Gene Therapy 1 article

Gene Therapy covers therapeutic delivery of genetic material to add, replace or express a gene in order to alter disease biology. In vivo and ex vivo approaches, viral and nonviral delivery, transgene expression, durability and immune responses all belong when gene transfer is the therapeutic mechanism. Genome Editing is kept separate because it changes endogenous genomic sequence, while Cell Therapy is broader and may involve therapeutic cells without gene transfer as the defining intervention.