Pasqualina Colella
7PUBLICATIONS
29CO-AUTHORS
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Publications (7)
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|Aug 12, 2024
Advances in Pompe Disease Treatment: From Enzyme Replacement to Gene Therapy.Pasqualina Colella
|Jul 05, 2024
CNS-wide repopulation by hematopoietic-derived microglia-like cells corrects progranulin deficiency in mice.Pasqualina Colella, Ruhi Sayana, Maria Valentina Suarez-Nieto
|Jun 07, 2022
Neonatal gene therapy achieves sustained disease rescue of maple syrup urine disease in mice.Clément Pontoizeau, Marcelo Simon-Sola, Clovis Gaborit
|Nov 05, 2021
Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates.Helena Costa-Verdera, Fanny Collaud, Christopher R Riling
|Aug 22, 2020
Author Correction: Engineering monocyte/macrophage-specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing.Samantha G Scharenberg, Edina Poletto, Katherine L Lucot
|Jul 05, 2020
Engineering monocyte/macrophage-specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing.Samantha G Scharenberg, Edina Poletto, Katherine L Lucot
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Frequent Collaborators
3 joint publications
Natalia Gomez-Ospina
2 joint publications
Matthew H Porteus
2 joint publications
Katherine L Lucot
2 joint publications
Giuseppe Ronzitti
2 joint publications
Edina Poletto
2 joint publications
Samantha G Scharenberg
1 joint publications
Maria G Biferi
1 joint publications
Deeksha Bali
1 joint publications
Romain Hardet
1 joint publications
Amine Meliani
