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Colella, Pasqualina
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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/..
 
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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/..
 
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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/..
 
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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/..
 
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