Slowing Brain Disease with Gene Therapy

Science, This Week in Science “X-linked adrenoleukodystrophy (ALD), the hereditary brain demyelinating disorder that was featured in the movie...

Science, This Week in Science

“X-linked adrenoleukodystrophy (ALD), the hereditary brain demyelinating disorder that was featured in the movie “Lorenzo’s Oil,” is typically treated by transplantation of bone marrow from matched donors.  This treatment slows progression of the disease by introducing cells that differentiate into myelin-producing cells.”
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