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An extracellular vesicle delivery platform based on the PTTG1IP protein

Journal article

Martin Perez C. et al, (2024), Extracellular Vesicle, 4, 100054 - 100054

Gene therapy for spinal muscular atrophy: timing is key.

Journal article

Servais L., (2024), Lancet Reg Health Eur, 47

Examining the role of common variants in rare neurodevelopmental conditions

Journal article

Huang QQ. et al, (2024), Nature

GeM-LR: Discovering predictive biomarkers for small datasets in vaccine studies.

Journal article

Lin L. et al, (2024), PLoS Comput Biol, 20

Spinal muscular atrophy in Brazil: from individual treatment to global management.

Journal article

Servais L. and Moreno CAM., (2024), J Pediatr (Rio J)

uORF-targeting steric block antisense oligonucleotides do not reproducibly increase RNASEH1 expression

Journal article

Ahlskog N. et al, (2024), Molecular Therapy - Nucleic Acids, 102406 - 102406

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