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Recently, genetically modified hematopoietic stem cells for ex vivo gene therapy have been tested as an alternative option and are expected to eventually become standard treatment for X-ALD

Pollizzi KN, Patel CH, Sun IH, Oh MH, Waickman AT, Wen J, et al

Activation of this pathway influences actin-myosin interactions and the organization of the cytoskeleton, which are essential for maintaining muscle tension and force generation