Lecture 24 - Genetic Therapies Flashcards
Model Systems?
Yeast to mice, technical difficulty relating t complexity of questions that can be answered
Bench to bedside translation?
disease gene identified, genes disease state is determined, design novel therapies, conventional drug-based OR molecular genetic-basd therapies designed
Gene Therapy Definition?
the transfer of DNA or RNA into cells of organisms to treat disease - somatic cells only at present
Criteria for use of gene therapy?
Life-threatening w no treatment, single gene cause, gene regulaton need not be precise for healthy function, technical problems must be resolvable
Remaining problems of gene therapy?
lack of permanence, immune response, viral vectors, rarity of single gene disorders, effects of insertional mutagenesis, misuse potential, Weismann barrier permeability
Mutation Correction in vivo?
required for gain-of-function mutations where gene augmentation is uneffective; repair at DNA or RNA level
Targeted inhibition of gene expression?
utility in cancer, infectious disease & disorders w gain-of-function mutation; target inhibition at DNA, RNA or protein levels
Recombinant Pharmaceuticals?
produced by expression cloning, large amounts of protein generated, reduces risk of pathogenic contamination, however need avoid side-effects of closely-related proteins
Genetically Engineered Antibodies?
artificial antibodies that target disease associated antigens, killing disease cells e.g. cancer, infection, autoimmune
Monoclonal Antibodies?
from animals cause immune response, technically difficult generation in humans; clonign Ig genes allow artificial combinations of antibody segments (antibody engineering)
Genetically engineered vaccine e.g and types?
e.g. Hep B; nucleic acid, genetic modification of antigens, viruses and microorganisms
