Abstract:
:Ex vivo gene therapy is emerging as a promising approach for the treatment of neurodegenerative diseases and central nervous system (CNS) trauma. We have shown previously that human adult astrocytes can be expanded in vitro and can express various therapeutic transgenes (Ridet et al. [1999] Hum. Gene Ther. 10:271-280; Serguera et al. [ 2001] Mol. Ther. 3:875-881). Here, we grafted normal and lentivirally-modified human adult astrocytes into the striatum and spinal cord of nude mice to test whether they are suitable candidates for ex vivo CNS gene therapy. Transplanted cells survived for at least 2 months (longest time analyzed) and sustained transgene expression. Importantly, the absence of proliferating cell nuclear antigen (PCNA) staining, a hallmark of cell division, ascertains the safety of these cells. Thus, adult human astrocytes are a promising tool for human CNS repair; they may make autologous ex vivo gene transfer feasible, thereby avoiding the problems of immunological rejection and the side effects of immunosuppressors.
journal_name
J Neurosci Resjournal_title
Journal of neuroscience researchauthors
Ridet JL,Sarkis C,Serguera C,Zennou V,Charneau P,Mallet Jdoi
10.1002/jnr.10617keywords:
subject
Has Abstractpub_date
2003-06-15 00:00:00pages
704-8issue
6eissn
0360-4012issn
1097-4547journal_volume
72pub_type
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