Identification of Hematopoietic Stem Cell–Specific miRNAs Enables Gene Therapy of Globoid Cell Leukodystrophy-Reference-Cited by-同舟云学术

Identification of Hematopoietic Stem Cell–Specific miRNAs Enables Gene Therapy of Globoid Cell Leukodystrophy

Published:2010-11-17 Issue:58 Volume:2 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Gentner Bernhard¹²,Visigalli Ilaria¹²,Hiramatsu Hidefumi³,Lechman Eric³,Ungari Silvia¹²,Giustacchini Alice¹²,Schira Giulia¹,Amendola Mario¹,Quattrini Angelo⁴,Martino Sabata⁵,Orlacchio Aldo⁵,Dick John E.³,Biffi Alessandra¹,Naldini Luigi¹²

Affiliation:

1. San Raffaele Telethon Institute for Gene Therapy, Division of Regenerative Medicine, Stem Cells and Gene Therapy, San Raffaele Scientific Institute, Milan 20132, Italy.

2. Vita Salute San Raffaele University, San Raffaele Scientific Institute, Milan 20132, Italy.

3. Division of Cell and Molecular Biology, University Health Network, Toronto, Ontario, Canada M5G 1L7.

4. Division of Neuroscience, Department of Neurology and Institute of Experimental Neurology, San Raffaele Scientific Institute, Milan 20132, Italy.

5. Department of Experimental Medicine and Biochemical Sciences, University of Perugia, Perugia 06100, Italy.

Abstract

Hematopoietic stem cell–specific microRNAs allow regulation of therapeutic transgene expression and enable effective gene therapy of globoid cell leukodystrophy.

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference35 articles.

1. Hematopoietic Stem-Cell Transplantation

2. Gene Therapy of Human Severe Combined Immunodeficiency (SCID)-X1 Disease

3. Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector

4. Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency

5. Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

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