Pip6a
Ac-RXRRBRRXRYQFLIRXRBRXRB-acid,
B is β-Alanine. X is Aminohexanoic acid
Description
Application Data
Description
-
PNA/PMO internalisation peptide 6a
Application Data
-
Catalogue number crb1000352 Molecular Weight 2952.60 Sequence (one letter code) Ac-RXRRBRRXRYQFLIRXRBRXRB-acid,
B is β-Alanine. X is Aminohexanoic acidSequence (three letter code) Ac-Arg-[Ahx]-Arg-Arg-[β-Ala]-Arg-Arg-[Ahx]-Arg-Tyr-Gln-Phe-Leu-Ile-Arg-[Ahx]-Arg-[β-Ala]-Arg-[Ahx]-Arg-[β-Ala]-OH,
β-Ala is β-Alanine and Ahx is Aminohexanoic acidModifications B and β-Ala is β-Alanine. X and Ahx is Aminohexanoic acid Purity >95% Storage -20°C References Betts et al (2012) Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment. Mol. Ther. Nucleic Acids 1 e38 PMID: 23344180
Hammond et al (2016) Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy. PNAS 113(39) 10962 PMID: 27621445
Manufactured in: United Kingdom Pip6a is part of a novel series of transduction peptides termed Pips (PNA/PMO internalisation peptides). Pip peptides were designed around an original R6-penetratin cell penetrating peptide (CPP) and are able to transport PNA/PMO molecules across cell membranes. Pip peptides can be covalently conjugated to PNAs/PMOs to deliver them to a variety of adult tissues, including liver, kidney, skeletal muscle, diaphragm, and heart.
Due to its ability to target the heart, pip6a has important implications for the development of therapeutic antisense oligonucleotide therapy using PMOs for diseases such as Duchenne muscular dystrophy (DMD). DMD causes progressive muscle weakening and often results in cardiac failure and death. Pip6a has also been studied for delivery of antisense oligonucleotide therapy in spinal muscular atrophy (SMA).