Gene Therapy Technique Slows Brain Disease (2 of 2) (IMAGE)
Caption
This figure represents four cells, four purified CD34+ (the CD34+ cell population comprises true hematopoetic stem cell) from patient P1. These cells were sampled/purifed from his bone marrow 2 years after gene therapy. The blue circles represent nucleus of cells. The red dots in one cell are peroxisomes (intracellular organelle) in which the ALD protein encoded by the integrated lentiviral vector is expressed. In other cells, the red dots are not detectable because these cells are not corrected after gene therapy and the mutation of ABCD1 gene in this patient results in complete absence of ALD protein expression in peroxisomes. The exact ratio of corrected/non-corrected CD34% cells in patient P1, two years after gene therapy, is 18 percent.
Credit
Image courtesy of Patrick Aubourg
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