Ontology highlight
ABSTRACT:
SUBMITTER: Carbonaro DA
PROVIDER: S-EPMC3944341 | biostudies-literature | 2014 Mar
REPOSITORIES: biostudies-literature
Carbonaro Denise A DA Zhang Lin L Jin Xiangyang X Montiel-Equihua Claudia C Geiger Sabine S Carmo Marlene M Cooper Aaron A Fairbanks Lynette L Kaufman Michael L ML Sebire Neil J NJ Hollis Roger P RP Blundell Michael P MP Senadheera Shantha S Fu Pei-Yu PY Sahaghian Arineh A Chan Rebecca Y RY Wang Xiaoyan X Cornetta Kenneth K Thrasher Adrian J AJ Kohn Donald B DB Gaspar H Bobby HB
Molecular therapy : the journal of the American Society of Gene Therapy 20131120 3
Gene transfer into autologous hematopoietic stem cells by γ-retroviral vectors (gRV) is an effective treatment for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID). However, current gRV have significant potential for insertional mutagenesis as reported in clinical trials for other primary immunodeficiencies. To improve the efficacy and safety of ADA-SCID gene therapy (GT), we generated a self-inactivating lentiviral vector (LV) with a codon-optimized human cADA gene un ...[more]