LCA2 patient-specific iPSC line (LVPEIi005-A), that has been gene edited to correct the pathogenic mutation, using the en31-FnCas9-ABE construct. The parental iPSC line carried a homozygous missense mutation in exon 9 of RPE65 (c.992G>A; p.Trp331Ter), which was then base edited and corrected as RPE65 (c.992A>G; p.Ter331Trp). This mutation corrected iPSC line has been stably expanded post-editing and genotyping and were characterized after passage 10. The line maintains a normal karyotype and expresses the stem cell markers OCT4, SOX2, NANOG, KLF4 and SSEA4. It also efficiently differentiates into all three germ cell lineages and formed retinal organoids and mature retinal pigmented epithelial (RPE) cells. The full length RPE65 protein expression is fully restored to normal levels in these mature iPSC-RPE cells. |
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Provider |
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| Generator | L.V. Prasad Eye Institute (LVPEI) |
General Information |
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hIPSC Derivation
General |
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The source cell information can be found in the parental cell line
LVPEIi005-A.
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