Question

Patient was treated with autologous cd34+ hematopoietic stem and progenitor cells transduced ex Vivo with self inactivating lentiviral vector encoding human ada. I can't understand what it is saying at all.​

Answer 1

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.

What is gene therapy?

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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