Health & Fitness
Boston Children's Hospital Lab Trying To Develop Drug To Treat Boy's Deadly Rare Disease
"This disease is cruel, it's brutal, and it can take Henry at any moment without warning," his mother Mary Saladino said.
January 19, 2024
BOSTON - Dr. Tim Yu refers to the letters of the genetic code as Lego blocks. His lab at Boston Children's Hospital has become known for putting the Legos together just right and using them to develop precision drugs called Antisense Oligonucleotides or ASOs to treat incredibly rare neurological diseases.
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"You build them like Lego blocks by hooking up the letters to one another. And, when you put them in the order, in the right string of letters, you can get them to target different parts of the genome," Yu told.
It may sound simple but the development of each ASO is tricky and fraught with risks. Yu should know. His lab has been involved in the development of four since 2017.
Find out what's happening in Bostonfor free with the latest updates from Patch.
Last year, scientists at the Northwestern Feinberg School of Medicine were testing ASO candidates in Henry's neurons. Each ASO is made of microscopic synthetic strands of DNA and/or RNA that bind to the genetic mutation that is producing the protein causing Henry's disease.
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