Process development and safety evaluation of ABCB5
ABCB5
Advanced-therapy medicinal product
GMP manufacturing
Limbal stem cell deficiency
Limbal stem cells
PAX6
p63
Journal
Stem cell research & therapy
ISSN: 1757-6512
Titre abrégé: Stem Cell Res Ther
Pays: England
ID NLM: 101527581
Informations de publication
Date de publication:
19 03 2021
19 03 2021
Historique:
received:
06
10
2020
accepted:
08
03
2021
entrez:
20
3
2021
pubmed:
21
3
2021
medline:
9
7
2021
Statut:
epublish
Résumé
While therapeutic success of the limbal tissue or cell transplantation to treat severe cases of limbal stem cell (LSC) deficiency (LSCD) strongly depends on the percentage of LSCs within the transplanted cells, prospective LSC enrichment has been hampered by the intranuclear localization of the previously reported LSC marker p63. The recent identification of the ATP-binding cassette transporter ABCB5 as a plasma membrane-spanning marker of LSCs that are capable of restoring the cornea and the development of an antibody directed against an extracellular loop of the ABCB5 molecule stimulated us to develop a novel treatment strategy based on the utilization of in vitro expanded allogeneic ABCB5 We developed and validated a Good Manufacturing Practice- and European Pharmacopeia-conform production and quality-control process, by which ABCB5 ABCB5 Building upon these data in conjunction with the previously shown cornea-restoring capacity of human ABCB5
Sections du résumé
BACKGROUND
While therapeutic success of the limbal tissue or cell transplantation to treat severe cases of limbal stem cell (LSC) deficiency (LSCD) strongly depends on the percentage of LSCs within the transplanted cells, prospective LSC enrichment has been hampered by the intranuclear localization of the previously reported LSC marker p63. The recent identification of the ATP-binding cassette transporter ABCB5 as a plasma membrane-spanning marker of LSCs that are capable of restoring the cornea and the development of an antibody directed against an extracellular loop of the ABCB5 molecule stimulated us to develop a novel treatment strategy based on the utilization of in vitro expanded allogeneic ABCB5
METHODS
We developed and validated a Good Manufacturing Practice- and European Pharmacopeia-conform production and quality-control process, by which ABCB5
RESULTS
ABCB5
CONCLUSION
Building upon these data in conjunction with the previously shown cornea-restoring capacity of human ABCB5
Identifiants
pubmed: 33741066
doi: 10.1186/s13287-021-02272-2
pii: 10.1186/s13287-021-02272-2
pmc: PMC7980611
doi:
Substances chimiques
ABCB5 protein, human
0
ATP Binding Cassette Transporter, Subfamily B
0
Types de publication
Journal Article
Research Support, N.I.H., Extramural
Langues
eng
Sous-ensembles de citation
IM
Pagination
194Subventions
Organisme : NEI NIH HHS
ID : R01 EY025794
Pays : United States
Organisme : NEI NIH HHS
ID : R24 EY028767
Pays : United States
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