Gene therapies for neurogenetic disorders

The discovery of monogenetic neurological disorders far outpaces the development of genetic treatments due to challenges in delivery, biodistribution, safety, efficacy, and costs. 

The need to cross the blood–brain barrier, the post-mitotic nature of neurons, and inefficient delivery platforms limit gene therapies for neurological diseases.

Genetic approaches such as RNA therapeutics, gene replacement, and gene editing are being investigated to treat neurogenetic diseases.

Neurogenetic therapies require a tailored approach for different diseases and potentially for different patient subgroups.

Innovation in regulatory and commercial pathways is needed to advance the translation and reduce the time and cost of developing gene therapies for neurogenetic disorders.

https://www.cell.com/trends/molecular-medicine/fulltext/S1471-4914(25)00015-2

https://sciencemission.com/Gene-therapies-for-neurogenetic-disorders