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How this scientific breakthrough could help treat genetic diseases

In this undated photo, DNA genotyping and sequencing is pictured at the Cancer Genomics Research Laboratory in the US. Researchers from two companies recently said that they have found promising resul
In this undated photo, DNA genotyping and sequencing is pictured at the Cancer Genomics Research Laboratory in the US. Researchers from two companies recently said that they have found promising results to treat sickle disease, using DNA sequencing technology. (Photo: Unsplash)
AMMAN — Genetic diseases, like sickle cell anemia, have long been thought of as permanent. But a new technology with the ability to change genes themselves is opening up new doors and allowing doctors to treat diseases once thought incurable.اضافة اعلان

Researchers from a study conducted by Vertex Pharmaceuticals and CRISPR Therapeutics have used the CRISPR gene-editing technique, which utilizes the protein Cas9 to locate the desired portion of DNA before removing, adding, or modifying sections of DNA sequences in the genome. 

CRISPR (which stands for clustered regularly interspaced short palindromic repeats) is a group of DNA sequences found in genomes of prokaryotic organisms such as bacteria. These sequences play a crucial role in the defence system of bacteria and have since been modified as this new technology for gene editing.

Delivering the Cas-9 protein allows for the targeting of specific segments of genetic code and the ability to edit or cut DNA sequences at specific locations. 


Genes are made up of DNA. DNA, or deoxyribonucleic acid is a molecule that contains the biological information that makes every living thing unique. However, an abnormality in a single gene can cause lifelong illnesses such as Sick Cell Anemia and Cystic Fibrosis.

CRISPR-Cas9 has not only opened the door for possible cures to genetic diseases but also for the early prevention and treatment of these diseases as well.

CRISPR’s potential is currently being studied with a few diseases, including cancer, neurodegenerative disorders, and blood disorders, according to a statement released by the two companies. The genetic engineering technology has already made it to various clinical trials and has shown promising outcomes, the statement added.

Today, we have far more knowledge of specific genes involved in these diseases and how they work. Using this information, scientists can repair the damage done to the gene, insert new genes, or disable harmful ones.

The trials performed by Vertex Pharmaceuticals and CRISPR Therapeutics have found that sickle cell anemia, a genetic blood disorder, is one of many conditions that have responded well to gene editing treatment. Clinical trial patients treated with CRISPR no longer needed blood transfusions and showed significant improvement of sickle cell anemia symptoms.

Another blood disorder called Beta thalassemia has also shown improvement in trials using CRISPR techniques, according to the study. Beta thalassemia, an inherited blood disorder, occurs when the body cannot make hemoglobin, the part of red blood cells that carries oxygen, properly. It can result in anemia and other medical issues.

As this technology is relatively new, many diseases are still being examined in clinical trials. Researchers, in the statement, said that they are hopeful about this technique as it has already shown promise in many diseases early on.

The majority of these diseases have been thought to be life-threatening in the past. However, as scientists continue to make advancements in medicine, many illnesses may be prevented before the need for a cure.

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