Liver-targeting Gene Therapy Reduces Extra Phenylalanine in PKU Mice

Liver-targeting Gene Therapy Reduces Extra Phenylalanine in PKU Mice

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Liver-targeting Gene Therapy Reduces Extra Phenylalanine in PKU Mice

Delivering the human PAL gene — phenylalanine amino lyase — into the liver cells of phenylketonuria (PKU) mouse models via gene therapy can safely reduce excess phenylalanine in the long-term, a recent study found. The researchers noted that, while prior approaches either reversed excess phenylalanine only in male animals or required lifelong injections, this gene therapy approach was effective in both male and female mice and its effects were durable, lasting at least six months.…

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