Researchers have designed a drug specifically for an 8-year-old girl with an ultra-rare genetic disease. It's thought to be the first example of a treatment created for just one person that's likely to work only for her. (commonhealth)
Mila Makovec with her grandparents, Jeni and Jim Vitarello, in the hospital after receiving a dose of milasen in 2019
The apparent success of milasen is stunning proof of how these designer genetic drugs, known as antisense oligonucleotides, can be personalized for individuals, researchers say. Mila Makovec walks on a beach as a young child before she was diagnosed with Batten disease, a rare genetic disorder. “I realized later that her vision was going," Vitarello says."She was not able to make as much sense of the world around her."
“This was a really unusual case,” he says. Genes are written like a sentence explaining how a protein should be put together. But in Mila’s case, an extra piece of genetic code was thrown into the middle of that sentence, Yu says. “It acts as a molecular patch,” Yu says. The custom genetic drug simply covers up the messed up part of the gene, like crossing out nonsense.
Milasen is far from a cure, however, and Vitarello says while Mila is getting better in some ways, she seems to be getting worse in others. She no longer responds as well or as quickly to her favorite songs and stories, for instance.
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