细胞技术

This chapter describes the manufacturing process to a certain level for a possible oligonucleotide cargo and a peptide API in a multi-kilogram scale from a manufacture’s point of view. In the concluding remarks, possible conjugation methods will be discussed from an industrial-scale pe ...

A major challenge confronting the further advancement of using molecular transporters conjugated to small molecular weight therapeutics in the clinic is the development of linkers that would allow for the controllable release of a free drug/probe only after cell entry. Development of ...

RNA-binding proteins (RBPs) are fundamental regulatory proteins for all forms of transcriptional and posttranscriptional control of gene expression. However, isolating RBPs is technically challenging for investigators. Currently, the most widely used techniques to isol ...

The first series of cell-penetrating penta-peptides (CPP5s) were discovered as cytoprotective penta-peptides designed from the Bax-inhibiting domain of Ku70. Bax is an inducer of programmed cell death, and Ku70 is a multifunctional protein maintaining genomic stability and pro ...

Cell-penetrating peptides (CPPs) are a very interesting class of molecules to be introduced in gene and siRNA vectors. They can be used to overcome one of the biggest hurdles in gene and siRNA delivery in vitro and in vivo, the transfer across cell membranes. This chapter describes protocols for the s ...

Cell-penetrating peptides (CPPs), in particular TATp, have been widely used for intracellular delivery of various cargoes, both in vitro and in vivo. Modifications of nanoparticles with CPPs require either covalent or noncovalent approach. Here we describe various methods to attach ...

Two of the most promising and complex areas in biologics development, either as research tools or potential therapeutics, are cell-penetrating peptides (CPPs) and RNA interference (RNAi) modulators. Consequently, the combined application of these technologies in pursuit of imp ...

Each normal organ and pathological condition expresses a distinct set of molecules on their vasculature. These molecular signatures have been efficiently profiled using in vivo phage display technology. Using this technology, several peptides homing specifically to tumour bl ...

Cell-penetrating peptides (CPPs) have been widely used for a cellular delivery of biologically relevant cargoes including antisense peptide nucleic acids (PNAs). Although chemical conjugation of PNA to a variety of CPPs significantly improves the cellular uptake of the PNAs, bioa ...

Nucleoside phosphates can bind to many functional proteins like G-proteins or other GTP-binding proteins in signal transduction or translation processes. Till now internalization of nucleoside phosphates into live cells remains a challenge. We study the internalization of a flu ...

One oligonucleotide-based approach that appear very promising for the treatment of different genetic disorders are based on so-called splice-correcting oligonucleotides (SCOs) that are exploited to manipulate splicing patterns. In order to increase the bioavailability, c ...

The development of short-interfering RNA (siRNA) has provided great hope for therapeutic targeting of specific genes responsible for pathological disorders. However, the poor cellular uptake of siRNA together with the low permeability of the cell membrane to negatively charged mo ...

A major hurdle in drug delivery today is for the drug to reach inside the cell to exert its biological effect. Many drug candidates are hydrophilic and are therefore not able to cross the hydrophobic plasma membrane, which serves to protect the cell from foreign molecules and pathogens. One promisi ...

Antisense oligonucleotides (AOs) are effective splice switching agents and have potential as therapeutics via the exclusion or inclusion of specific target gene exons to ameliorate and modify disease progression. The leading example is Duchenne muscular dystrophy (DMD), a fatal m ...

The discovery of cell-penetrating peptides (CPP) has provided the scientific community with relatively small and increasingly cost-effective molecular agents that readily cross the normally impermeable cell membrane. Thus, as either inert delivery vectors or biologically ...

Protein transduction domains (PTD) or cell-penetrating peptides (CPPs) are small peptides that are able to carry proteins, nucleic acid, and particles across the cellular membranes into cells. PTDs can be classified into three types: (1) positively charged, cationic peptides, compri ...

G protein-coupled receptors (GPCR) are a superfamily of receptors that are vital in a wide array of physiological processes. Modulation of GPCR signaling has been an intensive area of therapeutic study, mainly due to the diverse pathophysiological significance of GPCRs. Pepducins are c ...

Cell-Penetrating Peptides (CPPs) are small peptides internalized by live cells, gaining access to their cytoplasm and intracellular organelles (i.e., mitochondria, nucleus) and are used as pharmacological tools. This is indeed a very important issue, fully justifying the efforts of ...

Proteins are essential components of cellular processes inside cells, and their interactions between each other and with genes are important for the normal physiological functioning of cells as well as for disease states. Modulating protein interactions by different means can pote ...

As our knowledge increases about the diversity in uptake mechanisms displayed by cell-penetrating peptides (CPP), the concept of CPP uptake kinetics becomes increasingly complex. Here, we present three different assays that can be used for studying different kinetic aspects of CPP-m ...