Rare Disease Day

Gene corrected stem cells:


Autologous stem cells, that is, cells taken from the same patients where it is supposed to be used, can be useful in treatment of patients with rare genetic disorders. These cells are generally non toxic and have fewer side effects. Unfortunately the cells would have the mutant gene as these have been obtained from patients. If the genetic defect can be repaired, then the corrected stem cells can be useful in treatment. Such an approach has been recently successful for Duchene Muscular Dystrophy

Read more at: https://scienmag.com/scientists-replace-piece-of-gene-mutated-in-duchenne-muscular-dystrophy-in-effort-to-make-healthy/