Dual Purpose Vectors for Rare Neurological Diseases: Molecular Therapy
Por um escritor misterioso
Last updated 10 março 2025


Frontiers Vectored Immunotherapeutics for Infectious Diseases: Can rAAVs Be The Game Changers for Fighting Transmissible Pathogens?
Diagram of in vivo and ex vivo gene therapy. In vivo gene therapy

The therapeutic potential of bone marrow‐derived macrophages in neurological diseases - Zhou - 2022 - CNS Neuroscience & Therapeutics - Wiley Online Library

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

Illustration of gene therapy approaches

Gene Therapy Market Size, Share & Growth Forecast, 2023-2030

Clinical Development of Gene Therapies: The First Three Decades and Counting - ScienceDirect

Nucleic acid drug vectors for diagnosis and treatment of brain diseases

Manufacturing Challenges and Rational Formulation Development for AAV Viral Vectors - Journal of Pharmaceutical Sciences

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

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

Biomedicines, Free Full-Text

Epithelioid neoplasm of the spinal cord in a child with spinal muscular atrophy treated with onasemnogene abeparvovec: Molecular Therapy

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

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