The technique makes reference to a vector-based gene therapy in which the expression for a gene associated with Adenosine Deaminase Deficiency (ADA) is restored.
Gene therapy refers to the genetic engineering methodologies to correct the defective expression of certain genes.
Gene therapy is divided into methods that use viral vectors to incorporate the defective nucleic acid (in this case lentiviral vector) or non-based vectors (e.g. polysomes or polycations).
In conclusion, the technique is a vector-based gene therapy to restore Adenosine Deaminase Deficiency (ADA).
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