RNA - Genetherapys
Who we are - Spoke 1


University of Modena and Reggio Emilia (UNIMORE) scientists have pioneered stem cell and gene therapy research for epithelia, retina, blood, and neurodegeneration disorders. The UNIMORE Centre for Regenerative Medicine (CMR) is a 2000 sq metered GMP certified facility that has produced and registered the first stem cell based Advanced Therapy Medical Products (ATMPs) approved in Europe and is engaged in Ongoing Phase I/II clinical trials for ex-vivo gene therapy. Specific site expertise includes epithelial stem cell mediated cell therapy and tissue engineering, viral and non-viral methods to optimize the delivery of gene editing tools and gene therapy-based delivery of neuroprotective factors. Disease topic: Epidermolysis bullosa, Lamellar ichthyosis; Corneal dystrophies; Inherited retinal degeneration; Hereditary iron loading disorders; Sickle Cell Disease.

Antonello Pietrangelo
Full professor - (PI spoke)

Dipartimento di Scienze Mediche e Chirurgiche Materno-Infantili e dell’Adulto

Silvio Bicciato
Full professor

Dipartimento di Scienze della Vita

Fabio Biscarini
Full professor

Dipartimento di Scienze della Vita

Michele De Luca
Full professor

Dipartimento di Scienze della Vita

Francesca Fanelli
Full professor

Dipartimento di Scienze della Vita

Valeria Marigo
Full professor

Dipartimento di Scienze della Vita

Fulvio Mavilio
Full professor

Dipartimento di Scienze della Vita

Graziella Pellegrini
Full professor

Dipartimento di Scienze della Vita

Alessandra Recchia
Associate professor

Dipartimento di Scienze della Vita

Giovanni Tosi
Full professor

Dipartimento di Scienze della Vita

Daniela Benati
Researcher RTD-A

Dipartimento di Scienze della Vita

Eleonora Maurizi
Researcher RTD-A

Dipartimento di Scienze della Vita

Ilaria Ottonelli
Researcher RTD-A

Dipartimento di Scienze della Vita

Elisa Bergamini
PhD - XXXIX ciclo

Dipartimento di Scienze della Vita

Gaia Andrea Gozza
PhD - XXXIX ciclo

Dipartimento di Scienze della Vita

Luigi Palmieri
Full professor

Dipartimento di Bioscienze, Biotecnologie e Ambiente (Dbba)

Giuseppe Fiermonte
Full professor

Department of Biosciences, Biotechnologies and Environment – University of Bari

Annamaria De Luca
Full professor

Dipartimento di Farmacia – Scienze del Farmaco, Università degli Studi di Bari Aldo Moro

Luigi Viggiano
Researcher

Dipartimento di Bioscienze, Biotecnologie e Ambiente, Università degli Studi di Bari Aldo Moro


Chiesi Group is an international leader in the development of new therapies for patients suffering from rare diseases. Rare diseases represent one of the three pillars of Chiesi Group. In 2020, the Chiesi Group launched a Global Rare Diseases (GRD), which developed a portfolio consisting of ten drugs for patients with rare diseases. In November 2022, the company began the construction of a Biotech Center of Excellence that specializes in the development and production of therapeutic proteins, including enzymes produced with recombinant DNA technology. The company has participated in various research and development projects in the field of rare genetic diseases through innovative technologies such as gene and cell therapy and drug delivery systems in target organs and tissues. The goals and objectives of the Spoke 1 are fully in line with the strategic interests of the Chiesi Group, in particular those related to the field of rare hematological, rare neuro-ophthalmological and lysosomal storage disorders.


Telethon Institute of Genetics and Medicine (TIGEM) research focuses on understanding the disease mechanism and developing therapeutic strategies for inherited diseases. TIGEM investigators have gathered expertise in lysosomal biology, intracellular trafficking, and translational projects, especially gene therapy. The translational research is developed in the Molecular Therapy programme including investigators with expertise in clinical genetic, metabolic, and biochemical genetics. Research activity at TIGEM is supported by core facilities (Advanced microscopy and imaging, high content screening facility, Next generation sequencing facility). Disease topic: Birt-Hogg Dube’ syndrome and tuberous sclerosis; Mucopolysaccharidosis type VI; Leber optic neuropathy and Leigh syndrome; Pompe disease.

