Stem Cell Blog

Употребата на матичните клетки од папочна врвца рапидно се зголемува. Пред 10 години крвта од папочна врвца можеше да лекува околу 40 состојби, но денес таа бројка е над 80. Со нетрпение очекуваме нови терапии за болести и нарушувања како што се дијабет, аутизам и мозочен удар, можете да бидете во тек со најновите случувања во регенеративната медицина на нашиот блог за матични клетки.



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The outcome of a recent clinical trial conducted by pharmaceutical company Vertex indicates huge promise for the development of a stem cell cure for type 1 diabetes.

The phase 1/2 study, whose results were presented at the American Diabetes Association 84th Scientific Sessions this month, found that the infusion of VX-880 – a new stem cell derived therapy – occasioned significant improvements in diabetic patients’ wellbeing. [1]

What is type 1 diabetes? 

Type 1 diabetes is an autoimmune condition that stops the pancreas producing insulin. Insulin is the hormone the body produces to move glucose (sugar) from the bloodstream into cells to make energy. Without insulin, glucose builds up in the bloodstream and leads to hyperglycemia. [2]

Complications from hyperglycemia can be severe, affecting major organs like the heart and kidneys. Glucose build up can also damage nerves, as well as the blood vessels in the eyes which can result in blindness. [3]

Those who have type 1 diabetes are required to take insulin everyday, either with meals or at regular intervals, a process which can drastically alter lifestyle habits as well as quality of life. [4]

There is currently no known cure for type 1 diabetes and its root causes are unknown, although many believe the condition to be the result of either genetics or environmental factors like exposure to viruses.

According to Diabetes UK, around 1 in 10 people have type 1 diabetes. [5]

What were the results of the trial?

12 patients volunteered for the trial, all with type 1 diabetes and average haemoglobin A1C levels of 7.8%, a level that verges on risks from diabetic complications.

A1C levels measure the amount of haemoglobin with attached glucose in the bloodstream. A higher percentage equates to a higher level of glucose. The average A1C level for a non-diabetic is below 5.7%. [6]

The 12 patients underwent an infusion of VX-880, a new therapy containing islet cells – cell clusters that produce insulin in the pancreas – derived from stem cells.

Following the infusion, all patients demonstrated engraftment of the islet cells. By day 90, all patients also demonstrated glucose-responsive insulin production. All patients’ A1C level also dropped below 7%.

Out of 12 patients who received a full dose of VX-880, seven no longer needed daily insulin injections. Two more patients required about 70% less insulin to manage their blood sugar levels.

These results are incredibly promising and the trial has since been expanded to include 37 patients as research into the effectiveness of VX-880 continues. [7]

What is VX-880 stem cell therapy and how does it work?

VX-880 therapy involves introducing fresh islet cells derived from stem cells into the patient’s body.

Because stem cells have the unique ability to differentiate into other specialised cells in the body, they can be reprogrammed to become a specific cell type that can then be used to replace or restore certain cells that may be missing through disease.

The stem cells used in the trial were allogeneic, meaning that they were sourced from a donor and required both a good match in addition to immunosuppressant drugs to prevent them from being rejected by the patients’ immune systems.

The goal is for these new islet cells to restore the ability of the patients’ pancreases to produce insulin, effectively curing the disease and drastically reducing the need for them to have to administer insulin exogenously. [8]

Cord Blood Banking and Stem Cells

Breakthroughs like this underscore the incredible potential stem cells have in treating severe conditions like type 1 diabetes.

Though not yet an approved treatment, clinical trials like this one show that stem cells are at the forefront of developing regenerative therapies for diseases that are currently incurable.

By banking your baby’s umbilical cord stem cells, you can ensure that they have them ready and waiting should they ever need to access one of these regenerative therapies in the future.

To find out more about saving stem cells for your baby, fill out the form below for a free Welcome Pack.

