I work in gene therapy and can tell you that it has huge risks, which means that it’s mainly considered for diseases that have no other treatment and would otherwise be fatal. For example, the latest gene therapy trial for X-linked myotubular myopathy has had four children die, probably because of AAV toxicity. The reason gene therapy is being considered for XLMTM is because it involves loss of function of a single gene in a specific cell type, as well as meeting the criteria I said before. Mood disorders aren’t good candidates for gene therapy.
Yes, I saw that case. It seems that they were using an extremely high viral dosage. In my understanding one of the reasons AAV is typically used is the low immunogenicity at normal dosages and overall good safety profile. Obviously no pioneering method is without risk.
Whether mood disorders qualify under your criteria is debatable—certainly for a large proportion of people no currently available treatment is effective. Fatality rates may be relatively low, but I suspect a fairly large cohort of people living with mood disorders would rather tolerate some risk than live out the rest of their lives with no hope of treatment.
I work in gene therapy and can tell you that it has huge risks, which means that it’s mainly considered for diseases that have no other treatment and would otherwise be fatal. For example, the latest gene therapy trial for X-linked myotubular myopathy has had four children die, probably because of AAV toxicity. The reason gene therapy is being considered for XLMTM is because it involves loss of function of a single gene in a specific cell type, as well as meeting the criteria I said before. Mood disorders aren’t good candidates for gene therapy.
Yes, I saw that case. It seems that they were using an extremely high viral dosage. In my understanding one of the reasons AAV is typically used is the low immunogenicity at normal dosages and overall good safety profile. Obviously no pioneering method is without risk.
Whether mood disorders qualify under your criteria is debatable—certainly for a large proportion of people no currently available treatment is effective. Fatality rates may be relatively low, but I suspect a fairly large cohort of people living with mood disorders would rather tolerate some risk than live out the rest of their lives with no hope of treatment.