Dual Purpose Vectors for Rare Neurological Diseases: Molecular Therapy

Illustration of gene therapy approaches

Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff Mice: Molecular Therapy

Gene therapy comes of age

Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation: Molecular Therapy - Methods & Clinical Development

Frontiers AAV-mediated gene therapy: Advancing cardiovascular disease treatment

Viral Vector and gene therapy basics summarized - Biovian

Amplifying gene expression with RNA-targeted therapeutics

IJMS, Free Full-Text

GENE TARGET: A framework for evaluating Mendelian neurodevelopmental disorders for gene therapy: Molecular Therapy - Methods & Clinical Development

Advances in the diagnosis of inherited neuromuscular diseases and implications for therapy development - The Lancet Neurology

Gene-therapy-ready-comeback

Network analysis reveals rare disease signatures across multiple levels of biological organization

Frontiers Advanced genetic therapies for the treatment of Rett syndrome: state of the art and future perspectives

Biology, Free Full-Text