Dual Purpose Vectors for Rare Neurological Diseases: Molecular Therapy

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Vectors in gene therapy - Wikipedia

Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice: Molecular Therapy - Methods & Clinical Development

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

Gene therapy comes of age

Therapeutic advantages of combined gene/cell therapy strategies in a murine model of GM2 gangliosidosis - ScienceDirect

Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders

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

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