Cellular and molecular approaches to motor neuron therapy in amyotrophic lateral sclerosis and spinal muscular atrophy

Amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA) are progressive fatal neurodegenerative diseases. They differ in their disease development but have in common a loss of motor neuron as they progress. Research is ongoing to further understand the origin of these diseases but this common thread of motor neuron loss has provided a target for the development of therapies for both ALS and SMA. It is the linked fields of gene and cell therapy that are providing some of the most interesting therapeutic possibilities.

► This review covers the research in the areas of gene and cell therapy for SMA and ALS.
► It highlights the advances in delivery routes and the pros and cons of each.
► The translation into clinical trial of therapies are reviewed.
► The progress on the current ALS stem cell trial is discussed.