Molecular Analysis of T Lymphocyte-Directed Gene Therapy for Adenosine Deaminase Deficiency: Long-Term Expression In Vivo of Genes Introduced with a …
CA Mullen, K Snitzer, KW Culver, RA Morgan… - Human gene …, 1996 - liebertpub.com
CA Mullen, K Snitzer, KW Culver, RA Morgan, WF Anderson, RM Blaese
Human gene therapy, 1996•liebertpub.comPeripheral blood lymphocytes from a patient with adenosine deaminase (ADA) deficiency
were transduced in vitro with a replication-defective retroviral vector containing a human
ADA-cDNA. Eighteen months after the last of a series of infusions of autologous retroviral
vector-treated cells, vector sequences were detectable in DNA isolated from peripheral
blood mononuclear cells (PBMCs), with an average copy number approaching one per cell.
Increased ADA enzyme activity reaching approximately one-quarter normal levels was …
were transduced in vitro with a replication-defective retroviral vector containing a human
ADA-cDNA. Eighteen months after the last of a series of infusions of autologous retroviral
vector-treated cells, vector sequences were detectable in DNA isolated from peripheral
blood mononuclear cells (PBMCs), with an average copy number approaching one per cell.
Increased ADA enzyme activity reaching approximately one-quarter normal levels was …
Abstract
Peripheral blood lymphocytes from a patient with adenosine deaminase (ADA) deficiency were transduced in vitro with a replication-defective retroviral vector containing a human ADA-cDNA. Eighteen months after the last of a series of infusions of autologous retroviral vector-treated cells, vector sequences were detectable in DNA isolated from peripheral blood mononuclear cells (PBMCs), with an average copy number approaching one per cell. Increased ADA enzyme activity reaching approximately one-quarter normal levels was found in this population of cells. Other evidence of long-term retroviral vector expression in vivo included neomycin phosphotransferase (NPT) activity and demonstration of persistent vector mRNA by reverse transcriptase polymerase chain reaction (RT-PCR). No evidence of spontaneous reversion of either mutant endogenous ADA allele was found.
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