In silico and in vitro evaluation of exonic and intronic off-target effects form a critical element of therapeutic ASO gapmer optimization
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Emma L. Koppe | T. Gant | N. Gooderham | Paul A. Wilson | K. Maratou | L. Warnock | M. Edbrooke | K. Moores | J. Kitson | W. Wixted | J. Parry | S. A. Hughes | P. Kamola | A. Panjwani | Gemma Turner | Sofie Eriksson | Gaelle A. Douillard Guilloux | Kenneth L. Clark