Fabio Dell'Aquila Email & Phone Number

Pamplona, Nc, Es

Cracovia, Polonia

Napoli, Campania, Italia

• Member of UshTher preclinical studies and director of in vivo potency assay of GMP-like and GMP lots of dual adeno-associated viral (AAV) vectors (in collaboration with AAVantgarde Bio, https://www.aavantgarde.com/it/) •Drafted and managed the budget for the in vivo potency assay• Compared split-inteins delivered by AAV vectors in vitro and in vivo (in collaboration with AAVantgarde Bio) •Drafted and managed the budget for the comparison of inteins• Designed and tested homology-independent targeted integration (HITI) strategies at murine and human loci• Performed a dose-response of HITI delivered by AAV vectors to treat mouse models of systemic disorders• Administered neonatal and adult mouse models by temporal vein injection or retrorbital injection• Assessed phenotypic improvements in serum, urines, heart, kidney, liver, and spleen of treated animals• Tested improved versions of CRISPR/Cas9

Pozzuoli

•Designed and tested in vitro and in vivo a gene replacement therapy using adeno-associated viral 8 vectors encoding OAT (AAV8.OAT) for gyrate atrophy of the choroid and retina (GACR)•Wrote an animal project to work on GACR mouse model•Rescued pups mouse model of GACR from fatal hyperammonemia by intraperitoneal administration of arginine•Improved retinal function and morphology in a mouse model of GACR using AAV8.OAT•Member of preclinical studies for gene therapy of USH1B using dual AAV8 vectors encoding for MYO7A (dual AAV8.MYO7A) (UshTher, https://www.ushther.eu)•Optimized and characterized the plasmids for the good manufacturing practices (GMP) of dual AAV8.MYO7A• Wrote an animal project to work on shaker1 mouse model of USH1B•Designed a custom formulation buffer for the GMP lot of dual AAV8.MYO7A and ensured its safety in the subretinal space of mice•Performed a good-laboratory practice-like in vivo dose-response experiment using a GMP-like lot of dual AAV8.MYO7A administered intraocularly in shaker1•Contributed to assess safety, biodistribution, and expression of dual AAV8.MYO7A administered subretina in non-human primates•Filed standard operative procedures (SOPs) and presented final results to the project's key stakeholders

Pozzuoli

• Training and specialization in retinal electrophysiology of small and large rodents to study retinal diseases • Ensured safety of intein-mediated protein trans-splicing in the subretinal space of adult mice

Pozzuoli, Napoli, Italia

•Selected small molecules from Selleck library that specifically enhance dual AAV vectors transduction efficiency in vitro and in vivo•Dissected pathways involved in AAV transduction cascade by siRNA-mediated knock-down in vitro.

Executive Master In Project Management, Project Management

Activities and Societies: Courses on stakeholder engagement, budgeting, financing, entrepreneurship, waterfall and agile methodologies.

Ph.D. In Systems Medicine, Human Genetics, Proclaimed "Excellent Ph.D." By The University Of Naples "Federico Ii" Committee

Courses in human evolution, gene & cell therapy, cellular & molecular biology, microscope techniques, bioinformatics, biostatistics

Iii Spring School

Lectures and seminars on gene and cell therapy strategies in clinics

M.Sc. In Medical Biotechnologies, Biotechnologies, 110/110 L (Gpa 4.0)

Exams on: ▪ Biochemistry of complex systems and Bioinformatics ▪ Developmental Biology and Gene expression ▪ Genetics and Molecular.

Bachelor Of Health Biotechnology, Biotecnologie Mediche, 107/110

Accomplished exams: - Technologies for Gene and Cell Therapy - Cell, Molecular and Computational Biotechnologies - Biotechnologies.

Education record