Table 3 Overview over the preclinical in-vivo safety studies of ABCB5+ LSCs
Mouse strain | Number of animals | Model | Cell dose | Route and time of application | Observation period |
|---|---|---|---|---|---|
Local biodistribution study | |||||
 NSG | 10 (5 males, 5 females) | Corneal and limbal debridement [23] | 5000 ABCB5+ LSCs in fibrin gel | Topical, day 0 | 8 weeks |
Systemic biodistribution study | |||||
 NSG | 30 (15 males, 15 females) | healthy | 0.5 × 106 ABCB5+ LSCs in HRG solution | Subconjunctival, day 0 | 1 week (n = 10) |
12 weeks (n = 10) | |||||
20 weeks (n = 10) | |||||
Single-dose local toxicity study | |||||
 NSG | 20 (10 males, 10 females) | Corneal and limbal debridement [23] | 5000 ABCB5+ LSCs in fibrin gel (n = 10) | Topical, day 0 | 8 weeks |
Carrier (fibrin gel) only (n = 10) | |||||
Systemic repeated-dose toxicity and tumorigenicity study | |||||
 NSG | 50 (25 males, 25 females) | healthy | 0.5 × 106 ABCB5+ LSCs in HRG solution (n = 20) | Subconjunctival, days 0, 14, 28 | 20 weeks |
Vehicle (HRG solution) only (n = 20) | Subconjunctival, days 0, 14, 28 | ||||
1.25 × 104 HeLa cells in BSS solution (n = 10) | Subconjunctival, day 0 | ||||