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*Gene Therapy - Coggle Diagram
*Gene Therapy
Gene therapy
Gene therapy is the transfer of genetic material into cells with the goal of achieving a theraputic effect
Disease types
The two types of dominant diseases in regards to gene therapy are Gain of Function and Haploinsufficiency.
Gain of function
Gain of function mutations cause the gene to produce protein with altered function or loss of regulation
Haploinsufficiency
Haploinsufficiency mutations are when the presence of a single wild-type gene is not sufficient for physiological function.
Recessive Disease
Loss of function
Loss of function mutations cause the loss of expression of a gene or to the expression of an inactive protein
Approaches
There are four approaches to gene therapy: Gene augmentation Gene suppression Gene editing Suicide genes
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Gene suppression represses the expression of a mutant gene e.g. RNA-interface, antisense oligos
The four tools for accomplishing gene expression are: RNAi Artificial miRNA Small hairpin RNA (shRNA) Antisense oligonucleotides
Antisense oligonucleotides can target an mRNA of interest in gene suppression without needing a promoter
Gene editing direcly modifies the host genome at a desired target site e.g. molecular surgery, CRISPR
Suicide genes deliver toxic genes to kill specific cells e.g. thymidine kinase expression in malignant cells
Thymidine kinase is a suicide gene therapy tool that is delivered to tumor cells using an Adenoviral vector.
Route of administration
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Ex vivo gene therapy involves the insertion of a new gene into excised hematopoietic stem cells followed by infusion of those cells back into the host.
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Gene therapy approaches for correcting genetic diseases are based on the mutation pathogenic mechanism.
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A gene therapy expression cassette includes a promoter, a gene of interest and a poly-a signal.