Lucia Perone
Technologist

Maria Teresa Pizzo
Technologist

Roman Polishchuk
Researcher - Group Leader

Barbara Rossi
Researcher

Francesco Giuseppe Salierno
Technologist

Roberta Tammaro
Technologist

Maria Antonietta Tarallo
Technologist

Lucia De Stefano
Post-doc Researcher

Nadia Minipoli
Post-doc Researcher

Rossella Pennella
Junior Technologist

Leandro Raul Soria
Post-doc Researcher


University of Bari Aldo Moro (UNIBA) scientists are experts in cell and mitochondria biology, mitochondrial protein deficiencies, gene delivery, CRISPR/CAS9 gene manipulation, and in vitro embryo production of farm animal models. The RU disposes of BSL II cell biology rooms and equipment as high-resolution respirometry instrumentation, facilities of mass spectrometry for metabolomics and fluorescence microscopy, a cell sorter cytofluorimeter. Disease topic: mitochondrial protein deficiencies.

Luigi Palmieri
Full Professor

Maria Elena Dell'Aquila
Full Professor

Maria Barile
Full Professor

Nunzio Denora
Full Professor

Giuseppe Fiermonte
Full Professor

Francesco Massimo Lasorsa
Associate Professor

Claudia Rita Catacchio
Associate Professor

Rosa Maria Iacobazzi
Researcher RTD-B

Nicola Antonio Martino
Researcher RTD-B

Piero Leone
Researcher RTD-A

Veronica Addabbo
PhD Student

Isabella Mongelli
PhD Student

Asaam Rajab Mohamed Eljahesh
PhD Student

Antonello Caponio
PhD Student


University of Bologna (UNIBO) scientists are recognized internationally for their expertise in clinical and molecular genetics, in vivo and in vitro disease models, development of novel treatment strategies, gene therapy and gene editing, and clinical trials. High-tech equipment is also accessible at its Center of Applied Biomedical Research, a core facility, including animal facilities, multi-omics technologies,  biochemistry, histology, and microscopy laboratories, and a hot-room for radio-labeled experiments, where multi-disciplinary expertise are shared.  Disease topic: mitochondrial disorders.

Laura Calza'
Full professor

Dipartimento di Farmacia e Biotecnologie – FABIT

Valerio Carelli
Full professor

Dipartimento di Scienze Biomediche e Neuromotorie – DIBINEM

Caterina Garone
Associate professor

Dipartimento di Scienze Mediche e Chirurgiche – DIMEC

Giuseppe Gasparre
Full professor

Dipartimento di Scienze Mediche e Chirurgiche – DIMEC

Luisa Iommarini
Associate professor

Dipartimento di Farmacia e Biotecnologie – FABIT

Ivana Kurelac
Associate professor

Dipartimento di Scienze Mediche e Chirurgiche – DIMEC

Gloria Ravegnini
Researcher RTD

Dipartimento di Farmacia e Biotecnologie – FABIT

Sara Carli
Researcher RTD-A

Dipartimento di Scienze Mediche e Chirurgiche – DIMEC

Giorgio Medici
Researcher RTD-A

Dipartimento di Scienze Biomediche e Neuromotorie – DIBINEM

Gaia Tioli
Research Fellow

Dipartimento di Farmacia e Biotecnologie – FABIT

Silvia Sabeni
Phd student

Dipartimento di Scienze Mediche e Chirurgiche – DIMEC

Elisa Dell’Anna
Phd student

Dipartimento di Farmacia e Biotecnologie – FABIT


University of Cagliari (UNICA) research members have an established expertise in Hemoglobinopathies, Thalassemia, Iron overload, Iron Chelators and new curative and non-curative therapeutic approaches in thalassemic syndromes. They are among the first in Europe, in terms of the number of patients, with hemoglobinopathy, a particularly alpha and beta thalassemia. Furthermore, the UNICA team also makes use of internationally recognized expertise in the genomics, epigenomics, and transcriptomics of complex pathologies, drug delivery, and more recent recently 2D/3D cellular models, and cellular differentiation. All this thanks to the use of cutting-edge instruments available at University Research Service Center. Disease topic: Beta thalassemia.

