Never miss a story

Get subscribed to our newsletter


×
In the sickle cell, defective hemoglobin leads to deformed, crescent-shaped blood cells that don't carry oxygen well. Pixabay

Scientists are seeing promising early results from the first studies =for painful, inherited blood disorders that plague millions worldwide, especially Black people. Doctors hope the one-time treatment, which involves permanently altering DNA in blood cells with a tool called CRISPR, may treat and possibly cure sickle cell disease and beta-thalassemia.

Partial results were presented Saturday at an American Society of Hematology conference and some were published by The New England Journal of Medicine. Doctors described 10 patients who were at least several months removed from their treatment. All no longer needed regular blood transfusions and were free from the pain that plagued their lives before.


Follow NewsGram on Twitter to stay updated about the World news.

Victoria Gray, the first patient in the sickle cell study, had long suffered bouts of severe pain that often sent her to the hospital. “I had aching pains, sharp pains, burning pains, you name it. That’s all I’ve known my entire life,” said Gray, 35, of Forest, Mississippi. “I was hurting everywhere my blood flowed.” Since her treatment a year ago, Gray has weaned herself from pain medications she depended on to manage her symptoms. “It’s something I prayed for my whole life,” she said. “I pray everyone has the same results I did.”


Victoria Gray, the first patient in the sickle cell study, had long suffered bouts of severe pain that often sent her to the hospital. Pixabay

Who’s affected

Sickle cell affects millions, mostly Black people. Beta thalassemia strikes about one in 100,000 people. The only cure now is a bone marrow transplant from a closely matched donor without the disease, like a sibling, which most people don’t have. Both diseases involve mutations in a gene for hemoglobin, the substance in red blood cells that carries oxygen throughout the body.

In the sickle cell, defective hemoglobin leads to deformed, crescent-shaped blood cells that don’t carry oxygen well. They can stick together and clog small vessels, causing pain, organ damage, and strokes. Those with beta-thalassemia don’t have enough normal hemoglobin and suffer anemia, fatigue, shortness of breath, and other symptoms. Severe cases require transfusions every two to five weeks.

The treatment studied attacks the problem at its genetic roots. In the womb, fetuses make a special type of hemoglobin. After birth, when babies breathe on their own, a gene is activated that instructs cells to switch and make an adult form of hemoglobin instead. Adult hemoglobin is what’s defective in people with one of these diseases. CRISPR editing aims to cut out the switching gene. “What we are doing is turning that switch back off and making the cells think they are back in utero, basically,” so they make fetal hemoglobin again, said one study leader, Dr. Haydar Frangoul of the Sarah Cannon Research Institute in Nashville.

Switching gene-targeted

The treatment involves removing stem cells from the patient’s blood, then using CRISPR in a lab to knock out the switching gene. Patients are given strong medicines to kill off their other, flawed blood-producing cells. Then they are given back their own lab-altered stem cells. Saturday’s results were those for the first 10 patients, seven with beta-thalassemia and three with sickle cell. The two studies in Europe and the United States are ongoing and will enroll 45 patients each.


Patients are given strong medicines to kill off their other, flawed blood-producing cells. Pixabay

Tests so far suggest the gene editing is working as desired with no unintended effects, Frangoul said. “The preliminary results are extremely encouraging,” he said. The study was sponsored by the therapy’s makers — CRISPR Therapeutics, with headquarters in Zug, Switzerland, and Massachusetts-based Vertex Pharmaceuticals. Some study leaders consult for the companies. Separately, Dr. David Williams of Harvard-affiliated Boston Children’s Hospital gave partial results from a study testing a novel type of gene therapy that also seeks to restore fetal hemoglobin production for those with sickle cell.

ALSO READ: Gene Tied To Balding May Also Raise Covid Risks For Men

Six patients including one as young as 7 were given the treatment, in which some of their blood stem cells were removed and altered in the lab to muffle the hemoglobin switching gene. None have had pain crises, five of the six no longer need transfusions and all have near-normal hemoglobin, he reported at the conference and in the medical journal.

Government grants paid for the work. Williams is named on a patent for the therapy, which Boston Children’s has licensed to Bluebird Bio Inc. of Cambridge, Massachusetts. The company provided the therapy for the study, which will enroll 10 people in all to establish safety. A larger study to test effectiveness is planned. Williams, who was not involved in Frangoul’s study, said it “validates this approach” of targeting the hemoglobin switching gene to tackle sickle cell. (VOA/JC)


Popular

Photo by Pixabay

Upcoming medical colleges in Uttar Pradesh will be named after saints and sages

The new medical colleges being opened in Uttar Pradesh will be named after saints and sages.

