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itsallrighthere t1_j8ozif0 wrote

Reply to comment by Stephanie87-123 in Recently a gene therapy for a rare inherited neurodegenerative disorder was approved. How come cure is achieved through bone marrow transplantation? by nickoskal024

So if I understand, in the case of genetic metabolic disorders, introducing the missing metabolic function to modified blood cells helps support non modified cells. Do the toxic compounds move from the native cells to the modified cells where they are properly processed?

Do you know where we are in terms of safety for gene therapy?

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Stephanie87-123 t1_j8qwtfn wrote

It really depends on the specific disease and the mechanisms that causes damage to brain cells, but in some cases yes. For most of those disease the toxic compounds will be secreted from the cells, so they are in the extracellular space were the modified cells can take them up and process them.

Gene therapy is not my area of expertise, so someone else might be more up to date on this. The major risk of gene therapy is that the insertion/editing of a gene will lead to unintended disruption of other genes, which worse case scenario might make them more likely to become cancerous. The method used for this treatment, ex vivo gene therapy, were the gene therapy is performed outside of the body is more safe as it allows for extra checks on the cells before they are transplanted back into the patient.

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