Application of gene targeting by Small Fragment Homologous Replacement (SFHR) to human Cystic Fibrosis stem-like and differentiated cellular systems
cun id:
2317
TERAPIA GENICA
High throughput skills in neuronal RNA biology: role of non coding RNAs in motor neuron activity and their therapeutic exploitation in neurodegenerative diseases
Control of nuclear fragility
Study of tissue environmental cues inactivating the master regulator of hepatocyte differentiation, Hepatocyte Nuclear Factor 4, in liver diseases
Use of transposons PiggyBac to transduce T and CIK cells with FcgR-Chimeric Receptors (CAR-T and CAR-CIK) for adoptive cell therapy of AML
Molecular therapy of neonatal diabetes. Analysis of the rescue capability of lentivectors encoding a shRNA against INS1L39Y40delinsH mutation associated with Permanent Neonatal Diabetes
Application of a gene targeting strategy to a mouse reporter system and to stem-induced human primary Cystic Fibrosis epithelial cells.
Fanconi Anemia and Chromosome Instability
The personalized therapy of Cystic Fibrosis by theratyping and gene targeting
Circulating small nucleolar RNA in Huntington disease: reporter of a tipping point in disease development and potential biomarker to follow therapeutic approaches in early stages of disease