Health
How Science Is Solving Sickle Cell Disease
Scientists have used a DNA cutting technique to remove a gene responsible for sickle-cell disease (SCD) – a life-threatening blood genetic disorder.
According to the information scooped from irishtimes.com, the scientific breakthrough using Crispr (clustered regularly interspaced short palindromic repeats) genetic editing technology by a team including an Irish researcher has been outlined in findings reported in the journal Science.
“Crispr-Cas9 is a molecular scissors that we can use to cut and remove disease-causing mutations in our DNA,” explains Dr Ciaran Lee, a postdoctoral student at APC Microbiome Ireland research institute at University College Cork who took part in the research.
Dr Lee – a Marie Curie research fellow – did the experimental work at Rice University in Houston, Texas, and his data analysis was carried out on his return to Ireland. Leading researchers from Rice University, Stanford University and Aarhus University were also involved.
The life expectancy for people with SCD is between 42 and 47, and it affects in the region of 500 people in Ireland. It is most common among people of sub-Saharan African descent, while the numbers affected in Ireland have been growing over the past two decades.
Cas9 is a gene-cutting enzyme that uses a family of DNA sequences commonly known as Crispr, and then remove specific bits of DNA that scientists believe are linked with disease.
Haemoglobin defect
SCD is caused by a defect in adult haemoglobin, a protein in the red blood cells that carry oxygen around the body and give the blood its red colour. The defect causes red blood cells to become shaped like sickles or crescent moons. These abnormal cells can slow or block blood flow and cause pain, swelling, infections and vision problems.
In prior work scientists activated a type of haemoglobin present in the foetus, to compensate for defective adult haemoglobin. This was done, Lee says, by targeting regions of DNA called repressors.
“This approach is currently being tested in clinical trials with the early data showing great promise that Crispr-Cas9 gene therapy can result in a long-term cure,” Lee says.
The problem with this strategy, he explains, is that it does not remove the SCD causing genetic mutation from our cells. “Our study takes a different approach, using Crispr-Cas9 to target the disease mutation and replace it with the correct sequence.”
“While we and others have shown that this is possible in cells in a dish, this current study shows that when done at clinical scale this approach is efficient, safe and leads to correction of long-lived stem cells,” Lee says.
The study opens the door to similar gene-cutting strategies being adopted as a way to cure a range of genetic diseases. “These are all essential for developing an effective cure for sickle cell anaemia and other genetic diseases,” he points out.
The success of this research in a large-scale clinical trial on mice opens the door for trials in humans with SCD to begin. A US company specialising in use of gene-editing techniques to tackle disease is set to begin such trials later in 2021 or early in 2022.
A member representing Ibarapa East/Ido federal constituency of Oyo State at the House of Representatives, Engr. Aderemi Oseni, has announced a plan to launch a “Mobile Clinic” programme aimed at providing essential healthcare services directly to rural communities across the constituency.
Scheduled to begin in the first quarter of 2025, this initiative underscores Oseni’s commitment to expanding healthcare access, particularly for underserved populations.
Speaking on Saturday at the 10th-anniversary celebration of God’s Knot Hospital in Ologuneru, Ibadan, the lawmaker, who also chairs the House Committee on the Federal Roads Maintenance Agency (FERMA), highlighted the programme’s primary objective: bringing healthcare closer to the people.
“Every community deserves attention; healthcare should never be bound by geographical barriers,” Oseni stated.
He explained that the mobile clinics would deliver essential health check-ups, screenings, and preventive care in remote areas, allowing residents to access quality healthcare without traveling long distances.
As part of his broader healthcare agenda, Oseni informed, through a statement by his media aide, Idowu Ayodele, that he has already facilitated the construction of two fully equipped maternity centers within his constituency, both slated for commissioning before December 2024. He also disclosed plans to establish two modern general hospitals in 2025, further strengthening his commitment to building a healthier community.
The lawmaker expressed interest in collaborating with God’s Knot Hospital on the mobile clinic project, calling for partnerships to support sustainable healthcare initiatives that enhance the quality of life for vulnerable populations in the constituency.
“I resonate deeply with the mission of God’s Knot Hospital,” Oseni shared, adding that such partnerships would significantly improve healthcare in Oyo State”.
The anniversary event attracted distinguished guests, including former Ondo State Governor Dr. Olusegun Mimiko, whom Oseni commended for his contributions to healthcare, particularly through the Mother and Child Hospitals that transformed maternal and child healthcare in Ondo State.
Oseni urged philanthropists, government bodies, and international organizations to support healthcare projects like the upcoming mobile clinics, emphasising that the improvement of healthcare services is a shared responsibility.
The National Agency for Food and Drug Administration and Control (NAFDAC) has ordered the immediate closure of the Gomboru drug market in Maiduguri following severe flooding that submerged the area and contaminated the drugs sold within the market.
In a statement posted on its official X handle, NAFDAC disclosed that the decision was made after reports surfaced that contaminated drugs, which had been washed and sun-dried for resale, were being sold at the market.
“After the recent flooding in Maiduguri that submerged the Gomboru drug market where contaminated drugs were washed and sun-dried for resale, the @DGatNAFDAC ordered the immediate closure of the market to conduct a thorough shop-to-shop removal of these compromised drugs,” the agency wrote.
The flooding, which ravaged Maiduguri, was triggered by the rupture of the Alau dam on the Ngadda River, located 20 kilometers (12 miles) south of the city.
The floodwaters claimed at least 30 lives and displaced 400,000 residents, leaving large parts of the city and surrounding areas in devastation.
NAFDAC’s intervention is aimed at safeguarding public health by preventing the sale and consumption of contaminated drugs.
The agency has mobilized teams to inspect the market and ensure the removal of all compromised pharmaceutical products.
Engr. Aderemi Oseni, Chairman of the House Committee on Federal Road Maintenance Agency (FERMA) and the lawmaker representing Ibarapa East/Ido Federal Constituency of Oyo State has expressed deep concern following the recent outbreak of cholera cases in the state.
Cholera, a highly contagious bacterial infection transmitted through contaminated water and food, poses significant health risks, particularly in densely populated areas with inadequate sanitation.
Oseni urged residents of Oyo State, especially those in his constituency, to prioritise hygiene practices and strictly adhere to recommended safety measures, emphasising the critical importance of proactive measures to halt the disease’s spread.
In a statement issued on Monday by his media aide, Idowu Ayodele, in Ibadan, the state capital, the lawmaker underscored the necessity of maintaining clean water sources and practicing proper sanitation methods.
“Cholera outbreaks can be significantly reduced if we all take responsibility for our hygiene habits and ensure our surroundings are clean,” he stated.
The APC chieftain also stressed the urgency of seeking prompt medical attention at the first signs of symptoms such as diarrhea and vomiting, which are characteristic of cholera.
“Early detection and treatment are crucial in combating cholera and preventing its spread. Access to safe drinking water and adequate sanitation are fundamental rights that must be upheld, especially during health crises like this,” he asserted.
Oseni further called for unity and collective action in combating the cholera outbreak.
He expressed confidence that through concerted efforts and vigilant adherence to preventive measures, communities can contain and ultimately overcome this challenging public health issue.
“By working together, maintaining vigilance, and prioritising hygiene practices, we can effectively mitigate the impact of cholera on our communities,” the lawmaker concluded.
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