Cell and gene therapy approaches for rare neurological disorders are gaining momentum and are here to stay. A recent review article authored by LES Life Sciences Sector member, Booma Yandava, M.S., MBA, Principal at Alliance Partners, provides an overview of the advancements in cell and gene therapy (CGT) for neurological disorders, emphasizing the challenges posed by the complexity of the central nervous system and the innovative approaches being developed to overcome them. Key advancements include the use of improved adeno-associated virus (AAV) vectors, RNA-based therapies, and CRISPR/Cas9 gene-editing techniques. The article emphasizes that while significant progress has been made in gene and cell therapy for neurological disorders, there are still considerable challenges to overcome. The future of gene therapy in this field depends on integrating innovative technologies like CRISPR/Cas9, a deeper understanding of molecular mechanisms, and a holistic approach to patient care to develop more accurate diagnoses and effective treatments.
