Challenges for gene transfer to the respiratory muscles lessons from animal models

Table 3.1 shows a list of some of the more common or prototypical neuromuscular disorders which can affect the diaphragm and other respiratory muscles. As can be seen, respiratory muscle weakness can be caused by a large and diverse number of diseases acting at different levels of the nervous system, including the central nervous system, the spinal cord, the nerves, the neuromuscular junction, and the muscle. In principal, gene therapy could be used to treat several of these conditions, either by correcting an underlying genetic defect or by supplying some other therapeutic gene product. For the purposes of this chapter, we will limit our discussion to disorders which are potentially treatable by gene transfer to the muscle itself,

Table 3.1 Neuromuscular Disorders Affecting Respiratory Muscle Function Muscular Dystrophies

Duchenne Muscular Dystrophy Becker Muscular Dystrophy Limb-Girdle Muscular Dystrophy Myotonic Dystrophy

Facioscapulohumeral Muscular Dystrophy Congenital Muscular Dystrophy Metabolic Diseases of Muscle Acid Maltase Deficiency Mitochondrial Myopathies Carnitine Palmityl Transferase Deficiency Other Myopathies

Polymyositis or Dermatomyositis Corticosteroid-Induced Diseases of the Neuromuscular Junction Myasthenia Gravis Eaton-Lambert Syndrome Diseases of Peripheral Nerve Charcot-Marie-Tooth Disease Friedreich's Ataxia

Chronic Inflammatory Demyelinating Polyneuropathy Guillain-Barre Syndrome Diseases of the Motor Neuron Amyotrophic Lateral Sclerosis Postpolio Syndrome Spinal Muscular Atrophy Other Diseases Involving the Spinal Cord Traumatic Injury Multiple Sclerosis with a particular focus on the diaphragm. In animal models, gene transfer to the diaphragm has been accomplished through one of 3 major routes of vector administration: (1) direct intramuscular diaphragmatic injection; (2) intracavitary (pleural, peritoneal or in utero) injection; and (3) intravascular administration.

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