Gene therapies for neurogenetic disorders
Year: 2025
Type: review
Abstract: Pathogenic variants in over 1700 genes can cause neurogenetic disorders. Monogenetic diseases are ideal targets for genetic therapies; however, the blood-brain barrier (BBB), post-mitotic neurons, and inefficient delivery platforms make gene therapies for neurogenetic diseases challenging. Following nusinersen's 2016 approval, the development of gene therapies for neurogenetic disorders has advanced rapidly, with new delivery vehicles [e.g., BBB-crossing capsids, engineered viral-like proteins, lipid nanoparticles (LNPs)] and novel therapeutic strategies (e.g., regulatory elements, novel RNA therapeutics, tRNA therapies, epigenetic and gene editing). Patient-led disease foundations have accelerated treatment development by addressing trial readiness and supporting translational research. We review the current landscape and future directions in developing gene therapies for neurogenetic disorders.
Source: Trends in Molecular Medicine
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: hybrid
Estimated APC paid: $7,030
Funders: Children's Hospital of Philadelphia, Columbia University, University of Pennsylvania, Johns Hopkins University, School of Medicine, New York University, University of California, San Francisco, NYU Grossman School of Medicine, National Institute of General Medical Sciences, National Institute of Neurological Disorders and Stroke
OpenAlex ID: W4407645971