What is gene therapy?

Gene therapy is a treatment that works by modifying, correcting, replacing or adding genetic instructions — the DNA inside cells. For LAMA2-RD, approaches being investigated include delivering a working copy of the LAMA2 gene or directly correcting the mutated LAMA2 gene in cells around the body, so they can make the laminin alpha-2 protein the body is missing. Gene therapy is still experimental, but it is one of the most promising areas of research.