Nicolina Cristina Sorrentino is faculty researcher and head of translational Unit at TIGEM “Telethon Institute of Genetics and Medicine” of Naples.
She received the bachelor degree cum laude in Biological Sciences at “Federico II” University of Naples. In 2008, she started her PhD research at the Telethon Institute of Genetics and Medicine of Naples in the laboratory of Prof. Andrea Ballabio.  She worked on a research project based on “Developing a Systemic AAV-Medicated Gene Therapy Approach to Cross the Blood-Brain Barrier and Treat a CNS Pathology in Mucopolysaccharidosis Type IIIA”. This approach has been published in 2013  (Sorrentino NC et al. EMBO Mol Med. 2013) and is also under a patent (WO 2012085622 A1). In 2012 she received the Ph.D. degree in in Life and Bio-molecular Sciences from Open University of Oxford. In 2013, she joined the research group of Dr. Alessandro Fraldi at Tigem to start a postdoctoral position. During this period, she was responsible of a project in collaboration with” Shire Pharmaceutics” based on the Intrathecal AAV-mediated Gene Therapy approaches to treat the SNC pathology in large and small animal models of lysosomal storage disorders (Sorrentino NC et al. Molecular Therapy 2016; Sorrentino NC et al. Mol Ther Methods Clin Dev. 2019). In 2018, she started the new position as Head of translational unit and faculty researcher at Telethon Institute of Genetics and Medicine. The main aims of her research are the study and development of new gene and drug therapy approaches for the treatment of somatic and in particular CNS pathology in lysosomal storage disorders.