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

Hematopoietic Stem ◽

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

10.1055/s-0041-1739647 ◽

2021 ◽

Author(s):

Mirko Essing ◽

Alberto Zambon ◽

Vera Gallo ◽

Cristina Baldoli ◽

Federica Cugnata ◽

...

Keyword(s):

Gene Therapy ◽

Clinical Outcomes ◽

Progenitor Cell ◽

Hematopoietic Stem ◽

Lentiviral hematopoietic stem and progenitor cell gene therapy provides durable clinical benefit in early-symptomatic early-juvenile metachromatic leukodystrophy

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2020.12.081 ◽

2021 ◽

Vol 132 (2) ◽

pp. S40

Author(s):

Francesca Fumagalli ◽

Valeria Calbi ◽

Maria Sessa ◽

Alberto Zambon ◽

Elena Fratini ◽

...

Keyword(s):

Gene Therapy ◽

Clinical Benefit ◽

Progenitor Cell ◽

Hematopoietic Stem ◽

Early Juvenile ◽

Update on safety and efficacy of lentiviral hematopoietic stem cell gene therapy (HSC-GT) for metachromatic leukodystrophy (MLD)

European Journal of Paediatric Neurology ◽

10.1016/j.ejpn.2017.04.986 ◽

2017 ◽

Vol 21 ◽

pp. e20

Author(s):

F. Fumagalli ◽

V. Calbi ◽

A. Zambon ◽

F. Ciotti ◽

L. Lorioli ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Hematopoietic Stem ◽

Safety And Efficacy ◽

Cell Gene ◽

Stem Cell Gene ◽

Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy

Bone Marrow Transplantation ◽

10.1038/s41409-017-0085-1 ◽

2018 ◽

Vol 53 (7) ◽

pp. 913-917 ◽

Cited By ~ 5

Author(s):

Valeria Calbi ◽

Francesca Fumagalli ◽

Giulia Consiglieri ◽

Rachele Penati ◽

Serena Acquati ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Endothelial Injury ◽

Hematopoietic Stem ◽

Post Transplant ◽

Cell Gene ◽

Stem Cell Gene ◽

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

The CRISPR Journal ◽

10.1089/crispr.2021.0075 ◽

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 ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

880. Hematopoietic Stem Cell Gene Therapy for the Treatment of Metachromatic Leukodystrophy: Clinical Trial Design

Molecular Therapy ◽

10.1016/s1525-0016(16)40283-2 ◽

2008 ◽

Vol 16 ◽

pp. S328-S329

Keyword(s):

Clinical Trial ◽

Gene Therapy ◽

Stem Cell ◽

Trial Design ◽

Clinical Trial Design ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

Hematopoietic stem cell gene therapy for metachromatic leukodystrophy and other lysosomal storage diseases

International Journal of Clinical Pharmacology and Therapeutics ◽

10.5414/cpp48035 ◽

2009 ◽

Author(s):

A. Biffi

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Lysosomal Storage Diseases ◽

Lysosomal Storage ◽

Hematopoietic Stem ◽

Storage Diseases ◽

Cell Gene ◽

Stem Cell Gene ◽

Faculty Opinions recommendation of Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.718029893.793484556 ◽

2013 ◽

Author(s):

Gail Herman

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

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

New England Journal of Medicine ◽

10.1056/nejmoa2106596 ◽

2021 ◽

Vol 385 (21) ◽

pp. 1929-1940 ◽

Cited By ~ 1

Author(s):

Bernhard Gentner ◽

Francesca Tucci ◽

Stefania Galimberti ◽

Francesca Fumagalli ◽

Maurizio De Pellegrin ◽

...

Keyword(s):

Gene Therapy ◽

Progenitor Cell ◽

Hurler Syndrome ◽

Hematopoietic Stem ◽

Faculty Opinions recommendation of Repetitive busulfan administration after hematopoietic stem cell gene therapy associated with a dominant HDAC7 clone in a nonhuman primate.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.4242977.4039078 ◽

2010 ◽

Author(s):

Karen E Pollok ◽

Shanbao Cai

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Nonhuman Primate ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