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

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

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 ◽

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

10.1016/j.ymgme.2019.11.135 ◽

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 ◽

Hematopoietic Stem ◽

Ex vivo hematopoietic stem cell gene therapy for mucopolysaccharidosis type I (Hurler syndrome)

10.1016/j.ymgme.2020.12.087 ◽

2021 ◽

Vol 132 (2) ◽

pp. S42-S43

Author(s):

Bernhard Gentner ◽

Maria Ester Bernardo ◽

Francesca Tucci ◽

Francesca Fumagalli ◽

Silvia Pontesilli ◽

...

Keyword(s):

Gene Therapy ◽

Ex Vivo ◽

Type I ◽

Mucopolysaccharidosis Type ◽

Hurler Syndrome ◽

Mucopolysaccharidosis Type I ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

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 ◽

Faculty Opinions recommendation of Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction.

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

10.3410/f.720388464.793515025 ◽

2016 ◽

Author(s):

Fabio Candotti

Keyword(s):

Gene Therapy ◽

Severe Combined Immunodeficiency ◽

Combined Immunodeficiency ◽

Hematopoietic Stem ◽

Immunological Recovery ◽

Metabolic Correction ◽

Cell Gene ◽

Stem Cell Gene ◽

Long-term hematopoietic stem cell gene therapy corrects neuromuscular manifestations in preclinical study of Pompe mice

10.1016/j.ymgme.2020.12.263 ◽

2021 ◽

Vol 132 (2) ◽

pp. S107

Author(s):

Niek P. van Til ◽

Yildirim Dogan ◽

Cecilia Barese ◽

Zeenath Unnisa ◽

Swaroopa Guda ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Preclinical Study ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

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 ◽

Historical Perspective on the Current Renaissance for Hematopoietic Stem Cell Gene Therapy

Hematology/Oncology Clinics of North America ◽

10.1016/j.hoc.2017.06.006 ◽

2017 ◽

Vol 31 (5) ◽

pp. 721-735 ◽

Cited By ~ 11

Author(s):

Donald B. Kohn

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Historical Perspective ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE

Molecular Therapy — Methods & Clinical Development ◽

10.1016/j.omtm.2018.01.001 ◽

2018 ◽

Vol 8 ◽

pp. 152-165 ◽

Cited By ~ 11

Author(s):

Rana Yadak ◽

Raquel Cabrera-Pérez ◽

Javier Torres-Torronteras ◽

Marianna Bugiani ◽

Joost C. Haeck ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Mouse Model ◽

Safety Evaluation ◽

Efficacy And Safety ◽

Hematopoietic Stem ◽

Preclinical Efficacy ◽

Cell Gene ◽

Stem Cell Gene ◽