A GLB1 transgene with enhanced therapeutic potential for the preclinical development of ex-vivo gene therapy to treat mucopolysaccharidosis type IVB
Article in Molecular Therapy — Methods & Clinical Development (September 2024)
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Article in Molecular Therapy — Methods & Clinical Development (September 2024)
Article in Molecular Therapy (August 2024)
Article in Indian Journal of Pediatrics (April 2024)