Annalisa Bonfiglio
Full professor

Dipartimento di Ingegneria elettrica ed elettronica

Giovanni Caocci
Associate professor

Dipartimento di Scienze mediche e sanità pubblica

Giorgio La Nasa
Full professor

Dipartimento di Scienze mediche e sanità pubblica

Maria Letizia Manca
Associate professor

Dipartimento di Scienze della Vita e dell’Ambiente

Maria Manconi
Full professor

Dipartimento di Scienze della Vita e dell’Ambiente

Olga Mulas
Researcher

Dipartimento di Scienze mediche e sanità pubblica

Raffaella Origa
Researcher

Dipartimento di Scienze mediche e sanità pubblica

Patrizia Zavattari
Full professor

Dipartimento di Scienze biomediche

Eleonora Loi
Researcher

Dipartimento di Scienze biomediche

Matteo Perra
Researcher

Dipartimento di Scienze della Vita e dell’Ambiente

Angela Maria Mereu
Phd student

Dipartimento di Scienze mediche e sanità pubblica

Francesca Floris
Phd student

Dipartimento di Scienze biomediche


University of Milan (UNIMI) researchers specialize in unraveling the intricacies of mammalian skeletal muscle development and exploring the biological processes inherent in both normal and pathogenic muscle conditions. Their dedication extends to devising therapeutic approaches for Duchenne Muscular Dystrophy, with a focus on pioneering solutions. Additionally, their proficiency extends to a comprehensive examination of cellular and molecular processes associated with neuromuscular disorders, notably including a dedicated emphasis on Friedreich’s ataxia.

Federica Briani
Associate professor

Dipartimento di Bioscienze

Giacomo Pietro Comi
Full professor

Dipartimento di Fisiopatologia Medico-Chirurgica e dei Trapianti

Luca Del Giacco
Associate professor

Dipartimento di Bioscienze

Diletta Dolfini
Associate professor

Dipartimento di Bioscienze

Nerina Bruna Gnesutta
Associate professor

Dipartimento di Bioscienze

Graziella Messina
Full professor

Dipartimento di Bioscienze

Yvan Torrente
Associate professor

Dipartimento di Fisiopatologia Medico-Chirurgica e dei Trapianti

Chiara Villa
Researcher RTD-A

Dipartimento di Fisiopatologia Medico-Chirurgica e dei Trapianti

Alice Diroma
Phd student

Dipartimento di Fisiopatologia Medico-Chirurgica e dei Trapianti

Rebecca Oddone
Phd student

Dipartimento di Scienze Farmacologiche e Biomolecolari

Giorgia Careccia
Researcher RTD-A

Dipartimento di Bioscienze

Giuseppe Angelini
Research Fellow

Dipartimento di Bioscienze

Chiara Bonfanti
Technologist

Dipartimento di Bioscienze


University of Naples Federico II (UNINA) research members are leaders in clinical genetics, molecular genetics, experimental hematology, Next-Generation Sequencing (NGS), functional genomics, bioinformatics, genome editing, pre-clinical drug studies, biomarker discovery, and the development of genetic mice models to focus on hereditary red blood cell and iron metabolism defects. This multidisciplinary team also possesses significant proficiency in cancer genetics and the increasing field of mRNA therapies. The combination of expertise in both basic and clinical research areas allows UNINA to address complex scientific questions and develop innovative solutions. Disease topic: Hereditary defects of red blood cells / iron metabolism defects and neurodevelopmental disorders.

