Article

Patient-customized oligonucleotide therapy for a rare genetic disease

2020-10-23

Abstract excerpt

This remarkable case report - by authors from Boston Childrens Hospital and funded by Milas Miracle Foundation - describes the development and use of a patient-customised antisense oligonucleotide drug that was tailored specifically against the unique DNA sequence mutation in a 6-year old girl with Batten disease, a degenerative neurological disease due to neuronal accumulation of lipofuscin and characterised by...

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Identifiers and source

Literature Corpus work: de7774ed-3af7-5b7e-9eb5-1b39308c1ae8
DOI: 10.1530/ey.17.14.8

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