The state government has issued an order naming four district hospitals that are being converted into medical colleges.

These district hospitals are in Bijnor, Fatehpur, Chandauli, and Siddharth Nagar.

The Bijnor medical college has been named after Mahatma Vidur, a philosopher during the Mahabharata era and uncle of the Pandavas and Kauravas.

The Chandauli medical college has been named after Baba Keenaram, said to be the founder of the Aghori sect.

The Siddharth Nagar district hospital will be called Madhav Prasad Tripathi Medical College after the BJP politician from the region. Tripathi, popularly known as Madhav Babu, was also the first Uttar Pradesh BJP chief. He was elected MP from Domariyaganj in 1977, besides being two times Jan Sangh MLA and also a member of the UP legislative council.

The Fatehpur hospital has been named Amar Shaheed Jodha Singh Ataiya Thakur Dariyawn Singh Medical College, after the freedom fighter of 1857.

It is said that he was among the first to use Guerrilla warfare against the British, as taught by freedom fighter Tatya Tope.

Meanwhile, according to official sources, the medical college in Deoria will be named after Maharishi Devraha Baba and the medical college of Ghazipur in the name of Maharishi Vishwamitra.

The medical college of Mirzapur will be in the name of Maa Vindhyavasini, the medical college of Pratapgarh in the name of Dr. Sonelal Patel and the medical college of Etah will be named after Veerangana Avantibai Lodhi. (IANS/JB)

Keywords: Medical Colleges, Uttar Pradesh, Yogi Adityanath, India, Politics


Photo by Wikimedia Commons

Photo of Indian cricket team on the ground

Former Pakistan captain Inzamam-ul-Haq has picked India as the favourite to win the ongoing ICC Men's T20 World Cup in Oman and United Arab Emirates (UAE).

Inzamam feels that the Virat Kohli-led India have a greater chance of winning the trophy as the conditions in the Gulf nations are similar to the subcontinent, which makes India the most dangerous side in the event, according to Inzamam.

"In any tournament, it cannot be said for certain that a particular team will win' It's all about how much chance do they have of winning it. In my opinion, India have a greater chance than any other team of winning this tournament, especially in conditions like these. They have experienced T20 players as well," said Inzamam on his YouTube channel.

He said more than the Indian batters, the bowlers have a lot of experience of playing in the conditions. The Indian Premier League (IPL) was played recently in UAE and most of the Indian bowlers did well in that leg.

Inzy heaped praises on the Men in Blue for the confident manner in which they chased the target against Australia on a challenging track without needing Kohli's batting prowess.

"India played their warm-up fixture against Australia rather comfortably. On subcontinent pitches like these, India are the most dangerous T20 side in the world. Even today, if we see the 155 runs they chased down, they did not even need Virat Kohli to do so," he added.

Though he did not pick any favourite, Inzamam termed the India-Pakistan clash in the Super 12 on October 24 as the 'final before the final' and said the team winning it will go into the remaining matches high on morale,

"The match between India and Pakistan in the Super 12s is the final before the final. No match will be hyped as much as this one. Even in the 2017 Champions Trophy, India and Pakistan started and finished the tournament by facing each other, and both the matches felt like finals. The team winning that match will have their morale boosted and will also have 50 percent of pressure released from them," Inzamam added. (IANS/JB)

Keywords: India, Pakistan, Sports, ICC T20 World Cup, UAE.


Photo by Diana Akhmetianova on Unsplash

Skin problems like itchiness, dryness and flakiness can occur anytime if you're not moisturising your body enough.

Skin problems like itchiness, dryness and flakiness can occur anytime if you're not moisturising your body enough. It is commonly observed that while many people take their skincare routine seriously, a majority of them neglect to moisturise the body. It is important to keep in mind that timing matters a lot when it comes to applying moisturisers. Therefore, knowing the appropriate time to apply body lotion is essential.

Take a look at the ideal times to moisturise your body shared by Kimi Jain, Head of Retail, KIMRICA.

Morning
Moisturising the body in the morning sets your skin up to face countless irritants and environmental factors during the day. The skin is constantly exposed to harsh chemicals and pollutants when you're outside which is why using a protective and soothing moisturiser while going out is necessary. Kimirica's Five Elements Body Lotion comes with natural Aloe Vera extracts that act as a rich source of antioxidants and vitamins that helps protect your skin and provide a deep nourishing effect.

man in white crew neck t-shirt Moisturising the body in the morning sets your skin up to face countless irritants and environmental factors during the day. | Photo by The Creative Exchange on Unsplash

Keep reading... Show less