Zobrazeno 1 - 10
of 111
pro vyhledávání: '"Angela, Gritti"'
Autor:
Marco Luciani, Chiara Garsia, Stefano Beretta, Ingrid Cifola, Clelia Peano, Ivan Merelli, Luca Petiti, Annarita Miccio, Vasco Meneghini, Angela Gritti
Publikováno v:
Nature Communications, Vol 15, Iss 1, Pp 1-24 (2024)
Abstract Human induced pluripotent stem cell-derived neural stem/progenitor cells (hiPSC-NSCs) hold promise for treating neurodegenerative and demyelinating disorders. However, comprehensive studies on their identity and safety remain limited. In thi
Externí odkaz:
https://doaj.org/article/8daf1e448b9c4dbdbc60072df517206a
Autor:
Davide Sala, Francesca Ornaghi, Francesco Morena, Chiara Argentati, Manuela Valsecchi, Valeria Alberizzi, Roberta Di Guardo, Alessandra Bolino, Massimo Aureli, Sabata Martino, Angela Gritti
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 25, Iss , Pp 170-189 (2022)
Genetic deficiency of β-N-acetylhexosaminidase (Hex) functionality leads to accumulation of GM2 ganglioside in Tay-Sachs disease and Sandhoff disease (SD), which presently lack approved therapies. Current experimental gene therapy (GT) approaches wi
Externí odkaz:
https://doaj.org/article/61bdd9f6fc0d43f78a06d041a2b5f2f0
Publikováno v:
Frontiers in Medicine, Vol 8 (2022)
In vivo genetic engineering has recently shown remarkable potential as a novel effective treatment for an ever-growing number of diseases, as also witnessed by the recent marketing authorization of several in vivo gene therapy products. In vivo genet
Externí odkaz:
https://doaj.org/article/6b39da94f4434897acae9982911f8cab
Publikováno v:
Frontiers in Genome Editing, Vol 3 (2021)
Glial cells (astrocytes, oligodendrocytes, and microglia) are emerging as key players in several physiological and pathological processes of the central nervous system (CNS). Astrocytes and oligodendrocytes are not only supportive cells that release
Externí odkaz:
https://doaj.org/article/435d13ebf23345b1a1d5bd01a70c89d7
Publikováno v:
Frontiers in Molecular Biosciences, Vol 7 (2020)
Lysosomal storage diseases (LSDs) are a group of rare genetic conditions. The absence or deficiency of lysosomal proteins leads to excessive storage of undigested materials and drives secondary pathological mechanisms including autophagy, calcium hom
Externí odkaz:
https://doaj.org/article/e2dcf3f206c541cd8974108805b43091
Publikováno v:
Frontiers in Molecular Biosciences, Vol 7 (2020)
Globoid Cell Leukodystrophy (GLD) is a lysosomal storage disease (LSD) caused by inherited defects of the β-galactosylceramidase (GALC) gene. The infantile forms display a rapid and aggressive central and peripheral nervous system (CNS and PNS) dysf
Externí odkaz:
https://doaj.org/article/6cec4bd5071d42eca92974fa58d9cc24
Autor:
Francesca Ornaghi, Davide Sala, Fabiana Tedeschi, Maria Chiara Maffia, Martina Bazzucchi, Francesco Morena, Manuela Valsecchi, Massimo Aureli, Sabata Martino, Angela Gritti
Publikováno v:
Neurobiology of Disease, Vol 134, Iss , Pp - (2020)
The favorable outcome of in vivo and ex vivo gene therapy approaches in several Lysosomal Storage Diseases suggests that these treatment strategies might equally benefit GM2 gangliosidosis. Tay-Sachs and Sandhoff disease (the main forms of GM2 gangli
Externí odkaz:
https://doaj.org/article/e5ebadf6d2734a15bfc0f9c89dc37813
Autor:
Pietro Giuseppe Mazzara, Luca Massimino, Marta Pellegatta, Giulia Ronchi, Alessandra Ricca, Angelo Iannielli, Serena Gea Giannelli, Marco Cursi, Cinzia Cancellieri, Alessandro Sessa, Ubaldo Del Carro, Angelo Quattrini, Stefano Geuna, Angela Gritti, Carla Taveggia, Vania Broccoli
Publikováno v:
Nature Communications, Vol 8, Iss 1, Pp 1-13 (2017)
Schwann cells (SCs) myelinate peripheral nerve axons and offer opportunities for the treatment of injuries and demyelinating diseases but reliable and renewable sources of these cells are hard to come by. Here the authors reprogram rat, mouse and hum
Externí odkaz:
https://doaj.org/article/3e5822abc9d6496093cbde7fb6236024
Autor:
Vasco Meneghini, Annalisa Lattanzi, Luigi Tiradani, Gabriele Bravo, Francesco Morena, Francesca Sanvito, Andrea Calabria, John Bringas, Jeanne M Fisher‐Perkins, Jason P Dufour, Kate C Baker, Claudio Doglioni, Eugenio Montini, Bruce A Bunnell, Krystof Bankiewicz, Sabata Martino, Luigi Naldini, Angela Gritti
Publikováno v:
EMBO Molecular Medicine, Vol 8, Iss 5, Pp 489-510 (2016)
Abstract Metachromatic leukodystrophy (MLD) and globoid cell leukodystrophy (GLD or Krabbe disease) are severe neurodegenerative lysosomal storage diseases (LSD) caused by arylsulfatase A (ARSA) and galactosylceramidase (GALC) deficiency, respectivel
Externí odkaz:
https://doaj.org/article/df4a76df4d1e46e5afca6e405d504c4a
Publikováno v:
Methods in cell biology. 171
Human neural stem cells (hNSCs) hold great promises for the development of cell-based therapies for neurodegenerative diseases, given their capability to provide immunomodulatory and trophic support and to replace, to a limited extent, damaged, or lo
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=pmid________::a98beb9e305092b461f027abd808b6a4
https://pubmed.ncbi.nlm.nih.gov/35953197
https://pubmed.ncbi.nlm.nih.gov/35953197