Translation of gene therapy strategies for amyotrophic lateral sclerosis

Gene therapy (GT) can correct genetic deficiencies through the modulation of gene expression by gene replacement, addition, silencing, or editing. GT can be administered directly to the patient (in vivo), or in patient-derived cells > (ex vivo), which are then autologously transplanted to the patient. GT delivers nucleic acids [i.e., antisense oligonucleotides (ASOs) or siRNA to silence mRNA], or viral vectors [i.e., lentiviral vectors (LV) for local transduction or adeno-associated viral vectors (AAV) for stable, widespread, and safe gene expression]. Color code: gray represents diseased state, pink indicates therapeutic entities and their effects.