Research ArticleHEALTH AND MEDICINE
Intracerebroventricular delivery of hematopoietic progenitors results in rapid and robust engraftment of microglia-like cells
- Alessia Capotondo1,*,
- Rita Milazzo1,*,
- Jose M. Garcia-Manteiga2,
- Eleonora Cavalca1,3,
- Annita Montepeloso1,3,
- Brian S. Garrison4,5,
- Marco Peviani1,3,
- Derrick J. Rossi4,5 and
- Alessandra Biffi1,3,6,†
- 1San Raffaele Telethon Institute for Gene Therapy, Division of Regenerative Medicine, Stem Cell and Gene Therapy, San Raffaele Scientific Institute, Milano, Italy.
- 2Centre for Translational Genomics and Bioinformatics, IRCCS San Raffaele Scientific Institute, Milano, Italy.
- 3Gene Therapy Program, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Boston, MA 02115, USA.
- 4Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA.
- 5Program in Cellular and Molecular Medicine, Department of Medicine, Boston Children’s Hospital, Boston, MA 02115, USA.
- 6Gene Therapy Program, Department of Medicine, Boston Children’s Hospital, Boston, MA 02115, USA.
- ↵†Corresponding author. Email: alessandra.biffi{at}childrens.harvard.edu
↵* These authors contributed equally to this work.
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Science Advances 06 Dec 2017:
Vol. 3, no. 12, e1701211
DOI: 10.1126/sciadv.1701211
Vol. 3, no. 12, e1701211
DOI: 10.1126/sciadv.1701211
Alessia Capotondo
San Raffaele Telethon Institute for Gene Therapy, Division of Regenerative Medicine, Stem Cell and Gene Therapy, San Raffaele Scientific Institute, Milano, Italy.
Rita Milazzo
San Raffaele Telethon Institute for Gene Therapy, Division of Regenerative Medicine, Stem Cell and Gene Therapy, San Raffaele Scientific Institute, Milano, Italy.
Jose M. Garcia-Manteiga
Centre for Translational Genomics and Bioinformatics, IRCCS San Raffaele Scientific Institute, Milano, Italy.
Eleonora Cavalca
San Raffaele Telethon Institute for Gene Therapy, Division of Regenerative Medicine, Stem Cell and Gene Therapy, San Raffaele Scientific Institute, Milano, Italy.Gene Therapy Program, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Boston, MA 02115, USA.
Annita Montepeloso
San Raffaele Telethon Institute for Gene Therapy, Division of Regenerative Medicine, Stem Cell and Gene Therapy, San Raffaele Scientific Institute, Milano, Italy.Gene Therapy Program, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Boston, MA 02115, USA.
Brian S. Garrison
Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA.Program in Cellular and Molecular Medicine, Department of Medicine, Boston Children’s Hospital, Boston, MA 02115, USA.
Marco Peviani
San Raffaele Telethon Institute for Gene Therapy, Division of Regenerative Medicine, Stem Cell and Gene Therapy, San Raffaele Scientific Institute, Milano, Italy.Gene Therapy Program, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Boston, MA 02115, USA.
Derrick J. Rossi
Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA.Program in Cellular and Molecular Medicine, Department of Medicine, Boston Children’s Hospital, Boston, MA 02115, USA.
Alessandra Biffi
San Raffaele Telethon Institute for Gene Therapy, Division of Regenerative Medicine, Stem Cell and Gene Therapy, San Raffaele Scientific Institute, Milano, Italy.Gene Therapy Program, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Boston, MA 02115, USA.Gene Therapy Program, Department of Medicine, Boston Children’s Hospital, Boston, MA 02115, USA.