References

[1] (2024, June 21). Vertex Announces Positive Results From Ongoing Phase 1/2 Study of VX-880 for the Treatment of Type 1 Diabetes Presented at the American Diabetes Association 84th Scientific Sessions. Business Wire. https://www.businesswire.com/news/home/20240621506971/en/Vertex-Announces-Positive-Results-From-Ongoing-Phase-12-Study-of-VX-880-for-the-Treatment-of-Type-1-Diabetes-Presented-at-the-American-Diabetes-Association-84th-Scientific-Sessions

[2] Lucier J, Weinstock RS. Type 1 Diabetes. [Updated 2023 Mar 3]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK507713/

[3] (2024, March 27). Type 1 diabetes. Mayo Clinic. https://www.mayoclinic.org/diseases-conditions/type-1-diabetes/symptoms-causes/syc-20353011

[4] (2021, July 14). What is type 1 diabetes. NHS. https://www.nhs.uk/conditions/type-1-diabetes/about-type-1-diabetes/what-is-type-1-diabetes/

[5] Type 1 diabetes. Diabetes UK. https://www.diabetes.org.uk/diabetes-the-basics/types-of-diabetes/type-1

[6] The A1C Test & Diabetes. National Institute of Diabetes and Digestive and Kidney Diseases. https://www.niddk.nih.gov/health-information/diagnostic-tests/a1c-test/

[7] (2024, June 21). Vertex Announces Positive Results From Ongoing Phase 1/2 Study of VX-880 for the Treatment of Type 1 Diabetes Presented at the American Diabetes Association 84th Scientific Sessions. Business Wire. https://www.businesswire.com/news/home/20240621506971/en/Vertex-Announces-Positive-Results-From-Ongoing-Phase-12-Study-of-VX-880-for-the-Treatment-of-Type-1-Diabetes-Presented-at-the-American-Diabetes-Association-84th-Scientific-Sessions

[8] (2024, June 25). Stem Cell Therapy Could Be Breakthrough Against Type 1 Diabetes. U.S. News. https://www.usnews.com/news/health-news/articles/2024-06-25/stem-cell-therapy-could-be-breakthrough-against-type-1-diabetes#:~:text=June%2025%2C%202024%2C%20at%206%3A00%20a.m.&text=TUESDAY%2C%20June%2025%2C%202024%20(,early%20clinical%20trial%20results%20show.


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This year’s World Sickle Cell Day is on the 19th June, a day to recognise and raise awareness of the disease, the millions around the world who suffer from it, and ongoing research into how treatment is advancing.

With this year’s theme being ‘we are STRONGER TOGETHER’, we thought it would be a good opportunity to highlight how cord blood transplants between siblings have emerged as a viable treatment alternative for those suffering from the condition.

Making use of sibling cord blood necessitates having it in storage and so in celebration of World Sickle Cell day, we wanted to highlight how cord blood banking could provide a lifeline for those suffering from the disease, as well as how storing cord blood for your baby could help safeguard the health of your family.

What is sickle cell disease?

Sickle cell disease is the name for a group of inherited blood disorders that are passed down from parents to children.

Particularly common amongst those who have African or Caribbean heritage, sickle cell disease inhibits the haemoglobin in red blood cells from carrying oxygen.

This can lead to the red blood cells in those with the condition to become misshapen, inflexible and liable to sticking together and blocking blood flow, causing tremendous pain – known as sickle cell crises – in addition to an increased risk of stroke, lung problems, eye problems and infection.

The disease gets its name from the shape of the red blood cells affected by the condition: crescent or ‘sickle’ shaped rather than discoid. [1]

What treatments are available for sickle cell disease?

Currently, treatments for sickle cell disease focus primarily on the alleviation of symptoms.

While there have been significant steps forward in recent years, including the development of the Casgevy therapy which utilises genetically engineered bone marrow stem cells from the patients themselves in lieu of a donor transplant, medicines like antibiotics and painkillers remain the most prevalent way of combating sickle cell.

There is only one known cure for sickle cell disease: a stem cell or bone marrow transplant. In these treatments, healthy red blood cells are produced by the donated stem cells, replacing the ones that are affected by sickle cell. [2] 

However, difficulty in locating an unrelated donor match, in addition to the risks posed by graft-versus-host disease, hinders the ready availability of transplantation as a treatment option.

The benefits of cord blood banking for sickle cell disease

Within the last decade, sibling cord blood transplants have emerged as a viable alternative to bone marrow transplants as a treatment option for sickle cell disease.

