In recent years, genetic therapies have garnered much attention for their potential to modify the underlying pathological processes in neurological diseases. By correcting, replacing, or removing aberrant genetic material, gene therapies could revolutionize the treatment of many diseases, where symptomatic management is the only available option.
Examples of gene therapy methods include the use of CRISPR/Cas9 technologies to directly edit genetic mutations, vector-based gene therapy to deliver new genetic material, and antisense oligonucleotides (ASOs) to modulate gene expression.
The success of gene therapy in treating diseases like spinal muscular atrophy (SMA) has paved the way for similar approaches in amyotrophic lateral sclerosis (ALS), with several groundbreaking clinical trials taking place in recent years.
In this podcast, we are joined by leading neurologist Prof. Dame Pamela Shaw who will share her insights into the progress in genetic therapies for ALS and explain the challenges being faced, offering a glimpse into the future ALS therapy landscape.
With Prof. Dame Pamela Shaw, DBE, MBBS, MD, FRCP, FMedSci, FAAN, FANA, FAAAS, University of Sheffield, Sheffield, UK.
