Dr Alberto Malerba

Senior Lecturer

Department: Comparative Biomedical Sciences

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.

Publications 2016-2025:

Trundle J, Boulinguiez A, Lu Nguyen N, March J, Malerba A, Popplewell L. Periostin exon 17 skipping enhances the efficacy of local AAV-microdystrophin administration in a fibrotic model of Duchenne muscular dystrophy. Human gene therapy. 2025 Sep;36(17-18):1257-1267

Kordikowski Boix J, Bos E, Shademan M, Mallon S, van Zanen-Gerhardt S, Lu-Nguyen N, Malerba A, Coenen de Roo CJJ, Raz V. Histopathology of the tongue in a mouse model of oculopharyngeal muscular dystrophy reveals progressive PABPN1 deposition associated with myofiber structural defects. American Journal of Pathology. 2025 Apr;195(4):741-753.

Boulinguiez A, Dhiab J, Crisol B, Muraine L, Gaut L, Rouxel C, Flaire J, Mouigni HR, Lemaitre M, Giroux B, SaintPierre B, Ferry A, Mouly V, Butler-Browne G, Negroni E, Malerba A*,Trollet C*. Different outcomes of endurance and resistance exercise in skeletal muscles of Oculopharyngeal muscular dystrophy. Journal of sarcopenia, cachexia and muscle. 2024 Oct;15(5):1976-1988. 

Lu-Nguyen N, Snowden S, Popplewell L, Malerba A*. Systemic pharmacotherapeutic treatment of the ACTA1-MCM/FLExDUX4 preclinical mouse model of FSHD. International Journal of Molecular Science, 2024 Jun 26;25(13):6994. 

Trundle J, Cernisova V, Boulinguiez A, Lu Nguyen N, Malerba A*, Popplewell L. Expression of the pro-fibrotic marker Periostin in a mouse model of Duchenne muscular dystrophy. Biomedicines. 2024 Jan 18;12(1):216. 

Selvakumaran J, Ursu S, Bowerman M, Lu-Nguyen N, Wood MJ, Malerba A and Yáñez-Muñoz RJ. An induced pluripotent stem cell-derived human blood-brain barrier (BBB) model to test gene therapies for neuromuscular diseases. Biomedicines. 2023 Oct 4;11(10):2700.

Harish P, Malerba A, Kroon R, Shademan M, van Engelan B, Raz V, Popplewell L, Snowden S.G. Novel metabolomic approach for identifying pathology specific biomarkers in rare diseases: a case study in Oculopharyngeal muscular dystrophy. Metabolites, 2023 Jun 19;13(6):769.

Cernisova V, Lu Nguyen N, Herath S, Malerba A*, Popplewell L. Microdystrophin gene addition significantly improves muscle functionality and diaphragm muscle histopathology in a fibrotic mouse model of Duchenne muscular dystrophy. International Journal of Molecular Science, 2023, May 3;24(9):8174. 

Lu-Nguyen N, Dickson G, Malerba A*, Popplewell L. Long-term systemic treatment of a mouse model displaying chronic FSHD-like pathology with antisense therapeutics that inhibit DUX4 expression. Biomedicines. 2022, Jul 7;10(7):1623.

Bourdon A, François V, Zhang L, Lafoux A, Fraysse B, Toumaniantz G, Larcher T, Girard T, Ledevin M, Lebreton C, Hivonnait A, Creismeas A, Allais M, Marie B, Guguin J, Blouin V,  Huchet C, Malerba A, Kao B, Le Heron A, Moullier P, Dickson G, Popplewell L, Adjali O,  Montanaro F, Le Guiner C. Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model. Gene therapy. 2022 Sep;29(9):520-535.

Lu-Nguyen N, Malerba A, Antoni Pineda M, Dickson G, Popplewell L. Improving molecular and histopathology in diaphragm muscle of the double transgenic ACTA1-MCM/FLExDUX4 mouse model of FSHD with systemic antisense therapy. Human gene therapy. 2022 Sep;33(17-18):923-935

Lu-Nguyen N, Malerba A, Herath S, Dickson G, Popplewell L. Systemic antisense therapeutics inhibiting DUX4 expression ameliorates FSHD-like pathology in an FSHD mouse model. Human Molecular Genetics, 2021, Jul 9;30(15):1398-1412.

Kao B*, Malerba A*, Lu-Nguyen N, Harish P, McCarthy J, Popplewell L, Dickson G. RPL3L protein expression has a significant impact on skeletal muscle strength of a dystrophic mouse model of Duchenne muscular dystrophy. Nucleic Acid Therapeutics. 2021 Dec;31(6):457-464 

Malerba A*, Sidoli C, Lu-Nguyen, N. Herath S, Le Heron A, Abdul-Razak H, Jarmin S, VandenDriessche T, Chuah MK, Dickson G, Popplewell L. Dose-dependent microdystrophin expression enhancement in cardiac muscle by a cardiac specific regulatory element Human Gene Therapy, 2021, Oct;32(19-20):1138-1146. 