A comprehensive study in 2017 following the success rates of sibling cord blood transplants over a period of 20 years found that of the 28 patients with sickle cell who received cord blood from a sibling, all but one are both alive and free from sickle cell disease. [3]

With a reduced risk of graft-versus-host disease, in addition to a 25% chance of a perfect match and a 50% chance of a partial match, using the stem cells from a sibling’s umbilical cord blood alleviates many of the current obstacles to obtaining a transplant to treat sickle cell disease.

Moreover, because of the way sickle cell is passed down between parents and children, if one child is born with sickle cell disease then there’s a 75% chance that a subsequent child will not have the disease, making a cord blood transplant between siblings possible. [4]

Should I store my baby’s cord blood?

Underlying the breakthroughs in treating sickle cell through a sibling cord blood transplant is one crucial detail: whether or not that sibling has cord blood samples in storage. There’s only one opportunity to save their cord blood: in the minutes after they’re born.

Without cord blood samples in storage, availability to stem cell transplant treatments for sickle cell, and other blood or inherited conditions, becomes more difficult as it’s harder to find a suitable match.

Ensuring that you save cord blood for every child maximises opportunities for treatment, particularly in instances where a sibling transplant could provide a cure for conditions requiring an HLA match, like sickle cell disease.

Additionally, with the emergence of the aforementioned Casgevy therapy and ongoing trials exploring the possibility for autologous stem cell treatments for sickle cell disease, saving your baby’s stem cells means that they have improved access to cutting edge therapies using their own cord blood samples. [5]

Storing cord blood for every child is the only way to ensure that they have improved access to the benefits of cord blood banking.

If you want to learn more about how storing cord blood for your baby could provide protection for their health and the health of their siblings, fill out the form below for a free Welcome Pack.

References

[1] (2024, April 22). What Is Sickle Cell Disease? National Heart, Lung and Blood Institute. https://www.nhlbi.nih.gov/health/sickle-cell-disease

[2] (2022, November 30). Overview: Sickle Cell Disease. NHS. https://www.nhs.uk/conditions/sickle-cell-disease/

[3] Rafii, Hanadi et al. “Family cord blood banking for sickle cell disease: a twenty-year experience in two dedicated public cord blood banks.” Haematologica vol. 102,6 (2017): 976-983. doi:10.3324/haematol.2016.163055

[4] Autosomal Recessive: Cystic Fibrosis, Sickle Cell Anemia, Tay-Sachs Disease. University of Rochester Medical Center. https://www.urmc.rochester.edu/encyclopedia/content.aspx?ContentID=P02142&ContentTypeID=90

[5] (2024, March 19). Clinical Study of BRL-101 in the Treatment of Sickle Cell Disease. ClinicalTrials.gov. https://clinicaltrials.gov/study/NCT06287099


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The results of a new study have found that patients who had undergone an organ transplant were able to wean themselves off anti-rejection drugs with the help of stem cells. [1]

What are anti-rejection drugs and why are they taken?

Tolerance is the holy grail of organ transplantation, the name for the body accepting the donated organ without the immune system believing it to be a foreign body and attacking it.

Currently, however, patients who undergo organ transplants are required to take immunosuppressant drugs in order to prevent what’s known as organ ‘rejection’. [2]

These drugs have to be taken for life and come with a range of unfavourable side effects, including increased exposure to infection and cancer, as well as risking damage to the donated organ.

How does the stem cell treatment work?

By transferring stem cells from the donor to the patient receiving the donated organ – in this case, a kidney – researchers in the trial hoped to induce ‘mixed chimerism’, a phenomenon whereby the organ recipient’s immune system becomes a hybrid composed of their own and their donor’s cells. [3]

Once this is achieved, the transplanted organ theoretically remains free from attack as the body no longer considers it to be ‘foreign’.

We covered a similar instance of stem cells being used to reprogramme the immune system in our blog about Aditi Shankar, who successfully underwent pioneering treatment to aid with a donated kidney last year. You can read more about Aditi’s story here.

What did the study find?

What the study found, the results of which were presented at the American Transplant Congress in Philadelphia, was that in 16 out of 19 cases, organ donor recipients who received stem cells from their HLA matched sibling no longer had to continue taking the immunosuppressant drugs after two years.

