Gene therapy for peripheral nervous system diseases.
Peripheral nerve diseases, also known as peripheral neuropathies, affect 15-20 million of Americans and diabetic neuropathy is the most common condition. Currently, the treatment of peripheral neuropathies is more focused on managing pain rather than providing permissive conditions for regeneration. Despite advances in microsurgical techniques, including nerve grafting and reanastomosis, axonal regeneration after peripheral nerve injury remains suboptimal. Also, no satisfactory treatments are available at this time for peripheral neurodegeneration occurring in motor neuron diseases (MND), including amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA). Peripheral nerves have the inherent capacity of regeneration. Gene therapy strategies focused on neuroprotection may help optimizing axonal regrowth. A better understanding of the cellular and molecular events involved in axonal degeneration and regeneration have helped researchers to identify targets for intervention. This review summarizes the current state on the clinical experience as well as gene therapy strategies for peripheral neuropathies, including MND, peripheral nerve injury, neuropathic pain, and diabetic neuropathy.
Federici
Thais
T
Cleveland Clinic, Department of Neurosciences and Center for Neurological Restoration, 9500 Euclid Avenue, Cleveland, Ohio 44195, USA.
Boulis
Nicholas
N
D002607:Charcot-Marie-Tooth Disease / Q000628:therapy; D003929:Diabetic Neuropathies / Q000628:therapy; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006801:Humans; D008959:Models, Neurological; D016472:Motor Neuron Disease / Q000628:therapy; D009410:Nerve Degeneration / Q000628:therapy; D009416:Nerve Regeneration; D059408:Pain Management; D059348:Peripheral Nerve Injuries; D010523:Peripheral Nervous System Diseases / Q000628:therapy*; D014780:Viruses / Q000235:genetics