Gene therapy: Where are we? Where are we going?

Gene therapy has achieved significant advancements in the treatment of genetic diseases, especially in rare and monogenic diseases. Gene therapies have been developed and approved to treat diseases such as spinal muscular atrophy, offering hope to patients and demonstrating the effectiveness of this therapy. Currently, numerous clinical trials are being conducted to evaluate the safety and efficacy of gene therapy in various diseases, particularly in the field of pediatric neurology. These studies are generating encouraging data and contributing to the knowledge on how to improve gene therapy techniques.Despite the advancements, gene therapy faces significant challenges. It is a costly and technically complex therapy, limiting its accessibility. Additionally, aspects such as efficient gene delivery, immune response to vectors, and duration of therapeutic response require improvements and are actively being investigated. Regarding the future of gene therapy, advances in gene editing technology, such as CRISPR-Cas9, are expected to allow for greater precision and efficiency in gene modification.Research on gene therapy vectors is expected to enhance the delivery capacity and safety of treatments. New generations of viral and non-viral vectors are being developed that could overcome current limitations and enable more efficient and precise administration of therapeutic genes.