Bone marrow or hematopoietic stem cell (HSC) transplantation have recently been used to treat clinically severe autoimmune disease, however they are associated with a high risk of graft rejection or graft-versus-host disease. In contrast, Leonard Harrison and colleagues at the Walter and Eliza Hall Institute of Medical Research, Australia demonstrate that syngeneic transplantation of HSCs does not provoke a host immune response and is capable of preventing autoimmune diabetes in mice.
AUTHOR CONTACT:
Leonard C. Harrison
The Walter and Eliza Hall Institute of Medical Research, Parkville, Victoria, Australia.
Phone: 61-3-9345-2460
Fax: 61-3-9347-0852
E-mail: harrison@wehi.edu.au
View the PDF of this article at: https://www.the-jci.org/press/15995.pdf
Rare reversion of mutation provides a basis for natural gene therapy
A rare inherited disorder known as Wiskott-Aldrich syndrome (WAS) results from a mutation in the WAS protein. Two brothers have been identified that carry a second mutation in this protein, which restored protein function in a fraction of their T cells. Understanding the mechanisms underlying this natural form of gene therapy will be of great importance for developing new therapeutic strategies for genetic disorders.
AUTHOR CONTACT:
Fabio Candotti
NIH, Bethesda, Maryland, USA.
Phone: 301-435-2944
Fax: 301-480-3678
E-mail: fabio@nhgri.nih.gov
View the PDF of this article at: https://www.the-jci.org/press/15485.pdf
Disabling a constitutive CTL epitope allows suppression of autoimmune diabetes by intranasal proinsulin peptide
AUTHOR CONTACT
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Contact: Brooke Grindlinger
science_editor@the-jci.org
212-342-9006
Journal of Clinical Investigation
1-May-2003