Explain why enzyme replacement therapy cannot cure a genetic disease, and why gene therapy offers the only cure

What will be an ideal response?


The idea that some genetic disorders could be treated began in the mid-1960s, when a cell biologist, Christian DeDuve, proposed that symptoms of disorders caused by defective enzymes might be improved by providing the missing enzyme. He suggested that this procedure, called enzyme replacement therapy (ERT), could be used as a therapy for genetic disorders.

While ERT is successful in controlling the symptoms of genetic disease, the ultimate goal is to cure genetic diseases, and the most direct way to do this is by transferring a correct copy of a mutant gene into the body. This approach, called gene therapy, involves the delivery of a normal version of a mutant gene to cells in the body. Once in the cell, the expression of this gene will produce a functional protein that restores cellular function and results in a normal phenotype.

Biology & Microbiology

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Name one molecule that can readily diffuse across a lipid bilayer

What will be an ideal response?

Biology & Microbiology

Unlike phytoplankton, which are at the mercy of currents, zooplankton can move at will within a current

Indicate whether the statement is true or false

Biology & Microbiology

About 3 million years ago, the Isthmus of Panama (a narrow strip of land connecting North and South America) formed, dividing marine organisms into Pacific and Caribbean populations. Researchers have examined species of snapping shrimp on both sides of the isthmus. Based on the morphological species concept, there appeared to be seven pairs of species, with one species of each pair in the Pacific and the other in the Caribbean. The different species pairs live at somewhat different depths in the ocean. Using mitochondrial DNA sequences, the researchers estimated phylogenies and found that each of these species pairs, separated by the isthmus, were indeed each other's closest relatives. The researchers investigated mating in the lab and found that many species pairs were not very

interested in courting with each other, and any that did mate almost never produced fertile offspring. (N. Knowlton, L. A. Weigt, L. A. Solorzano, D. K. Mills, and E. Bermingham. 1993. Divergence in proteins, mitochondrial DNA, and reproductive incompatibility across the Isthmus of Panama. Science 260:1629-32.) Refer to the paragraph about the formation of the Isthmus of Panama. The sister populations on opposite sides of the isthmus are true species under which species concept? A. the phylogenetic species concept B. both the morphological species and phylogenetic species concepts C. the morphological species concept D. the biological species concept E. the morpholoogical species, biological species, and phylogenetic species concepts

Biology & Microbiology

Parasitic lampreys have

A) bony jaws. B) radial proportions. C) sucker-like mouths lined with teeth. D) a three-chambered heart. E) internal fertilization.

Biology & Microbiology