CALD and MLD
Автор: ASGCT
Загружено: 2019-02-19
Просмотров: 7607
Описание:
Leukodystrophies are a set of rare genetic disorders that affect how the central nervous system functions. Since these disorders are all caused by a faulty genes, gene therapy might be able to help.
Leukodystrophies are genetic disorders that lead to the deterioration or malformation of the myelin sheath, a fatty insulation that wraps around nerve axons and is essential for proper neuron function. When the myelin sheath is altered it makes it hard for our nervous system to send messages to different parts of our body, which can impair walking, speech, and vision.
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