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Ideggyógyászati Szemle

1995. MÁRCIUS 01.

THE PRINCIPLES OF GENE THERAPY FOR GENETIC DISEASES OF MUSCLE (DUCHENNE DYSTROPHY), SPINAL MOTOR NEURONS (SPINAL MUSCULAR ATROPHY) AND SCHWANN CELLS (SCHWANNOPATHIES)

GYULA Acsadi, ÁGNES Jani, HANNS Lochmüller, GEORGE Kárpáti

Gene replacement therapy using adenoviral vectors in genetic diseases of the motor unit is a potential treatment modality. ln preclinical experiments, promising results have been obtained by dystrophin minigene replacement in mdx mice. There are several serious problems that remain to be resolved (i. e. immunologically mediated elimination of transduced cells) before this approach can be used in human clinical trials.