Gene therapy for peripheral nervous system diseases

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Curr Gene Ther. 2007 Aug;7(4):239-48.

Gene therapy for peripheral nervous system diseases.

Federici T, Boulis N.

Cleveland Clinic, Department of Neurosciences and Center for

Neurological Restoration, 9500 Euclid Avenue, Cleveland, Ohio 44195,

USA.

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.