Transplantation of wild-type mouse hematopoietic stem and progenitor cells ameliorates deficits in a mouse model of Friedreich’s ataxia-Reference-Cited by-同舟云学术

Transplantation of wild-type mouse hematopoietic stem and progenitor cells ameliorates deficits in a mouse model of Friedreich’s ataxia

Published:2017-10-25 Issue:413 Volume:9 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Rocca Celine J.¹^ORCID,Goodman Spencer M.¹,Dulin Jennifer N.²^ORCID,Haquang Joseph H.¹,Gertsman Ilya¹^ORCID,Blondelle Jordan³^ORCID,Smith Janell L. M.¹,Heyser Charles J.²^ORCID,Cherqui Stephanie¹^ORCID

Affiliation:

1. Division of Genetics, Department of Pediatrics, University of California, San Diego, La Jolla, CA 92093, USA.

2. Department of Neurosciences, University of California, San Diego, La Jolla, CA 92093, USA.

3. Division of Cardiovascular Medicine, University of California, San Diego, La Jolla, CA 92093, USA.

Abstract

YG8R mice, a model of Friedreich’s ataxia, show amelioration of the disease phenotype when transplanted with wild-type mouse hematopoietic stem and progenitor cells.

Funder

National Institutes of Health

National Institute of Health

The Cystinosis Research Foundation

The Sanford Stem Cell Clinical Center

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference65 articles.

1. Friedreich's Ataxia: Autosomal Recessive Disease Caused by an Intronic GAA Triplet Repeat Expansion

2. Studies of human, mouse and yeast homologues indicate a mitochondrial function for frataxin