Tech News – Cure for incurable diseases

A.D. In 2020, Jennifer Duud, a professor at Berkeley University in California, and Emmanuel Carpentier, co-founder and managing director of the Max Planck Department for Pathogenic Science in Berlin, developed CRISPR (Regularly False Pulmonromic Frequency) Technology.

CRISPR-Cas9 technology

CRISPR-Cas9 technology is a simple but extremely powerful tool for debugging genomes. It allows researchers to modify DNA sequences and modify the function of genes. CRISPRs are specialized regions of DNA that contain the enzyme Cas9, which acts as a pair of molecular scissors and can break down DNA.

CRISPR technology is based on the natural defense against bacteria and germs (single-celled microorganisms). When the bacterial immune system is compromised, the CRISPR-Cas system produces a second enzyme that triggers the enzyme, which helps to cut and destroy the invading DNA.


A.D. In 2011, Emmanuel Carpenter discovered that CRISPR-Cas could be used to disguise viruses in bacteria, and then collaborated with Jennifer Dudna to successfully reproduce the bacterium in a test tube. They also rearranged the genetic scissors by simplifying the molecular components and making sure that they can control the cutting of any DNA molecule at a predetermined location. This would allow scientists to rewrite the life code from which DNA was cut. When CRISPR technology is used in more complex organisms, it allows it to modify genes or “genetic modification”.

Numerous applications

CRISPR-Cas9 has become popular in recent years and has paved the way for many applications in basic science, medicine and agriculture. It has been used to create probiotic cultures in the food and agricultural industries, to vaccinate industrial cultures (such as yogurt) against viruses, and to improve production, drought tolerance, and crop nutrition. CRISPR promises to increase barrenness in the disease vector, female Anopheles Gambian mosquitoes, to control the spread of invaders and to eliminate invasive species and to create gene drives to change pesticides and pesticides.

But the most exciting and promising use of CRISPR is to modify the human genome to correct genetic defects, and thus not only cure many diseases but also prevent them. A.D. In 2013, the technology was first published in laboratory and animal studies to correct genetic defects in human diseases such as cystic fibrosis, cataracts, and fungal anemia. ). Other promising applications are hypertrophic cardiomyopathy (heart muscle obesity), Huntington’s disease (damage to nerve cells in the brain), and some mutations associated with breast and cervical cancer. Scientists have even shown that CRISPR can fight HIV infections by infecting HIV-infected T cells.

Incurable treatment

In June 2021, scientists in the New England Journal of Medicine published the first results of a clinical trial that could be safely and efficiently distributed in the human body to treat genetic diseases. In this small experiment, six people with an abnormal and fatal hereditary condition called transitine amyloidos (an abnormal accumulation of amyloid proteins in organs and tissues) received one treatment with a protein-targeted gene-specific treatment. Primarily in the liver. All patients showed a significant decrease in the TTR protein level associated with the disease by up to 96%. If harmful protein production is stopped, the symptoms will stop growing and may even be reversed in some cases. Until now, there has been little that doctors can do to cure this painful and incurable disease.

However, although CRISPR technology is revolutionary, it is simple and safe. The mechanism requires that DNA fragment enzyme, Cas9, and RNA regulation be directed to the correct location in the human genome. Because they supply blood, RNA and C9 molecules containing the RNA molecules are captured in nanoparticles called lipids, which are taken by the liver and protected from damage. The instruction then sends R9 to Cas9 to stop the production of TTR protein.

Many incurable diseases

Although most other CRISPR studies have been performed on animals, the direct delivery method developed by Intelia Therapists and Regeneron Pharmacists is promising for human treatment. Vascular transplantation may not require difficult and dangerous bone marrow transplantation to be used in future genetic modifications. To date, most genetic therapies involve the removal of cells from the body, the mutation of genes, and the replacement of cells.

In Cambridge, Massachusetts, the editorial drug CRISPR-Cas9 has been tested in people with hereditary disease (birth defects). In this case, the virus must be injected directly into the eye, where genetic therapy is performed.

Other studies around the world have focused on cancer, beta-thalassemia, muscle dystrophy, and even covide-19. Scientists at Stanford University have developed a method of using CRISPR / Cas13a to cut and destroy the genetic material of the CV-19 virus to prevent human lung cancer. This approach reduces the virus load by 90%. Scientists at the Georgia Institute of Technology have used a similar technique in animals to kill the virus before it enters the cells.

Hope for the future

Techniques for delivering CRISPR-Cas9 to different parts of the body are rapidly evolving and we are looking forward to a more extensive genome editing in the future. CRISPR, in fact, seems to have the potential to cure many incurable genetic diseases in the future through human DNA editing. This immunization will definitely change the course of treatment and introduce a new era of medicine.

Professor Louis CH Fourie Technology Strategy

The views expressed herein are not necessarily IOL or topic sites.

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