Prevention of Cardiac, Neurologic, and Skeletal Manifestations of Murine MPS I by Either Systemic or CSF-Directed AAV9-IDUA/RGX-111 Administered at Minimal Effective Dose

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Journal

Molecular Therapy — Methods & Clinical Development

Topics

Medicine

Molecular Biology

Cellular And Molecular Neuroscience

Rheumatology

Anesthesia

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DOI

10.1016/j.omtm.2024.101369

License

CC-BY-NC-ND