Dual Purpose Vectors for Rare Neurological Diseases: Molecular Therapy

RNA therapy: rich history, various applications and unlimited future prospects

Frontiers Delivering gene therapy for mucopolysaccharide diseases

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

Potential disease-modifying therapies for Huntington's disease: lessons learned and future opportunities - The Lancet Neurology

Frontiers Therapeutic Strategies For Tay-Sachs Disease

Intranasal delivery of mesenchymal stem cells‐derived extracellular vesicles for the treatment of neurological diseases - Herman - 2021 - STEM CELLS - Wiley Online Library

Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access - The Lancet

Frontiers Gene-Editing Technologies Paired With Viral Vectors for Translational Research Into Neurodegenerative Diseases

Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe Disease: Molecular Therapy - Methods & Clinical Development

Emergence of Small Interfering RNA-Based Gene Drugs for Various Diseases

Nonviral nanoparticle gene delivery into the CNS for neurological disorders and brain cancer applications - Yang - 2023 - WIREs Nanomedicine and Nanobiotechnology - Wiley Online Library

In vivo targeting of a variant causing vanishing white matter using CRISPR/Cas9: Molecular Therapy - Methods & Clinical Development

Coave Therapeutics – Coave Therapeutics is a clinical stage biotechnology company focused on developing life changing gene therapies in rare ocular and CNS (Central Nervous System) diseases.

Frontiers Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage Diseases

Non-Viral Vector-Mediated Gene Therapy for ALS: Challenges and Future Perspectives