PHRM3021 Lecture Notes - Lecture 15: Polylysine, Protamine, Acid Dissociation Constant
Document Summary
Introduction of genes into defective cells of particular organs to treat inherited disorders (haemophilia (factor viii)), cystic fibrosis (cf transport regulator) or acquired genetic disorders (e. g. cancer tumour suppression protein p53) Introduction of gene sequences that encode key antigens (epitopes) from pathogens or non-self cells (cancer) Cellular synthesis cell mediated immune response. Antisense/sirna/crispr-cas9: designed to hybridize to specific regions of mrna to interfere with normal processing. Reduced expression of coded protein or edit the gene sequence by removing and replacing nucleic acids (gene editing) Relatively large to huge oligo/poly nucleo(cid:415)des, typically at least 10 to > 5 k bases/base-pairs. Hydrophilic: phosphate backbone, deoxyribose/ribose (sugars), hydrophilic bases. Traffic through the cytoplasm and into the nucleus. Poor membrane permeability: large molecular volume (double helix, negatively charged (repulsion from surface of eukaryotes) Unstable in biological environment (short half-life: antisense oligonucleotide completely degraded in 30 min.
