Question

If CRISPR can cure rare genetic diseases, why shouldn't scientists get to the point where this can be done safely and accurately. Basic needs here would be to define: "What is SAFE when it comes to genetic engineering of humans?" What do we need to see that it is safe.

Answer 1

Genetic engineering involves direct manipulation of an organism's genetic makeup using biotechnology. It allows so by removing or inserting specific DNA of interest into the genome of an organism. The invention of CRISPR/Cas 9 technology for genetic engineering has opened to immense possibilities in the development of biotechnology products and treatment of diseases. CRISPR is a family of short palindromic DNA sequences that are found in the genome of some prokaryotes. In prokaryotes, these sequences present a defence mechanism against pathogens for protection from infections. Cas 9 is an enzyme that can use CRISPR sequences to identify complementary DNA sequences and cleave them up.

CRISPR based technologies were recently used for the production of genetically modified crops with increased yield and resistance to pathogens. Theoretically, this technology can be used to treat deadly genetic disorders and viral infections including HIV. Some scientists have have already used it to treat genetic disorder including sickle cell anemia.

The concerns regarding gene editing using CRISPR comes when it is used to do germline modifications. Any modification in the germline would mean that these changes would be incorporated permanently into the genome of an organism and it would appear in all future generations. Suppose you are to knockout a particular gene in hope of eradicating a deadly disease in an embryo, you cannot readily predict what all other complications that baby would have. Most of the proteins in our body performs more than one function and can be involved in more than one regulatory or developmental pathways. For instance, our scientific community is still actively involved in elucidating functions of single proteins and every other discovery links a protein to multiple pathways. So, modifying a gene on the basis of it's critical involvement in a disease won't predict the outlying complications.

Also, ethical concerns surround this technology implying that it is unnantural and amounts to playing GOD. Many countries prohibit the use of germline gene therapy in humans for ethial reasons and possible risks to future generations.