Eva Also‐Rallo

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

Adeno-associated virus as a delivery vector for gene therapy of human diseases

Synaptic defects in type I spinal muscular atrophy in human development

Treatment of spinal muscular atrophy cells with drugs that upregulate SMN expression reveals inter- and intra-patient variability