Skip to main content

Personalized gene therapy for ALS patient set to proceed for the second time ever.


Amyotrophic Lateral Sclerosis(ALS) is a progressive neurodegenerative disease that affects nerve cells (motor neurons) of the brain and the Spinal cord. Motor neurons connect the brain to the spinal cord and from the spinal cord to the muscles of the body. Through motor neurons, the brain controls the movement of our bodies. In a patient suffering from ALS, motor neurons degenerate. Then the brain lost the ability to control muscle movement, and the patient started to loses the ability to speak, eat, move and breathe.
There are two types of ALS: Familial and sporadic. Sporadic is the most common form of ALS which accounts for 90-95 % of the cases. It may affect anyone, anywhere. Familial ALS is inherited. In those families, there is a 50% chance of each offspring will inherit a gene mutation and may develop the disease.
Jaci Hermstad (center) and her parents. Harvest Moon Photography by Hilary
A young woman who has developed an aggressive, early-onset form of ALS which killed her twin sister is set to receive a cutting-edge form of personalized medicine, gene therapy is yet to be approved for use. It involves designing a molecule that binds to the mutated strands of the gene and prevents the production of mutated protein that causes diseases.
The woman will become the second ever to try the approach. The first use was on another neurodegenerative disease, Batten disease.




References: Tailored treatment for ALS poised to move ahead. NATURE. 30th May 2019.
                  doi: 10.1038/d41591-019-00013-w

Comments

Our top Stories

Normal human body temperature is not 37 degree celsius any more.

  It's not 37 degrees anymore.

Do you know what is the temperature of the human body? If your answer is 370C, you have missed the updated information. A scientist from Stanford has found that our body temperature has dropped by 0.6 0C since the civil war. Previously in the past, scientists have observed this change in our body temperature. But they could not believe the drop in temperature. So, they blame the thermometers for the discrepancy. But recent studies have confirmed that our body temperature has indeed dropped by 0.60C.
The reason behind the temperature dropped of our body The main reason is the reduction in the overall level of inflammation thanks to vaccines, antibiotics and quality water. When inflammation occurs, chemicals from the body's white blood cells are released into the blood or affected tissues to protect your body from foreign substances. This release of chemicals increases the blood flow to the area of injury or infection and result in warmth. Because of…

Type A blood converted to Universal donor, type O with bacterial enzymes.

Blood group A converted into group O by gut bacterial enzymes.

Blood transfusions are a daily routine procedure in hospitals. In a day, 16k liters of blood are transfused daily in the United States. Human being has four types of blood group- A, B, AB, O. Blood type A, B, and AB have their own antigens on their surface. Blood Antigens are basically a polysaccharide on the surface of red blood cells.  Type O does not have any antigens on their surface. The demand for blood all over the world is very high, especially during emergency surgeries and operations. Blood transfusion is only possible when the blood group of donor and recipient are matched. If they are not matched, the immune cells of the recipient will attack and destroy the RBCs of the donor. Blood group O does not have an antigen on their surface, thus making them transfusable to any recipient irrespective of the blood group of the recipient. Hence, Blood group 0 are called a universal donor. However, there is a shortage of …

Scientists just found out that the gut microbiota influences learning capacity.

The researchers found that manipulation of gut microbiota of mice shows a significant difference in learning in comparison with a mouse of the normal gut. Microbiota refers to a complex consortium of viruses, bacteria, fungi, and parasites. In mammals,

New Antibiotic discovered for the first time in 60 years in the nematode Photorhabdus.

A new antibiotic that selectively acts against Gram-negative bacteria have been discovered in the nematode, Photorhabdus. They named the antibiotic Darobactin.Darobactinis coded by a silent operon and has an unusual structure with two fused rings that are formed post-translationally. It is produced in very low quantity in laboratory conditions and is synthesized

Biodegradable burial pod turns your body into a tree.

Whenever a person dies, it is a burden for the family emotionally and financially. We spend thousands of dollars in funerals paying for the casket, flowers, and monuments. Environmentally, those caskets and monuments left behind an enormous carbon footprint.
Recently, many people have begun to embracethe idea of green burials. People are environmentally more aware and there is a shift from cremation and the use of wood, the synthetic cushioning and the metals generally used in traditional coffins to green burial. Green burial is a method of burying the body in a wooden coffin without a concrete burial vault. The body along with the wooden coffin is degraded easily by the bacteria in the soil. This type of burial allows the graveyard to return to nature. Nearly 54 percent of Americans are considering green burial and 74 percent of cemeteries are reporting an increased demand for green burial according to National Funeral Directors Association.
Italian designers, Raoul Bretzel and Anna Ci…