Marco Petrillo

Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study

Darryl C. De Vivo, Enrico Bertini, Kathryn J. Swoboda et al. · Neuromuscular Disorders · 2019 · 602 citations · Full text

Spinal Disorder, Pediatrics, Nurture Study +7

A self-splicing RNA excises an intron lariat

Craig L. Peebles, Philip S. Perlman, Kirk L. Mecklenburg et al. · Cell · 1986 · 449 citations

Transcriptional Regulation, Natural Sciences, Genetics +9

Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice

Mariacarmela Allocca, Monica Doria, Marco Petrillo et al. · Journal of Clinical Investigation · 2008 · 282 citations · Full text

Ocular Disease, Large Genes, In Vivo Gene Therapy +20

Novel Adeno-Associated Virus Serotypes Efficiently Transduce Murine Photoreceptors

Mariacarmela Allocca, Claudio Mussolino, Maria García‐Hoyos et al. · Journal of Virology · 2007 · 234 citations · Full text

Neurofilament as a potential biomarker for spinal muscular atrophy

Basil T. Darras, Thomas O. Crawford, Richard S. Finkel et al. · Annals of Clinical and Translational Neurology · 2019 · 200 citations · Full text