Table 1. Sequence of the oligonucleotides tested for in vivo gene repair

These oligonucleotides (ODNs) were used in the gene repair experiments detailed in the text. Sequences are presented 5' to 3', left to right. "WTS" is wild type sense sequence and "WTAS" is wild type antisense sequence. Treatment with these two ODNs is hypothesized to lead to conversion of the rd1 mutation to wild type in genomic DNA. "WTSscr7" is wild type sense sequence at the 5' and 3' ends, but the middle seven bases, though the same as those in wild type, are out of order, or scrambled (i.e., containing mismatches to the genomic sequence). "WTSscr25" contains the same bases as WTS but with all bases out of order compared to wild type sequence. "rd1S" is rd1 sense sequence. That is, it contains the rd1 point mutation, an adenine, rather than the wild type cytosine, at the 13^th position, but is otherwise identical to WTS. The last three ODNs should not be able to induce genomic base conversion. Asterisk (*) represents phoshphorothiate linkages, which were used to prevent nuclease degradation.

ODN name                 Sequence
--------   -------------------------------------
WTS        C*C*T*T*C*C*AACCTACGTAGCA*G*A*A*A*G*T
WTAS       A*C*T*T*T*C*TGCTACGTAGGTT*G*G*A*A*G*G
WTSscr7    C*C*T*T*C*C*AACAACGTCTGCA*G*A*A*A*G*T
WTSscr25   A*A*T*C*A*C*AGTTGCCTATAGG*A*C*C*C*C*A
rd1S       C*C*T*T*C*C*AACCTAAGTAGCA*G*A*A*A*G*T