Designing an 'n of 1' drug for Batten disease | Boston Children's Hospital

Описание к видео Designing an 'n of 1' drug for Batten disease | Boston Children's Hospital

Mila Makovec's CLN7 Batten disease is caused by a unique and unusual genetic mutation. Learn how the mutation damages Mila's nerve cells and how an oligonucleotide drug, designed at Boston Children's Hospital specially for her, fixes her gene.

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