The potential role of cell penetrating peptides in the intracellular delivery of proteins for therapy of erythroid related disorders.

ABSTRACT: The erythroid related disorders (ERDs) represent a large group of hematological diseases, which in most cases are attributed either to the deficiency or malfunction of biosynthetic enzymes or oxygen transport proteins. Current treatments for these disorders include histo-compatible erythrocyte transfusions or allogeneic hematopoietic stem cell (HSC) transplantation. Gene therapy delivered via suitable viral vectors or genetically modified HSCs have been under way. Protein Transduction Domain (PTD) technology has allowed the production and intracellular delivery of recombinant therapeutic proteins, bearing Cell Penetrating Peptides (CPPs), into a variety of mammalian cells. Remarkable progress in the field of protein transduction leads to the development of novel protein therapeutics (CPP-m

SUBMITTER: Papadopoulou LC 

PROVIDER: S-EPMC3816679 | biostudies-literature | 2013 Jan

REPOSITORIES: biostudies-literature