P
Pasqualina Colella
Researcher at Université Paris-Saclay
Publications - 37
Citations - 4169
Pasqualina Colella is an academic researcher from Université Paris-Saclay. The author has contributed to research in topics: Transgene & Genetic enhancement. The author has an hindex of 15, co-authored 33 publications receiving 3277 citations. Previous affiliations of Pasqualina Colella include Stanford University & École pratique des hautes études.
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Journal ArticleDOI
TFEB links autophagy to lysosomal biogenesis.
Carmine Settembre,Chiara Di Malta,Vinicia Assunta Polito,Vinicia Assunta Polito,Moises Garcia Arencibia,Francesco Vetrini,Serkan Erdin,Serkan Erdin,Serpil Uckac Erdin,Serpil Uckac Erdin,Tuong Huynh,Tuong Huynh,Diego L. Medina,Pasqualina Colella,Marco Sardiello,Marco Sardiello,David C. Rubinsztein,Andrea Ballabio +17 more
TL;DR: A mitogen-activated protein kinase–dependent mechanism regulates autophagy by controlling the biogenesis and partnership of two distinct cellular organelles during starvation.
Journal ArticleDOI
Emerging Issues in AAV-Mediated In Vivo Gene Therapy
TL;DR: The liver will be used as a model target tissue for gene transfer based on the large amount of data available from preclinical and clinical studies, and key achievements and emerging issues in the field are presented.
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Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice
Mariacarmela Allocca,Monica Doria,Marco Petrillo,Pasqualina Colella,Maria Garcia-Hoyos,Daniel Gibbs,So Ra Kim,Albert M. Maguire,Tonia S. Rex,Umberto Di Vicino,Luisa Cutillo,Janet R. Sparrow,David S. Williams,Jean Bennett,Alberto Auricchio +14 more
TL;DR: It is demonstrated that vectors with an AAV5 capsid incorporated up to 8.9 kb of genome more efficiently than 6 other serotypes tested, independent of the efficiency of the rAAV2/5 production process.
Journal ArticleDOI
Effective delivery of large genes to the retina by dual AAV vectors
Ivana Trapani,Pasqualina Colella,Andrea Sommella,Carolina Iodice,Giulia Cesi,Sonia de Simone,Elena Marrocco,Settimio Rossi,Massimo Giunti,Arpad Palfi,Gwyneth Jane Farrar,Roman S. Polishchuk,Alberto Auricchio +12 more
TL;DR: It is found that dual trans‐splicing and hybrid vectors transduce efficiently mouse and pig photoreceptors to levels that, albeit lower than those achieved with a single AAV, resulted in significant improvement of the retinal phenotype of mouse models of STGD and USH1B.
Journal ArticleDOI
Efficient gene delivery to the cone-enriched pig retina by dual AAV vectors.
Pasqualina Colella,Ivana Trapani,Giulia Cesi,Andrea Sommella,Anna Manfredi,Agostina Puppo,Carolina Iodice,Settimio Rossi,Francesca Simonelli,Massimo Giunti,Maria Laura Bacci,Alberto Auricchio +11 more
TL;DR: The data support the potential of dual AAV vectors for large gene reconstitution in the cone-enriched pig retina that is a relevant preclinical model and shows that subretinal delivery in pigs ofDual AAV trans-splicing and hybrid vectors successfully reconstitute the expression of the large genes ABCA4 and MYO7A mutated in STGD and USH1B.