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Intracellular components of autologous red blood cells refers to the internal biological environment and molecular constituents, such as hemoglobin and various enzymes, found within a patient's own erythrocytes [Rossi et al., 2016, Expert Opin Drug Deliv]. In the context of modern pharmacology, this is not a traditional molecular target like a receptor or ion channel, but rather a specialized biological carrier used in erythrocyte-mediated drug delivery systems (EDDS) [Chessa et al., 2014, Orphanet J Rare Dis]. This technology involves the ex vivo loading of therapeutic agents—including corticosteroids, enzymes, or oligonucleotides—into the cytoplasm of autologous red blood cells, which are then re-infused into the patient [EryDel SpA, 2023, Technology Overview]. The red blood cells serve as biocompatible bioreactors or long-circulating reservoirs that protect the encapsulated payload from premature degradation and immune recognition [Godfrin et al., 2012, Leukemia & Lymphoma]. This approach is primarily investigated for treating rare genetic diseases, such as Ataxia-Telangiectasia, and metabolic disorders like Phenylketonuria, by providing a sustained release of the drug or allowing intracellular enzymatic activity to process systemic metabolites [Chessa et al., 2014, Orphanet J Rare Dis]. Consequently, while the target is the internal space of the cell for encapsulation purposes, the therapeutic effect is mediated by the payload drug acting on its own specific molecular targets.
Encapsulation of therapeutic agents within the cytoplasm of autologous red blood cells to extend pharmacological half-life, reduce systemic toxicity, and shield the payload from immune recognition [Rossi et al., 2016, Expert Opin Drug Deliv].
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