Additionally, researchers also noted improved quality of life outcomes for those who successfully underwent the treatment. [4]

What could this mean for future organ donor recipients?

Researchers in the trial are hoping that their findings can eventually result in future organ transplant recipients foregoing the need to take anti-rejection drugs. Not only are these drugs harmful but the damage they cause to the donated organ often leads to failure, resulting in a backlog of transplants. [5]

With the new treatment, any new organ transplants should theoretically be able to last a lifetime, expanding the pool of available organs in the process.

More research is needed to discover whether this treatment is viable for all types of organ transplant, beyond kidneys in this case, in addition to identifying whether the treatment is viable in cases where donated organs have been sourced through donors other than siblings.

Either way, this research is a huge step forward in the long held ambition of achieving tolerance for all organ transplant outcomes.

In this treatment researchers took advantage of the good chances of stem cell matches between siblings. The chance of a perfect stem cell match between full siblings is 1 in 4, with a 50% chance of a partial match. [6]

By saving stem cells for your baby you could not only safeguard their health, but also the health of their siblings.

If you want to know more about how saving stem cells could help protect your baby’s health, fill out your details below for a free Welcome Pack.

References

[1] National Library of Medicine (2024, June 7). Cellular Immunotherapy in Recipients of HLA-matched, Living Donor Kidney Transplants. Clinical Trials.gov. https://www.clinicaltrials.gov/study/NCT03363945?cond=Kidney%20Transplantation&term=NCT03363945&distance=50&rank=1

[2] NHS – Blood and Transplant (n.d.). Rejection of a transplanted kidney. Organ Transplantation. Retrieved June 11, 2024, from https://www.nhsbt.nhs.uk/organ-transplantation/kidney/benefits-and-risks-of-a-kidney-transplant/risks-of-a-kidney-transplant/rejection-of-a-transplanted-kidney/

[3] Sachs, David H et al. “Induction of tolerance through mixed chimerism.” Cold Spring Harbor perspectives in medicine vol. 4,1 a015529. 1 Jan. 2014, doi:10.1101/cshperspect.a015529

[4] Syal, A., M.D., & Herzberg, J. (2024, June 4). Adding stem cells to a kidney transplant could get patients off anti-rejection drugs, trial finds. NBC News. Retrieved June 11, 2024, https://www.nbcnews.com/health/health-news/adding-stem-cells-kidney-transplant-get-patients-anti-rejection-drugs-rcna155486

[5] Ruiz, Richard, and Allan D. Kirk. “Long-Term Toxicity of Immunosuppressive Therapy.” Transplantation of the Liver (2015): 1354–1363. doi:10.1016/B978-1-4557-0268-8.00097-X

[6] (2023, January 10). Donating your stem cells to a relative. Anthony Nolan. https://www.anthonynolan.org/patients-and-families/support-parents-family-and-friends/donating-your-stem-cells-a-relative


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Researchers in Shanghai have claimed to have achieved the world’s first successful cure for type 2 diabetes using stem cell-derived islet transplantation.

Publishing their findings in the journal Cell Discovery, the team of scientists believe that this new therapy offers hope for the estimated 420 million people living with type 2 diabetes around the world. [1]

What is type 2 diabetes?

Type 2 diabetes is a metabolic disorder that arises as a result of the body either being unable to produce insulin or being ineffective at using the insulin it produces in order to regulate blood sugar levels. [2]

Those with type 2 diabetes risk developing hyperglycemia, otherwise known as high blood glucose levels, as a result of being unable to metabolise glucose. Hyperglycemia can cause complications over time, including damage to organs.

Current management strategies primarily involve glycemic control through medications including insulin, as well as strict diet management to monitor blood sugar levels, leaving a substantial unmet need for a definitive cure. [3]

How did researchers develop the cure?

Scientists undertook a study centred around a 59-year-old male patient with a 25-year history of type 2 diabetes. Following a kidney transplant in 2017, the patient experienced a decline in pancreatic islet function, necessitating daily multi-dose insulin injections.

