Question 1

How does CRISPR/Cas9 work as a genome editing tool?

Accepted Answer

CRISPR/Cas9 functions as molecular scissors that cut DNA at specific locations. Guide RNA sequences direct the Cas9 enzyme to target sites in the genome, allowing scientists to add, remove, or alter genetic material with precision. This system originated as a prokaryotic immune response and has been adapted for editing genetic information in research and therapeutic applications.

Question 2

What is the difference between a knockout and a knockin in genetic engineering?

Accepted Answer

A knockout removes a gene from an organism's genome, eliminating its function entirely. A knockin introduces a new gene into the genome through gene targeting, which relies on homologous recombination—genetic exchange between DNA molecules sharing similar sequences. Both techniques modify endogenous genes but achieve opposite outcomes: deletion versus insertion.

Question 3

How can gene therapy treat genetic diseases?

Accepted Answer

Gene therapy introduces a new gene into a patient's cells so the protein it encodes can be expressed and function properly. Scientists often use modified viruses to deliver correct copies of defective genes to diseased cells. This approach provides potential cures or treatments for serious genetic disorders like Severe Combined Immunodeficiency (SCID) that were previously untreatable.

Question 4

What are transgenic organisms and how are they created?

Accepted Answer

Transgenic organisms contain genes from another species inserted into their genome, creating recombinant DNA. Scientists combine DNA from different sources and introduce it into a host organism, which becomes a genetically modified organism (GMO). Examples include transgenic goats producing spider silk in their milk and plants modified for improved nutritional content and pest resistance.

Question 5

Why is human germline editing considered ethically problematic?

Accepted Answer

Germline editing modifies reproductive cells, so genetic changes are inherited by offspring and affect future populations. Most gene therapies modify somatic cells, affecting only the individual treated. The 2018 case of allegedly gene-edited babies sparked global concern about unintended health consequences, effects on future generations, and the broader implications for humanity.

Question 6

What practical applications does genetic engineering have beyond medicine?

Accepted Answer

Genetic engineering has transformed agriculture and industry. Plants are genetically modified to enhance nutritional content and pest resistance, with nearly 450 million acres of GM crops grown globally. Transgenic animals produce valuable proteins for industrial use. Bacteria were the first genetically engineered organisms, modified to produce synthetic insulin for diabetes treatment and other therapeutic proteins.

Question 7

Why is the universal genetic code important for genetic engineering?

Accepted Answer

The genetic code and DNA structure are universal across all life forms, enabling scientists to transfer genes between different organisms. This universality allows researchers to insert genes from one species into another and have them function properly. This principle underlies the creation of transgenic organisms and the success of genetic engineering across bacteria, plants, and animals.

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