The Promising Future of Gene Therapy with Duchenne Muscular Dystrophy
Current research status
The gene therapy, originally conducted to dogs, is yet to reach its conclusion. Further clinical trials still have to be done, especially to humans, to ensure that it is both effective and safe for children with DMD. All of which will also not be possible without sufficient funds too. Currently, gene therapy research is looking into ways to resize large dystrophin gene, deliver the right amount of new genes to muscles without harming other tissues, and avoid unwanted immune system consequences to patients.
