Gene Therapy in ALS and SMA: Advances, Challenges and Perspectives.

ABSTRACT: Gene therapy is defined as the administration of genetic material to modify, manipulate gene expression or alter the properties of living cells for therapeutic purposes. Recent advances and improvements in this field have led to many breakthroughs in the treatment of various diseases. As a result, there has been an increasing interest in the use of these therapies to treat motor neuron diseases (MNDs), for which many potential molecular targets have been discovered. MNDs are neurodegenerative disorders that, in their most severe forms, can lead to respiratory failure and death, for instance, spinal muscular atrophy (SMA) or amyotrophic lateral sclerosis (ALS). Despite the fact that SMA has been known for many years, it is still one of the most common genetic diseases causing infant mortali

SUBMITTER: Lejman J 

PROVIDER: S-EPMC9860634 | biostudies-literature | 2023 Jan

REPOSITORIES: biostudies-literature