Stefano Amente
Associate professor

Dipartimento di Medicina Molecolare e Biotecnologie Mediche

Immacolata Andolfo
Researcher RTD-B

Dipartimento di Medicina Molecolare e Biotecnologie Mediche

Mario Capasso
Associate professor

Dipartimento di Medicina Molecolare e Biotecnologie Mediche

Antonio Cittadini
Full professor

Dipartimento di Scienze Mediche Traslazionali

Veronica Ferrucci
Researcher RTD-A

Dipartimento di Medicina Molecolare e Biotecnologie Mediche

Achille Iolascon
Full professor

Dipartimento di Medicina Molecolare e Biotecnologie Mediche

Alberto M. Marra
Associate professor

Dipartimento di Scienze Mediche Traslazionali

Roberta Russo
Researcher RTD-B

Dipartimento di Medicina Molecolare e Biotecnologie Mediche

Massimo Zollo
Full professor

Dipartimento di Medicina Molecolare e Biotecnologie Mediche

Mariarosaria De Luca
Research Fellow

Dipartimento di Scienze Mediche Traslazionali

Myrhiam Cassese
Phd student

Dipartimento di Medicina Molecolare e Biotecnologie Mediche

Filippo Russo
Phd student

Dipartimento di Medicina Molecolare e Biotecnologie Mediche


University of Padua (UNIPD) scientists have pioneered the fields of hematopoietic stem cell and gene therapy for inherited neurometabolic diseases and for thrombotic and bleeding disorders. UNIPD is a world leader in mitochondrial biology, pathophysiology, diseases and gene therapy, with more than 40 laboratories and more than 200 researchers, of which include four EMBO and three Academia Europaea members, working on the topic. UNIPD researchers enjoy access to dedicated infrastructures including the novel Metabolic and Imaging Infrastructure “Miniature” that integrates advanced imaging, automated screening facilities, metabolomic and metabolic imaging in a unique platform, and a state of the art CryoEM. Disease topic: NCL1, leukodystrophies; Hemophilia; Mitochondrial disorders.

Paolo Simioni
Full professor

Dipartimento di Medicina

Elena Campello
Researcher RTD-B

Dipartimento di Medicina

Giorgia Nuozzi
Research Fellow

Dipartimento di Medicina

Yahima Frion Herrera
Research Fellow

Dipartimento di Medicina

Paolo Bernardi
Full professor

Dipartimento di Scienze Biomediche

Alessandra Biffi
Full professor

Dipartimento della Salute della Donna e del Bambino

Vanessa Checchetto
Researcher RTD-A

Dipartimento di Biologia

Marta Giacomello
Associate professor

Dipartimento di Biologia

Luca Scorrano
Full professor

Dipartimento di Biologia

Eva Trevisson
Associate professor

Dipartimento della Salute della Donna e del Bambino

Carlo Fiore Viscomi
Associate professor

Dipartimento di Scienze Biomediche

Michela Carraro
Researcher RTD-A

Dipartimento di Scienze Biomediche

Martina Semenzato
Researcher RTD-A

Dipartimento di Biologia

Valeria Roxana Balmaceda Valdez
Research Fellow

Dipartimento di Scienze Biomediche

Andre Djalalvandi
Research Fellow

Dipartimento di Biologia

Jules Mérian
Research Fellow

Dipartimento di Biologia

Linda Rossini
PhD student

Dipartimento della Salute della Donna e del Bambino

Giulia Toffanin
PhD student

Dipartimento della Salute della Donna e del Bambino


University of Siena (UNISI) team members have extensive expertise in Molecular Genetics, cellular and molecular biology of the Central Nervous System (CNS), disease modeling, gene editing. Genetics, cellular and molecular biology of skeletal muscle tissue, cellular and animal models of diseases, confocal microscopy. UNISI teams are supported by UpToDate platforms for molecular genetics, cell biology and gene expression, bioinformatics analysis and a center for Electron and Confocal Microscopy. Disease topic: Rett syndrome, Alport syndrome, Skeletal Muscle/Central Core Disease.

Vincenzo Sorrentino
Full professor

Dipartimento di Medicina Molecolare e dello Sviluppo

Alessandra Gamberucci
Associate professor

Dipartimento di Medicina Molecolare e dello Sviluppo

Enrico Pierantozzi
Associate professor

Dipartimento di Medicina Molecolare e dello Sviluppo

Paola Marcolongo
Researcher

Dipartimento di Medicina Molecolare e dello Sviluppo

Cinzia Signorini
Associate professor

Dipartimento di Medicina Molecolare e dello Sviluppo

Giulia Collodel
Associate professor

Dipartimento di Medicina Molecolare e dello Sviluppo

Elena Moretti
Associate professor

Dipartimento di Medicina Molecolare e dello Sviluppo

Alessandra Renieri
Full professor

Dipartimento di Biotecnologie Mediche

Ilaria Meloni
Associate professor

Dipartimento di Biotecnologie Mediche

Francesca Ariani
Associate professor

Dipartimento di Biotecnologie Mediche

Sergio Daga
Researcher RTD-A

Dipartimento di Biotecnologie Mediche

Matteo Serano
Researcher RTD-A

Dipartimento di Medicina Molecolare e dello Sviluppo

Margherita Baldassarri
Researcher RTD-A

Dipartimento di Biotecnologie Mediche

Egidio Maria Rubino
Research Fellow

Dipartimento di Medicina Molecolare e dello Sviluppo