Skip to main content

Table 3 Overview over the preclinical in-vivo safety studies of ABCB5+ LSCs

From: Process development and safety evaluation of ABCB5+ limbal stem cells as advanced-therapy medicinal product to treat limbal stem cell deficiency

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
  1. LSC limbal stem cell, LSCD limbal stem cell deficiency, HRG Ringer’s lactate solution human serum albumin and glucose, BSS balanced salt solution