Dr Alberto Malerba
Department: Comparative Biomedical Sciences
Campus: Camden
Alberto is a Senior Lecturer working in the field of gene therapy for neuromuscular conditions. He develops novel strategies to treat rare genetic diseases affecting children and adults, using cellular and animal models of disease.
Alberto was awarded a PhD in Biotechnologies at the Padua University, working on a project studying the role of inflammation in muscle regeneration. Afterwards, he joined the gene therapy laboratory at Royal Holloway, where he contributed to the development of novel gene therapy agents and antisense therapeutics for the treatment of muscular dystrophies and other muscle conditions. He initially focused on the development of antisense reagents for exon skipping as a treatment for Duchenne muscular dystrophy. Afterwards, he was appointed as research fellow at the Royal Veterinary College in London, where he developed a scientific program based on new splicing-modulating molecules for the treatment of ischemic diseases in muscle. He later re-joined the gene therapy laboratory at Royal Holloway as project manager to work on the optimization of gene therapies and antisense therapeutics for a number of muscle diseases. He collaborated on the development of a gene therapy AAV vector for Oculopharyngeal Muscular Dystrophy (OPMD), a rare muscle disease affecting adults. This vector is currently in clinical trial in US. He also worked within the Unite DMD consortium to complete the preclinical development of an AAV-microdystrophin gene therapy that is now in clinical trial in France and UK. He became a Lecturer in Gene Therapy in 2022 and joined the RVC in October 2025.
Alberto’s laboratory is focused on finding new treatments for muscle pathologies. He uses gene therapy technologies to target molecular defects in pre-clinical models of rare neuromuscular diseases such as Duchenne muscular dystrophy (DMD), Oculopharyngeal muscular dystrophy (OPMD) and Facioscapulohumeral muscular dystrophy (FSHD). These include:
- Antisense oligonucleotides (AO) to induce exon skipping and siRNA therapeutics to silence gene expression;
- Gene replacement approaches based on viral (Adeno-associated virus and Lentivirus) and non-viral vectors;
- Gene editing applications to correct genetic defects or to modify transcriptional processes;
- Pharmacological agents that can improve the dystrophic pathology and that can have additive or synergistic effects when combined with gene therapy applications.
Alberto has solid collaborations with national and international academics and with Industry partners to develop gene therapy reagents, antisense therapeutics and pharmacological agents that have potential for clinical translation.
Alberto is a Fellow of the Higher Education Academy. He teaches in the “Integrated Physiology 1”, “The moving animal” and “Problem Definition and Investigation” modules.
