Semi-automated closed system manufacturing of lentivirus gene-modified haematopoietic stem cells for gene therapy.
Nat Commun
; 7: 13173, 2016 10 20.
Article
de En
| MEDLINE
| ID: mdl-27762266
ABSTRACT
Haematopoietic stem cell (HSC) gene therapy has demonstrated potential to treat many diseases. However, current state of the art requires sophisticated ex vivo gene transfer in a dedicated Good Manufacturing Practices facility, limiting availability. An automated process would improve the availability and standardized manufacture of HSC gene therapy. Here, we develop a novel program for semi-automated cell isolation and culture equipment to permit complete benchtop generation of gene-modified CD34+ blood cell products for transplantation. These cell products meet current manufacturing quality standards for both mobilized leukapheresis and bone marrow, and reconstitute human haematopoiesis in immunocompromised mice. Importantly, nonhuman primate autologous gene-modified CD34+ cell products are capable of stable, polyclonal multilineage reconstitution with follow-up of more than 1 year. These data demonstrate proof of concept for point-of-care delivery of HSC gene therapy. Given the many target diseases for gene therapy, there is enormous potential for this approach to treat patients on a global scale.
Texte intégral:
1
Collection:
01-internacional
Base de données:
MEDLINE
Sujet principal:
Transduction génétique
/
Cellules souches hématopoïétiques
/
Thérapie génétique
/
Séparation cellulaire
/
Lentivirus
/
Laboratoire automatique
Type d'étude:
Guideline
Limites:
Animals
/
Humans
Langue:
En
Journal:
Nat Commun
Sujet du journal:
BIOLOGIA
/
CIENCIA
Année:
2016
Type de document:
Article
Pays d'affiliation:
États-Unis d'Amérique