Individualized antisense oligonucleotide treatment eligibility of patients living with neurodevelopmental diseases
Annemieke Aartsma-Rus, Claudio Melo de Gusmao, Scott Demarest, Willeke van Roon-Mom, Holm Graessner, Lynn Wein Bush, Charlotte Kogel, Timothy Wei-Wen Yu, Matthis Synofzik, Rebecca Schuele · 2026
Read Individualized antisense oligonucleotide treatment eligibility of patients living with neurodevelopmental diseases by Annemieke Aartsma-Rus, Claudio Melo de Gusmao, Scott Demarest, Willeke van Roon-Mom, Holm Graessner, Lynn Wein Bush, Charlotte Kogel, Timothy Wei-Wen Yu, Matthis Synofzik, Rebecca Schuele (2026) in the Tower of Babel, a free online library of public-domain works preserved in clean, searchable text. No sign-up. The full text is served as a plain-text file at a predictable URL, where text is present: https://antarctic-labs.com/tower-of-babel/pmc-13589468.txt.
Description
SummaryWith advances in genetic diagnostic tools, pathogenic variants in patients with genetic diseases are being identified at an accelerated pace. For a subset of these patients, individualized genetic interventions such as antisense oligonucleotides (ASOs) would address the disease cause. These individualized, or n-of-1, ASOs are currently being clinically applied in dozens of cases but mainly in patients with neurodegenerative diseases.
Catalog record
| CREATOR | Annemieke Aartsma-Rus, Claudio Melo de Gusmao, Scott Demarest, Willeke van Roon-Mom, Holm Graessner, Lynn Wein Bush, Charlotte Kogel, Timothy Wei-Wen Yu, Matthis Synofzik, Rebecca Schuele |
|---|---|
| YEAR | 2026 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 31 KB |
| VERSION | 1.0 |
| LICENSE | CC BY 4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |