Pierpaolo Ala

Combined Notch and PDGF Signaling Enhances Migration and Expression of Stem Cell Markers while Inducing Perivascular Cell Features in Muscle Satellite Cells

M Gerli, Louise A. Moyle, Sara Benedetti et al. · Stem Cell Reports · 2019 · 54 citations · Full text

Gapmer Antisense Oligonucleotides Suppress the Mutant Allele of COL6A3 and Restore Functional Protein in Ullrich Muscular Dystrophy

Elena Marrosu, Pierpaolo Ala, Francesco Muntoni et al. · Molecular Therapy — Nucleic Acids · 2017 · 47 citations · Full text

Mutant Allele, Protein Function, Restore Functional Protein +9

Blood-derived biomarkers correlate with clinical progression in Duchenne muscular dystrophy

Kristin Strandberg, Burcu Ayoglu, Andreas Roos et al. · Journal of Neuromuscular Diseases · 2020 · 38 citations · Full text

Exon-Skipping Oligonucleotides Restore Functional Collagen VI by Correcting a Common COL6A1 Mutation in Ullrich CMD

Sara Aguti, Véronique Bolduc, Pierpaolo Ala et al. · Molecular Therapy — Nucleic Acids · 2020 · 34 citations · Full text

<i>TRAPPC11</i> ‐related muscular dystrophy with hypoglycosylation of alpha‐dystroglycan in skeletal muscle and brain

Pinki Munot, Nadine McCrea, Silvia Torelli et al. · Neuropathology and Applied Neurobiology · 2021 · 27 citations · Full text