Strings V*, Malerba A*, Harbaran S, Roth F, Chaytow H, Cordova G, Lu-Nguyen N, Cappellari O, Roelvink P, Trollet C, Dickson G, Suhy D. Single AAV vector system for gene therapy treatment of oculopharyngeal muscular dystrophy (OPMD). Molecular Therapy Nucleic Acids, 2021, Feb 18;24:67-78. 

Echevarría L, Malerba A, Arechavala-Gomeza V. Researcher's Perceptions on Publishing "Negative" Results and Open Access. Nucleic Acid Therapeutics. 2021 Jun;31(3):185-189.

Harish P, Dickson G, Malerba A*, Popplewell L* Myostatin inhibition improves the pathology in aged murine model of oculopharyngeal muscular dystrophy (OPMD). Front Physiol. 2020 Mar 5;11:184. 

Zhou H, Meng J, Malerba A, Catapano F, Sintusek P, Jarmin S, Feng L, Lu-Nguyen N, Sun L, Mariot V, Dumonceaux J, Morgan JE, Gissen P, Dickson G, Muntoni F. Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy. Journal of Sarcopenia, Cachexia and Muscle, 2020. Jun;11(3):768-782.  

Malerba A*, Klein P, Lu-Nguyen N, Cappellari O, Strings-Ufombah V, Harbaran S, Roelvink P, Suhy D, Trollet C, Dickson G. Established PABPN1 intranuclear inclusions in OPMD muscle can be efficiently reversed by AAV-mediated knock-down and replacement of mutant expanded PABPN1. Human Molecular Genetics, 2019 Oct 1;28(19):3301-3308. 

Lu-Nguyen N, Ferry A, Schnell FJ, Hanson GJ, Popplewell P, Dickson G, Malerba A*. Functional muscle recovery following dystrophin and myostatin exon splice modulation in aged mdx mice. Human Molecular Genetics, 2019 Sep 15;28(18):3091-3100. 

Harish P*, Malerba A*, Lu-Nguyen N, Forrest L, Cappellari O, Roth F, Dihab J, Trollet C, Popplewell L, Dickson G. Myostatin inhibition increases muscle mass and improves the pathology in a murine model of oculopharyngeal muscular dystrophy (OPMD). Journal of Sarcopenia, Cachexia and Muscle, 2019 May 7. doi: 10.1002/jcsm.12438 

Malerba A, Roth F, Harish P, Dhiab J, Lu-Nguyen N, Cappellari O, Jarmin S, Mahoudeau A, Ythier V,  Lainé J, Negroni E, Abgueguen E, Simonelig M, Guedat P, Mouly V, Butler-Browne G, Voisset C, Dickson G, Trollet C. Pharmacological modulation of ER stress response in oculopharyngeal muscular dystrophy. Human Molecular Genetics, 2019 May 15;28(10):1694-1708.

Koo TY, Lu-Nguyen N, Malerba A, Kim E, Kim D, Cappellari O, Cho HY, Dickson G, Popplewell L, Kim JS. Functional rescue of dystrophin deficiency in mice caused by frameshift mutations using Campylobacter jejuni Cas9. Molecular Therapy, 2018 Jun 6;26(6):1529-1538.

Le Guiner C, Servais L, Montus M, Larcher T, Fraysse B, Moullec S, Allais M, François V, Dutilleul M, Malerba A, Koo T, Thibaut JL, Matot B, Devaux M, Le Duff J, Deschamps JY, Barthelemy I, Blot S, Testault I, Wahbi K, Ederhy S, Martin S, Veron P, Georger C, Athanasopoulos T, Masurier C, Mingozzi F, Carlier P, Gjata B, Hogrel JY, Adjali O, Mavilio F, Voit T, Moullier P, Dickson G. Long term microdystrophin gene therapy improves phenotype in a canine model of Duchenne muscular dystrophy. Nature communications. 2017 Jul 25;8:16105.

Malerba A, Klein P, Bachtarzi H, Jarmin SA, Cordova G, Ferry A, Strings V, Espinoza MP,  Mamchaoui K, Blumen SC, Lacau St Guily J, Mouly V, Graham M, Butler-Browne G, Suhy D, Trollet C, Dickson G. PABPN1 gene therapy for Oculopharyngeal muscular dystrophy. Nature Communications. 2017 Mar 31;8:14848

Lu-Nguyen N*, Malerba A*, Popplewell L, Schnell F, Dickson G. Dual systemic antisense therapy ameliorates pathology in an adult animal model of DMD. Molecular Therapy – Nucleic acids, 2017 Mar 17;6:15-28 

Klein P, Oloko M, Montel V, Malerba A, Jarmin SA, Gidaro T, Perie S,Lacau St Guily J, De la Grange P, Antoniou M, Dickson G, Butler-Browne G, Bastide B, Mouly V, Trollet C. Splicing misregulation in OPMD as a result of mRNA trapping in PABPN1 inclusions. Nucleic Acid Research, 2016 Dec 15; 44(22): 10929-10945.

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.

Top of page