[HTML][HTML] Chromosome targeting at short polypurine sites by cationic triplex-forming oligonucleotides

KM Vasquez, JM Dagle, DL Weeks… - Journal of Biological …, 2001 - ASBMB
Triplex-forming oligonucleotides (TFOs) bind specifically to duplex DNA and provide a
strategy for site-directed modification of genomic DNA. Recently we demonstrated TFO-
mediated targeted gene knockout following systemic administration in animals. However, a
limitation to this approach is the requirement for a polypurine tract (typically 15–30 base
pairs (bp)) in the target DNA to afford high affinity third strand binding, thus restricting the
number of sites available for effective targeting. To overcome this limitation, we have …