Table 2 Engraftment, vector copy number, cytometry, gene editing status and deep sequencing results from transplant.
From: Molecular Evidence of Genome Editing in a Mouse Model of Immunodeficiency
Observations | Bone marrow qPCR | Cytometry | Prkdc gene editing assessed by deep sequencing (% corrected sequences in genomic DNA) | |||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
Engraftment | VCN | Blood CD3+ | Thymus CD4+CD8+ | Spleen CD3+ proliferation | Status | Blood | Thymus | Spleen | Spleen CD8+ | Spleen CD3+ | ||
Primary Transplant | ||||||||||||
Balb/c | ||||||||||||
1.2 | secondary transplant donor | 93.1% | NA | 0.00% | ||||||||
scid IPLV-eGFP | ||||||||||||
2.2 | secondary transplant donor | 100.0% | 0.80 | |||||||||
scid-IPLV-ZFN | ||||||||||||
3.2 | secondary transplant donor | 95.5% | 0.48 | + | + | + | 5.42%, 5.80% | 13.73%, 11.81%, 0.39% | 0.45% | 31.70%, 22.18% | ||
3.3 | splenomegaly | 27.2% | 0.14 | − | + | + | 0.10% | 0.01% | ||||
3.4 | 47.9% | 1.12 | − | + | − | 0.00% | 0.00% | |||||
scid-IDLV-ZFN | ||||||||||||
4.1 | 89.0% | 0.02 | − | + | + | 0.00% | 0.02%, 0.07% | |||||
4.2 | splenomegaly | − | − | − | 0.00% | 0.00% | ||||||
4.3 | secondary transplant donor | 62.4% | 0.01 | + | + | + | 0.01% | 0.00% | 0.03% | 0.34% | ||
4.4 | − | − | − | 0.00% | 0.00% | |||||||
4.5 | − | − | + | 0.00% | 0.28% | |||||||
Secondary Transplant | ||||||||||||
Balb/c | ||||||||||||
1.2.1 | donor: 1.2 | 64.6% | 0.57 | |||||||||
scid IPLV-eGFP | ||||||||||||
2.2.1 | donor: 2.2 | 25.1% | 0.14 | |||||||||
2.2.2. | donor: 2.2; leukemia | 57.2% | 0.20 | |||||||||
scid-IPLV-ZFN | ||||||||||||
3.2.1 | donor: 3.2 | 30.0% | 0.64 | + | + | 2.70% | ||||||
3.2.2 | donor: 3.2 | 77.3% | 0.69 | + | + | + | 1.58% | 0.93% | ||||
3.2.3 | donor: 3.2 | 35.9% | 0.50 | + | + | + | 3.41%, 7.44% | 8.77% | 24.58% | |||
scid-IDLV-ZFN | ||||||||||||
4.3.1 | donor: 4.3 | 4.2% | 0.01 | −/+ | −/+ | + | + | 4.92% | 0.00% | 0.00% | 0.00% | |
4.3.2 | donor: 4.3 | 45.8% | 0.01 | −/+ | + | + | 0.01% | 0.00% | 0.00% | |||
4.3.3 | donor: 4.3; thymomegaly | 5.5% | 0.01 | + | + | − | 0.00% | 0.00% | 0.00% | |||
