- Hunter’s syndrome is a rare genetic disorder that occurs as a result of a mutation in a gene that produces an enzyme that helps in breakdown of certain sugars.
- The enzyme activates parts in the cells in order to breakdown sugars.
- However, in the absence of the enzyme, these sugars build up in tissues causing damage to organs such as the heart, lungs, brain etc.
First in-vivo Gene Editing
- In a bid to treat Hunter’s syndrome, scientists conducted gene editing inside the human body (in-vivo) for the first time ever using a gene editing technique called zinc finger nucleases (ZNF).
- Under the zinc finger nucleases (ZNF) technique, the corrective gene was inserted in the DNA of the patient through Intravenous therapy (IV).
- While the in-vivo gene editing tool was found to be safe, its therapeutic efficacy could not be completely established.
- As per the Preliminary results, patients now have a corrective gene at very low levels, which may not be enough to make the therapy a success. However, it still represents a scientific milestone toward one day doctoring DNA to treat many diseases caused by faulty genes.
- The therapeutic efficacy will be established once the patient’s body will start making the missing enzyme that breaks down the sugars on its own.
What is Gene Editing?
- Gene editing is a form of gene therapy that is used to rewrite DNA.
- DNA is a biological code that guides the building of an organism.
- If there is a problem in this information storehouse, generally called a mutation, the organism may not be able to manufacture that protein and hence that organ in the body.
- Thus, a mutation could lead to absence of an organ or presence of a defective organ in the body.
- Gene Editing is the process of disabling faulty genes, correct the harmful mutation, insert a missing gene or change the activity of specific genes in living organisms.
Applications of gene editing
- Treating genetic disorders like cystic fibrosis, sickle cell anaemia and muscular dystrophy.
- Gene editing is widely used to modify immune cells to fight cancer, increase resistance in HIV cases.
- It can be used correct defective genes in human embryos and prevent inheritance of genetic disorders
- Gene editing techniques are faster, cheaper and more precise than conventional techniques in genetic modification of crops.
- Further gene editing techniques are extremely helpful in gene therapies including building of new diagnostic tools, drugs, organ transplantations etc.
Section : Science & Tech