Name	Target Gene	Target Site	Description	Patient	Sequence 5'->3'
Nontargeting	Nontargeting	None	Negative Control ASO	All	UUAGCAUGUCCUGCCAGCUGUUCUA
SCR-AUG	Nontargeting	None	ASO optimization Scramble Control	Unaffected	UUAGUUGUCUUGCCGAUCUGCACCA
MM-AUG	Nontargeting	None	ASO optimization Mismatch Control	Unaffected	UCUAAGUCACACAUGCUCUCAGCUC
AUG	TNNT2	Translation start site	ASO optimization	Unaffected	UCUAUGUCAGACAUGGUCUCUGCUC
Splice Donor	TNNT2	Splice donor site	ASO optimization	Unaffected	CCAGAGACUUACCUUCUGCCCUGGU
Splice Acceptor	TNNT2	Splice acceptor site	ASO optimization	Unaffected	UCUUCUUCAUCUUCUAAAUGAAACA
Approved	DMD	Exon 45	FDA approved ASO	Patient 1	CAAUGCCAUCCUGGAGUUCCUG
Phase II	DMD	Exon 45	ASO in clinical trials	Patient 1	CGCUGCCCAAUGCCAUCC
MM-1	Nontargeting	None	Patient-specific ASO Mismatch Control	Patients 2a/2b	GUUCACAGGCAAAUUAGAAACCAUG
SCR-1	Nontargeting	None	Patient-specific ASO Scramble Control	Patients 2a/2b	AUAUUAGGUCUAAGUGCCGGAAGUA
ASO-1	DMD	Deep intronic variant	Patient-specific ASO	Patients 2a/2b	GUUCUCAGGGAAAUUUGAAAGCAUG
MM-2	Nontargeting	None	Patient-specific ASO Mismatch Control	Patients 2a/2b	GUAGAUCUCUGGGAAUUUUGUAAGC
SCR-2	Nontargeting	None	Patient-specific ASO Scramble Control	Patients 2a/2b	GUGGCAGGUUGACUUAUAAGAUACA
ASO-2	DMD	Deep intronic variant	Patient-specific ASO	Patients 2a/2b	GUAGUUCUCAGGGAAAUUUGAAAGC