Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Metachromatic Leukodystrophy (MLD): Clinical Outcomes from 38 Patients

2021 ◽  
Author(s):  
Mirko Essing ◽  
Alberto Zambon ◽  
Vera Gallo ◽  
Cristina Baldoli ◽  
Federica Cugnata ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Clinical Outcomes ◽  
Progenitor Cell ◽  
Metachromatic Leukodystrophy ◽  
Hematopoietic Stem ◽  
Cell Gene

Lentiviral hematopoietic stem and progenitor cell gene therapy (HSPC-GT) for metachromatic leukodystrophy (MLD): Clinical outcomes from 33 patients

2020 ◽  
Vol 129 (2) ◽  
pp. S59
Author(s):  
Francesca Fumagalli ◽  
Valeria Calbi ◽  
Maria Sessa ◽  
Alberto Zambon ◽  
Cristina Baldoli ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Clinical Outcomes ◽  
Progenitor Cell ◽  
Metachromatic Leukodystrophy ◽  
Hematopoietic Stem ◽  
Cell Gene

A Mutation-Agnostic Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy

2021 ◽  
Author(s):  
Justin S. Antony ◽  
Alberto Daniel-Moreno ◽  
Andrés Lamsfus-Calle ◽  
Janani Raju ◽  
Merve Kaftancioglu ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Metachromatic Leukodystrophy ◽  
Hematopoietic Stem ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome

2021 ◽  
Vol 385 (21) ◽  
pp. 1929-1940 ◽  
Author(s):  
Bernhard Gentner ◽  
Francesca Tucci ◽  
Stefania Galimberti ◽  
Francesca Fumagalli ◽  
Maurizio De Pellegrin ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Progenitor Cell ◽  
Hurler Syndrome ◽  
Hematopoietic Stem ◽  
Cell Gene
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