Francesca Ornaghi
Milan, Italy
Last Updated: 09/26/2026
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Found 8 publications
Cross-Correction in HSC Gene Therapy for Metachromatic Leukodystrophy.
Journal: The New England journal of medicine
Published: July 29, 2026
Therapeutic advantages of combined gene/cell therapy strategies in a murine model of GM2 gangliosidosis.
Journal: Molecular therapy. Methods & clinical development
Published: December 27, 2021
Novel bicistronic lentiviral vectors correct β-Hexosaminidase deficiency in neural and hematopoietic stem cells and progeny: implications for in vivo and ex vivo gene therapy of GM2 gangliosidosis.
Journal: Neurobiology of disease
Published: July 26, 2019
Corrigendum: Neuregulin 1 type III improves peripheral nerve myelination in a mouse model of congenital hypomyelinating neuropathy.
Journal: Human molecular genetics
Published: June 21, 2019
Corrigendum: Neuregulin 1 type III improves peripheral nerve myelination in a mouse model of congenital hypomyelinating neuropathy.
Journal: Human molecular genetics
Published: August 29, 2018
Neuregulin 1 type III improves peripheral nerve myelination in a mouse model of congenital hypomyelinating neuropathy.
Journal: Human molecular genetics
Published: August 29, 2018
A nonsense mutation in myelin protein zero causes congenital hypomyelination neuropathy through altered P0 membrane targeting and gain of abnormal function.
Journal: Human molecular genetics
Published: July 20, 2018
Generation of a LacZ reporter transgenic mouse line for the stereological analysis of oligodendrocyte loss in galactosylceramidase deficiency.
Journal: Journal of neuroscience research
Published: April 19, 2016
Showing 1-8 of 8
Last Updated: 09/26/2026