Peptide nucleic acids: on the road to new gene therapeutic drugs

TY - JOUR

T1 - Peptide nucleic acids

T2 - on the road to new gene therapeutic drugs

AU - Nielsen, Peter E.

PY - 2000/1

Y1 - 2000/1

N2 - Peptide nucleic acid (PNA) is a DNA mimic based on a pseudopeptide (polyamide) backbone. PNA oligomers bind strongly and with high sequence specificity to complementary targets in RNA (or DNA), and they show very high biological stability. Furthermore, studies in cell free systems have demonstrated potent antisense (inhibition of translation) and antigene (inhibition of transcription) activity of PNA. Recently, several studies reporting methods for cellular delivery of PNA as well as antisense effects of PNA in cells ex vivo and in rats have appeared. The potential of developing PNA derived gene therapeutic drugs is discussed.

AB - Peptide nucleic acid (PNA) is a DNA mimic based on a pseudopeptide (polyamide) backbone. PNA oligomers bind strongly and with high sequence specificity to complementary targets in RNA (or DNA), and they show very high biological stability. Furthermore, studies in cell free systems have demonstrated potent antisense (inhibition of translation) and antigene (inhibition of transcription) activity of PNA. Recently, several studies reporting methods for cellular delivery of PNA as well as antisense effects of PNA in cells ex vivo and in rats have appeared. The potential of developing PNA derived gene therapeutic drugs is discussed.

KW - Amino Acid Sequence

KW - Animals

KW - Antisense Elements (Genetics)

KW - Base Sequence

KW - DNA/genetics

KW - Genetic Therapy/methods

KW - Humans

KW - Molecular Mimicry

KW - Molecular Sequence Data

KW - Peptide Nucleic Acids/genetics

M3 - Review

C2 - 10720100

SN - 0901-9928

VL - 86

SP - 3

EP - 7

JO - Pharmacology and Toxicology

JF - Pharmacology and Toxicology

IS - 1

ER -