Cell penetrating peptides

a cell-penetration and peptide technology, applied in the field of peptides, can solve the problems of poor observation of pmo in cells and large effort, and achieve the effect of increasing stability and resistance to degradation

Inactive Publication Date: 2014-02-20
MEDICAL RESEARCH COUNCIL
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

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Benefits of technology

The present invention provides peptides that can be used to treat Duchenne Muscle Dystrophy (DMD) by inducing exon skipping and producing normal dystophin. These peptides have high serum stability and can be modified to increase their effectiveness, such as by adding lysine residues to improve water solubility or adding cysteine at the N-terminus to allow for disulphide bond formation or bromoacetylation. The use of PNADMD or PMODMD in conjunction with another type of molecule, such as an oligonucleotide, can further improve the efficiency of peptides in treating DMD.

Problems solved by technology

In cell culture, both PNA and PMO are observed to enter cells only rather poorly and therefore much effort has been expended to develop methods of enhancing cell delivery.

Method used

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Embodiment Construction

[0187]The details of one or more embodiments of the invention are set forth in the accompanying description below including specific details of the best mode contemplated by the inventors for carrying out the invention, by way of example. It will be apparent to one skilled in the art that the present invention may be practiced without limitation to these specific details.

[0188]The inventors have sought to design improved cell penetrating peptides. In particular they have sought a CPP that a) is stabilized to proteolysis and is thus suitable for in vivo use, b) has enhanced activity over R6Pen and (R-Ahx-R)4 in a HeLa cell nuclear activity model when conjugated to a PNA sequence targeting the luciferase model intron and c) shows enhanced activity over naked PNA when conjugated to a PNA targeting exon 23 in the mouse mdx model.

[0189]In view of this, the inventors have designed a series of peptides, called PNA Internalizing Peptides (Pip) that differ in several key aspects from the pre...

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Abstract

The present invention discloses cell penetrating peptides and conjugates of a cell penetrating peptide and a cargo molecule.

Description

CROSS REFERENCE TO RELATED APPLICATIONS[0001]The present application is a continuation application and claims priority from U.S. patent application Ser. No. 12 / 996,307, filed Dec. 3, 2010, which is the National Stage application of PCT Application No. PCT / GB2009 / 001261, filed May 15, 2009, which claims priority from U.S. Provisional Application Ser. No. 61 / 058,722, filed Jun. 4, 2008, all of which are incorporated by reference in their entireties herein.FIELD OF THE INVENTION[0002]The present invention relates to peptides particularly, although not exclusively, to cell penetrating peptides and to conjugates of a cell penetrating peptide and a cargo molecule.BACKGROUND TO THE INVENTION[0003]Oligonucleotides (ONs) that target essential RNA sequences have found numerous recent applications in the modulation of gene expression in cells and as potential therapeutics1,2. A mechanistic advantage of steric blocking ONs over RNase H-inducing antisense ONs and RISC-inducing siRNA reagents is ...

Claims

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Application Information

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Patent Type & Authority Applications(United States)
IPC IPC(8): A61K47/48A61K31/7125
CPCA61K31/7125A61K47/48246C07K7/02C07K14/001C07K2319/10A61K47/64A61P17/00A61P21/00A61P21/04A61P25/14A61P25/16A61P25/28A61P3/00A61P35/00A61P7/06A61P9/00
Inventor GAIT, MICHAEL JOHNARZUMANOV, ANDREY ALEXANDROVICHIVANOVA, GABRIELA DIMITROVAL
Owner MEDICAL RESEARCH COUNCIL
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