Norberto Guelbert

Efficient site-specific integration of large genes in mammalian cells via continuously evolved recombinases and prime editing

Possibilities and limitations of antisense oligonucleotide therapies for the treatment of monogenic disorders

Impact of long‐term elosulfase alfa treatment on respiratory function in patients with Morquio A syndrome

Early treatment with laronidase improves clinical outcomes in patients with attenuated MPS I: a retrospective case series analysis of nine sibships