To address this challenge, the research team led by Dr. Yin Hao turned to a pioneering stem cell treatment. Utilising the patient’s own endoderm stem cells (EnSCs), they were able to differentiate these stem cells into functional pancreatic islet cells – cells in the pancreas that secrete hormones, including insulin and glucagon, that help regulate blood sugar levels. [4]

Once differentiated, these so-called ‘E-islet cells’ could then be implanted into the patient. [5]

What were the results of the transplant?

In July 2021, the patient underwent the autologous islet cell transplantation. Notably, the patient achieved insulin independence within a mere 11 weeks post-transplantation. Oral medication for diabetes management was gradually reduced and ultimately discontinued a year later.

Follow-up examinations conducted over a prolonged period revealed restored pancreatic function, with the patient no longer requiring exogenous insulin or oral medications.

Additionally, normal kidney function was maintained, suggesting a potential long-term cure for both type 2 diabetes and the underlying complications associated with the initial kidney transplant. [6]

What are the next steps in treating diabetes with stem cells?

This groundbreaking achievement signifies a paradigm shift in the treatment of type 2 diabetes.

The successful application of stem cell therapy offers a path towards a potential cure, paving the way for further research into the large-scale implementation and long-term efficacy of this novel approach.

While further clinical trials are warranted to validate these findings, this case study undoubtedly sparks hope for a future free from the burden of diabetes.

If you want to learn more about saving stem cells for your baby, download your free Welcome Pack by filling out the form below.

References

[1] WHO (n.d.). Diabetes. World Health Organization. https://www.who.int/health-topics/diabetes#tab=tab_1

[2] Watts, M. (2023, December 14). Type 2 Diabetes. Diabetes.co.uk. https://www.diabetes.co.uk/type2-diabetes.html

[3] Chong K, Chang JK-J, Chuang L-M.Recent advances in the treatment of type 2 diabetes mellitus using new drug therapies. Kaohsiung J Med Sci. 2024;40(3):212–20. https://doi.org/10.1002/kjm2.12800220

[4] Wu, J., Li, T., Guo, M. et al. Treating a type 2 diabetic patient with impaired pancreatic islet function by personalized endoderm stem cell-derived islet tissue. Cell Discov 10, 45 (2024). https://doi.org/10.1038/s41421-024-00662-3

[5] Da Silva Xavier, Gabriela. “The Cells of the Islets of Langerhans.” Journal of clinical medicine vol. 7,3 54. 12 Mar. 2018, doi:10.3390/jcm7030054

[6] Wu, J., Li, T., Guo, M. et al. Treating a type 2 diabetic patient with impaired pancreatic islet function by personalized endoderm stem cell-derived islet tissue. Cell Discov 10, 45 (2024). https://doi.org/10.1038/s41421-024-00662-3


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In a study first published in April’s edition of Advanced Functional Materials, researchers found that stem cells boost natural repair following cardiac arrest.

The most common consequence of cardiac arrest is brain injury. Decreased blood flow and oxygen to the brain can result in damage to specific areas such as the temporal lobe, which is responsible for memories. [1]

According to the British Heart Foundation, there are around 30,000 out of hospital cardiac arrests in the UK per year, with survival rates of only 1 in 10.

While there are currently steps towards improving these survival rates – mainly public awareness and educational campaigns focused on immediate response [2] – better survival rates potentially means more patients suffering from brain injury, which can range in severity.

What researchers at the University of Maryland School of Medicine in the U.S. have found is that neural stem cells can help with repairing post-cardiac arrest brain damage when their carbohydrate structure is manipulated. [3]

In an animal study using rats, scientists applied sugar molecules to the neural stem cells in a process called glycoengineering.

It’s thought that the application of these sugar molecules provide the neural stem cells with a better chance of retention and integration within the harsh microenvironment of the brain.

Researchers examined the efficacy of the ‘sugar-coated’ neural stem cells that had been pretreated with TProp (the name of the modified sugar molecule applied) with naive human neural stem cells.

They found through subsequent testing that the stem cells that had been pretreated with TProp improved brain function substantially, along with reducing anxiety and depression-related behaviours.

The ability for synapses to modify the strength of their connections (otherwise known as synapse plasticity) also improved, with the TProp group also demonstrating a reduction in neuroinflammation in the central nervous system.

