Pasqualina Colella

Evry, France

Last Updated: 09/26/2026

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Found 22 publications

Autologous genome-edited hematopoietic stem cells correct Gaucher disease and establish a platform for clinical translation.

Autologous genome-edited hematopoietic stem cells correct Gaucher disease and establish a platform for clinical translation.

Journal: Research square

Published: September 02, 2025

Advances in Pompe Disease Treatment: From Enzyme Replacement to Gene Therapy.

Advances in Pompe Disease Treatment: From Enzyme Replacement to Gene Therapy.

Journal: Molecular diagnosis & therapy

Published: July 23, 2024

CNS Repopulation by Hematopoietic-Derived Microglia-Like Cells Corrects Progranulin deficiency.

CNS Repopulation by Hematopoietic-Derived Microglia-Like Cells Corrects Progranulin deficiency.

Journal: Research square

Published: October 04, 2023

CNS-wide repopulation by hematopoietic-derived microglia-like cells corrects progranulin deficiency in mice.

CNS-wide repopulation by hematopoietic-derived microglia-like cells corrects progranulin deficiency in mice.

Journal: Nature communications

Published: August 21, 2023

Editorial: Ex-vivo and in-vivo genome engineering for metabolic and neurometabolic diseases.

Editorial: Ex-vivo and in-vivo genome engineering for metabolic and neurometabolic diseases.

Journal: Frontiers in genome editing

Published: June 27, 2023

Successful treatment of severe MSUD in Bckdhb-/- mice with neonatal AAV gene therapy.

Successful treatment of severe MSUD in Bckdhb-/- mice with neonatal AAV gene therapy.

Journal: Journal of inherited metabolic disease

Published: November 30, 2022

Improved engraftment and therapeutic efficacy by human genome-edited hematopoietic stem cells with Busulfan-based myeloablation.

Improved engraftment and therapeutic efficacy by human genome-edited hematopoietic stem cells with Busulfan-based myeloablation.

Journal: Molecular therapy. Methods & clinical development

Published: October 29, 2021

Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates.

Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates.

Journal: Nature communications

Published: February 16, 2021

Neonatal gene therapy achieves sustained disease rescue of maple syrup urine disease in mice.

Neonatal gene therapy achieves sustained disease rescue of maple syrup urine disease in mice.

Journal: Nature communications

Published: August 25, 2020

Author Correction: Engineering monocyte/macrophage-specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing.

Author Correction: Engineering monocyte/macrophage-specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing.

Journal: Nature communications

Published: August 21, 2020

Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects.

Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects.

Journal: EBioMedicine

Published: June 15, 2020

Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase.

Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase.

Journal: Molecular therapy : the journal of the American Society of Gene Therapy

Published: November 13, 2019

Showing 1-12 of 22

Last Updated: 09/26/2026