Molecular genetic approaches in the treatment of amyotrophic lateral sclerosis

Abstract

Amyotrophic lateral sclerosis is a complex and fatal neurological disorder whose underlying mechanisms are not fully understood, and there are no effective treatments available to slow or stop its progression. However, recent progress in ALS genomics has connected specific genes with observable characteristics, leading to the development of innovative therapeutic strategies and providing researchers with valuable tools like genetically modified rodent models that mimic ALS pathology.