Gene therapy 1
Gene therapy is introduction of normal genes in place of missing or defective gene to correct a genetic disorder.
Types.
removal of defective genes and replacing it with normal ones. Gene replacement.
Correction of defective gene by introducing complementary part gene targeting
Introduction of new copies of a gene. Gene augmentation
Method
Isolation of desired genes/DNA
Transfer of desired DNA into vector
Transfer of DNA through vector to recipient cells
Techniques for injection
Electroporation
Gene gun
Nanoparticles
Retrovirus or adenovirus
Magnetofection
First therapy in 1990 for immunodeficiency disease. Can treat adenosine deaminase deficiency ADA, Thalassemia and hunters disease.
2019 crisper based gene editing in human editing gene therapy will be conducted. To treat leber congenital amaurosis.
Advantages.
Treat rare and difficult diseases like cancer.
Better immune system.
Cheaper and permanent cure in the long run
Precision
Risks
Unknown effects in the long run
Increases genetic load in humans
ethical concerns and potential to start race for superhumans where the rich will benefit and poor will be left behind