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• W2115720194 abstract "Patients with Duchenne muscular dystrophy (DMD), an X-linked lethal muscle-wasting disease, have abnormal expression of the protein dystrophin within their muscle fibres. In the mdx mouse model of this condition, both germline and neonatal somatic gene transfers of dystrophin cDNAs have demonstrated the potential of gene therapy in treating DMD. However, in many DMD patients, there appears to be no dystrophin expression when muscle biopsies are immunostained or western blots are performed. This raises the possibility that the expression of dystrophin following gene transfer might trigger a destructive immune response against this 'neoantigen'. Immune responses can also be generated against the gene transfer vector used to transfect the dystrophic muscle, and the combined immune response could further damage the already inflamed muscle. These problems are now beginning to be investigated in immunocompetent mdx mice. Although much work remains to be done, there are promising indications that these immune responses might not prove as much of a concern as originally envisaged." @default.
• W2115720194 created "2016-06-24" @default.
• W2115720194 creator A5021347280 @default.
• W2115720194 creator A5040149448 @default.
• W2115720194 creator A5075050642 @default.
• W2115720194 date "2002-11-04" @default.
• W2115720194 modified "2023-09-23" @default.
• W2115720194 title "Immunological hurdles in the path to gene therapy for Duchenne muscular dystrophy" @default.
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