Dual Purpose Vectors for Rare Neurological Diseases: Molecular Therapy
Por um escritor misterioso
Last updated 01 abril 2025


Frontiers Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord

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

Gene therapy clinical trials, where do we go? An overview - ScienceDirect

The intersection of vector biology, gene therapy, and hemophilia - Research and Practice in Thrombosis and Haemostasis

AAV vectors: The Rubik's cube of human gene therapy: Molecular Therapy

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

Pharmaceutics, Free Full-Text

Gene Therapy Preserves Retinal Structure and Function in a Mouse Model of NMNAT1-Associated Retinal Degeneration: Molecular Therapy - Methods & Clinical Development

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