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Is DNA editing a cure-all?

Is DNA editing a cure-all?

Genetic disorders account for the vast majority of health disparities in the US. Most of the developed countries have been successful enough in controlling nutritional and infectious diseases in the population. However, genetic disorders continue to have a great impact on the general health and quality of life of individuals across the world. The science of genetics has seen enormous growth in terms of research which lets us unveil the secrets of the human genome (the whole set of genes in an individual). Today we have good evidence to support the fact that nearly all diseases have a genetic component in them. Genetic disorders refer to aberrations in DNA, which is the fundamental unit of the gene in a human being. These disorders often pose a challenge for doctors to manage. 1

What are scientists looking at?2

With the materialization of new and sophisticated genome techniques, it is now technically feasible to update genes to control a human from maturing a genetic disease. A new gene-modification technology called CRISPR-Cas-9 (clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9) was developed by the scientists. CRISPR can slash a particular DNA template by updating it with an RNA from the target sequence. The DNA can now be modified by adding, deleting, or replacing entities within the target DNA sequence.

This technology outstands the formerly used gene-modification methods in many ways. It does not require sketching or blueprinting a custom DNA cutting enzyme for each sequence. An RNA guide is prepared that is easier to incorporate. To gain access to previously isolated genes to delete diseases, scientists are looking at DNA modifiers that include the bacterial defense system CRISPR. Pioneering genetic coding technologies is crucial to optimizing CRISPR capability, which indeed would set an excellent standard in approaching challenges posed by genetic diseases.

What are the diseases that could be cured by this technology?3

  In April 2020, a study conducted in the West China Hospital among patients with lung cancer found that the gene-editing approach was feasible and safe to cure certain types of lung cancer. However, this approach was later found to have many limitations.

Another attempt by scientists where the patient’s stem cells were harvested from bone marrow and later used with CRISPR technology in vitro to make them produce fetal hemoglobin was a safe way to delete certain blood disorders in newborns.

There have been reports in medicine about the utilization of this technology to cure blindness in humans. Other diseases like HIV, muscular dystrophy, cystic fibrosis, and Huntington’s disease could also be cured by editing DNA.

How safe is gene editing?

Although the technology has gained greater acceptance among the public, trying out these techniques in human beings is of greater ethical concern as gene editing has the potential to cause undesirable changes in the individual as well.

While scientists have been victorious in a few cases, several difficulties stand in the way of trouble-free use of the approach. Technical challenges include proper delivery of the gene into the intended cells followed by accurate integration into the genome and immune reactions to the protein or the vector used. Owing to these reasons, gene editing is not yet a routine practice in medicine. Nevertheless, gene editing has a lot of uses at multiple levels. It can be extended in addressing and fighting against Diseases, Improving Lifespan, Enhancing Food Production and Its efficiency, and designing Pest-resistant Crops.

 

References

  1. https://www.genome.gov/For-Patients-and-Families/Genetic-Disorders
  2. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4191047/?_ga=2.232479071.1375611481.1637086682-1144799815.1636737596
  3. https://www.labiotech.eu/best-biotech/crispr-technology-cure-disease/