Ontology highlight
ABSTRACT:
SUBMITTER: Kiem HP
PROVIDER: S-EPMC4448740 | biostudies-literature | 2014
REPOSITORIES: biostudies-literature
Kiem Hans-Peter HP Arumugam Paritha I PI Burtner Christopher R CR Fox Catherine F CF Beard Brian C BC Dexheimer Phillip P Adair Jennifer E JE Malik Punam P
Molecular therapy. Methods & clinical development 20141217
Safely achieving long-term engraftment of genetically modified hematopoietic stem cells (HSCs) that maintain therapeutic transgene expression is the benchmark for successful application of gene therapy for hemoglobinopathies. We used the pigtailed macaque HSC transplantation model to ascertain the long-term safety and stability of a γ-globin lentivirus vector. We observed stable gene-modified cells and fetal hemoglobin expression for 3 years. Retrovirus integration site (RIS) analysis spanning 6 ...[more]