Overall, the findings from the University of Maryland are promising as they indicate that these glycoengineered stem cells could help regenerate connections between synapses in the brain that have been affected by cardiac arrest related injury in humans.

The next steps will involve tests on larger animals before hopefully moving to a clinical study.

If you want to know more about how you can save your baby’s powerful stem cells, fill out the form below for your free welcome pack.

References

[1] (2023, July 7). Can a heart attack cause brain damage? Medical News Today. https://www.medicalnewstoday.com/articles/heart-attack-brain-damage#effects

[2] Horriar, Lina et al. “Improving survival after cardiac arrest in Europe: The synergetic effect of rescue chain strategies.” Resuscitation plus vol. 17 100533. 21 Dec. 2023, doi:10.1016/j.resplu.2023.100533

[3] (2024, May 9). Stem Cell Therapy Boosts Natural Repair After Cardiac Arrest. University of Maryland Baltimore. https://www.umaryland.edu/news/archived-news/may-2024/stem-cell-therapy-boosts-natural-repair-after-cardiac-arrest.php


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Researchers at Vienna University of Technology have taken an important step in making lab-grown cartilage a possibility by utilising a new technique involving 3D printing and stem cells. [1]

The process involves a high-resolution 3D printing process to create small, football-shaped spheres that act like porous scaffolds within which differentiated cartilage stem cells can sit.

These spheroid scaffolds can then be molded into various shapes in order to fit like puzzle pieces into seamless tissue structures.

One of the main challenges in attempting to form artificial cartilage using stem cells thus far has been the inability for scientists to leverage much control over the shape of the resulting tissue.

The key advantage of the 3D printed spheroid, cage-like structures, which are around a third of a millimetre in diameter, is that they’ve enabled the researchers in Austria to form combinable, compact building blocks from which to grow cartilage tissue.

Importantly, the team at TU Wien also showed that when combined, neighbouring spheroids actually grow together, with the cells from one spheroid migrating to another, connecting in a closed, continuous structure. [2]

The 3D printed plastic scaffolds provide mechanical stability to the tissue as it continues to grow, up until the point at which they are no longer needed. The spheroids then degrade, leaving behind cartilage tissue shaped in the way desired.

A huge breakthrough for facilitating the regenerative potential promised by stem cells  – particularly mesenchymal stem cells, which have the ability to differentiate into a range of specialised cells [3] – this new technique could be used in growing other tissues beyond cartilage into shapes required for repair at the cellular level.

For the time being, however, the researchers’ next aim is to attempt to use their 3D printed spheroids in the formation of tailormade pieces of cartilage tissue that can then be inserted into damaged areas of the body following injury.

If you’re interested in learning more about how saving stem cells for your baby could give them access to future regenerative treatments, download our free welcome pack below.

References

[1] Vienna University of Technology. “Artificial cartilage with the help of 3D printing.” ScienceDaily. ScienceDaily, 12 February 2024. <www.sciencedaily.com/releases/2024/02/240212133139.htm>

[2] Oliver Kopinski-Grünwald, Olivier Guillaume, Tamara Ferner, Barbara Schädl, Aleksandr Ovsianikov. Scaffolded spheroids as building blocks for bottom-up cartilage tissue engineering show enhanced bioassembly dynamics. Acta Biomaterialia, 2024; 174: 163 DOI: 10.1016/j.actbio.2023.12.001

[3] Vasanthan, Jayavardini et al. “Role of Human Mesenchymal Stem Cells in Regenerative Therapy.” Cells vol. 10,1 54. 31 Dec. 2020, doi:10.3390/cells10010054


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A new study conducted by researchers at the Mayo Clinic has found that stem cell treatment for patients with advanced heart failure offers an improved quality of life.

As many as 100,000 people are admitted to hospital in the UK every year due to heart attacks [1]. Heart attacks can lead to heart failure, where damage to cardiac muscles makes it harder for blood to be pumped around the body.


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Medical regulators in the UK have approved a gene-editing treatment involving bone marrow stem cells designed to cure two blood diseases, including sickle cell disease, in what is a world first.

The therapy, called Casgevy, has been given the green light by the Medicines and Healthcare products Regulatory Agency (MHRA) to treat sickle cell disease and beta thalassemia, two painful blood conditions. [1]