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Colella, Pasqualina
106
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Online (106)
Mediatypes
Articles (Online) (37)
OpenAccess-fulltext (69)
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1
Genome-edited hematopoietic stem cells as a curative approa..:
Pimentel Vera, Luisa N.
;
Gastou, Marc
;
Arozqueta-Basurto, Jessica
...
Molecular Genetics and Metabolism. 141 (2024) 2 - p. 107998 , 2024
Link:
https://doi.org/10.1016/..
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2
CNS-wide repopulation by hematopoietic-derived microglia-li..:
Colella, Pasqualina
;
Sayana, Ruhi
;
Suarez-Nieto, Maria Valentina
...
Nature Communications. 15 (2024) 1 - p. , 2024
Link:
https://doi.org/10.1038/..
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3
Advances in Pompe Disease Treatment: From Enzyme Replacemen..:
Colella, Pasqualina
Molecular Diagnosis & Therapy. , 2024
Link:
https://doi.org/10.1007/..
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4
Editorial: Ex-vivo and in-vivo genome engineering for metab..:
Colella, Pasqualina
;
Meneghini, Vasco
;
Baldo, Guilherme
.
Frontiers in Genome Editing. 5 (2023) - p. , 2023
Link:
https://doi.org/10.3389/..
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5
Successful treatment of severe MSUD in Bckdhb−/− mice with ..:
Pontoizeau, Clément
;
Gaborit, Clovis
;
Tual, Nolan
...
Journal of Inherited Metabolic Disease. 47 (2023) 1 - p. 41-49 , 2023
Link:
https://doi.org/10.1002/..
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6
Neonatal gene therapy achieves sustained disease rescue of ..:
Pontoizeau, Clément
;
Simon-Sola, Marcelo
;
Gaborit, Clovis
...
Nature Communications. 13 (2022) 1 - p. , 2022
Link:
https://doi.org/10.1038/..
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7
Improved engraftment and therapeutic efficacy by human geno..:
Poletto, Edina
;
Colella, Pasqualina
;
Pimentel Vera, Luisa N.
...
Molecular Therapy - Methods & Clinical Development. 25 (2022) - p. 392-409 , 2022
Link:
https://doi.org/10.1016/..
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8
Hepatic expression of GAA results in enhanced enzyme bioava..:
Costa-Verdera, Helena
;
Collaud, Fanny
;
Riling, Christopher R.
...
Nature Communications. 12 (2021) 1 - p. , 2021
Link:
https://doi.org/10.1038/..
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9
Busulfan conditioning allows high engraftment of human geno..:
Poletto, Edina
;
Colella, Pasqualina
;
Sheikali, Adam
..
Molecular Genetics and Metabolism. 132 (2021) 2 - p. S87 , 2021
Link:
https://doi.org/10.1016/..
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10
Autologous transplantation of human genome-edited hematopoi..:
Gomez-Ospina, Natalia
;
Colella, Pasqualina
;
Khan, Shaukat
..
Molecular Genetics and Metabolism. 132 (2021) - p. S62 , 2021
Link:
https://doi.org/10.1016/..
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11
Rescue of Advanced Pompe Disease in Mice with Hepatic Expre..:
Cagin, Umut
;
Puzzo, Francesco
;
Gomez, Manuel Jose
...
Molecular Therapy. 28 (2020) 9 - p. 2056-2072 , 2020
Link:
https://doi.org/10.1016/..
?
12
Engineering monocyte/macrophage−specific glucocerebrosidase..:
Scharenberg, Samantha G.
;
Poletto, Edina
;
Lucot, Katherine L.
...
Nature Communications. 11 (2020) 1 - p. , 2020
Link:
https://doi.org/10.1038/..
?
13
Gene therapy with secreted acid alpha-glucosidase rescues P..:
Colella, Pasqualina
;
Sellier, Pauline
;
Gomez, Manuel J.
...
EBioMedicine. 61 (2020) - p. 103052 , 2020
Link:
https://doi.org/10.1016/..
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14
Author Correction: Engineering monocyte/macrophage−specific..:
Scharenberg, Samantha G.
;
Poletto, Edina
;
Lucot, Katherine L.
...
Nature Communications. 11 (2020) 1 - p. , 2020
Link:
https://doi.org/10.1038/..
?
15
Preclinical development of , an investigational liver-direc..:
Armour, Sean M.
;
Nordin, Jayme M.L.
;
Verdera, Helena Costa
...
Molecular Genetics and Metabolism. 129 (2020) 2 - p. S22-S23 , 2020
Link:
https://doi.org/10.1016/..
1-15