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.
Leonard C. Harrison
The Walter and Eliza Hall Institute of Medical Research, Parkville, Victoria, Australia.
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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.
NIH, Bethesda, Maryland, USA.
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