Gene therapy in neurology: review of ongoing clinical trials

Author(s): Maria Judit Molnar,George Nemeth

Gene therapy has entered into its third decade since the first human clinical trial in 1990. It is an expanding field and the use of sequence-targeted regulatory molecules may pave one path to the personalized therapy of genetic disorders. However, gene therapy may not only help patients with monogenic disorders, but may also establish a gene reservoir of the theraputic protein in polygenic, multifactorial disorders and help to destroy malignant tumor cells. Several clinical trials for neurologic syndromes appear quite promising. The field of gene therapy has been driven by genomic technologies, including identifying disease-causing genes and mutations, design of genomic interacting elements to regulate transcription and splicing of specific precursor mRNAs and use of novel noncoding regulatory RNAs. This review covers gene therapy strategies to ameliorate neurologic syndromes of different etiologies, including muscular dystrophies, lysosomal storage diseases, amyloidosis, Alzheimer’s and Parkinson’s disease, amyotrophic lateral sclerosis, pain and brain tumors. The advances in clinical arenas, the successes in this field and the remaining obstacles are highlighted.