Introduction to the special volume: Gene therapy for neurologic disease

摘要

It is 40 years since Friedmann and Robin first proposed in an article in Science that an approach they termed "gene therapy" might be used to treat human disease [5], and more than 20 years since the first human clinical trial was initiated in 1990. In the early years attention focused on the correction of common monogenic diseases by inserting a normal copy of the relevant gene, and although that promise has not yet been fulfilled, there has been some success in treating rare monogenic diseases affecting hematopoietic cells [1-3]. Studies of gene therapy for diseases affecting the nervous system have largely (though not exclusively) focused on the use of viral vectors for gene transfer to provide targeted delivery of therapeutic molecules into brain or peripheral nervous system. In studies now involving several hundred patients there have been no serious adverse events related to gene transfer into the brain, muscle, or peripheral nerve. But if that is the good news, the bad news is that there have there been no dramatic successes in treating otherwise intractable diseases.