With all the talk about it in the scientific community, there is probably no surprise that this is yet another article discussing CRISPR. However, this may be one of the most profound discussions around the technology as it involves what could arguably be the potential flagship use of the gene editing tool – human therapy.
Lately, a major step forward has been made in using CRISPR technology in neurosciences. Over the past few years, scientists have been using gene sequencing to uncover genes that are important in brain development and in neurological diseases. The next step is to figure out if disrupting these genes can cause any of these diseases.
Advances in gene editing technology have spurred considerable progress towards a treatment for Duchenne muscular dystrophy (DMD). Although the disease is rare – affecting roughly 1 in 5,000 male births – its consequences are devastating: patients are confined to wheelchairs at an early age and often succumb to heart or respiratory failure in their twenties or thirties.