Can thalassemia be cured by gene therapy?

Can thalassemia be cured by gene therapy?

Depending upon whether the genetic defects or deletion lies in transmission of α or β globin chain gene, thalassemias are classified into α and β-thalassemias. Thus, thalassemias could be cured by introducing or correcting a gene into the hematopoietic compartment or a single stem cell.

What is gene therapy for thalassemia?

How gene therapy for beta thalassemia works. Blood stem cells are collected from a patient and genetically edited outside the body to block the expression of the BCL11A gene, which normally shuts off the production of fetal hemoglobin shortly after birth.

Is there gene therapy for beta thalassemia?

β-thalassemia gene therapy is based on the transfer of a human β-globin gene into autologous HSCs, which resolves the absence of compatible donors and eliminates the risk of GVHD and graft failure associated with allogeneic BMT.

What is hematopoietic stem cell gene therapy?

Hematopoietic stem cells (HSCs) isolated from bone marrow can be modified ex vivo and transferred back to the recipient to produce functional, terminally-differentiated cells. Specific cellular targets and the relevant diseases and genes for gene therapy include the following: HSCs: Fanconi Anemia (FANC A–F).

Who has done research on thalassemia?

After three decades of research, Memorial Sloan Kettering Cancer Center investigators may have found a new treatment option for patients with an inherited blood disorder called beta (β)-thalassemia. The approach, led by MSK physician-scientist Michel Sadelain, involves using a new stem-cell-based form of gene therapy.

Can thalassemia show up later in life?

When the disorder develops later during life, a diagnosis of beta thalassemia intermedia is given; individuals may only require blood transfusions on rare, specific instances. Beta thalassemia major, also known as Cooley’s anemia, is the most severe form of beta thalassemia.

What research is being done for thalassemia?

Scientists are working to develop a gene therapy that may offer a cure for thalassemia. Such a treatment might involve inserting a normal beta globin gene (the gene that is abnormal in this disease) into the patient’s stem cells, the immature bone marrow cells that are the precursors of all other cells in the blood.

How does hydroxyurea work in thalassemia?

Hydroxyurea (HU) is an important factor in synthesis of fetal haemoglobin (HbF) and increases the level of HbF markedly [3]. Hydroxyurea (HU) increases the level of gamma globin and it may be effective in patients with β-thalassemia and improves clinical and haematological abnormalities of thalassemia intermedia [4].

Can Crispr cure thalassemia?

New results from two studies of CRISPR Therapeutics and Vertex Pharmaceuticals’ gene editing therapy give further evidence treatment can dramatically benefit, and potentially functionally cure, people with the inherited blood diseases sickle cell and beta thalassemia.

Can Crispr treat thalassemia?

In the past few years, CRISPR technology has been tested for treating several diseases. For instance, CRISPR gene therapy has made huge strides for treating beta thalassemia and sickle cell anemia—inherited blood disorders caused by mutations in the gene encoding hemoglobin.

How are stem cells used in gene therapy?

In the same way that viruses replicate by injecting their genetic material into living cells, gene therapy uses viruses to insert therapeutic genes into stem cells. For the treatment of ADA-SCID, stem cells are harvested from the patient, and a virus is used to insert a healthy version of the ADA gene.

Is stem cell therapy genetic engineering?

Genetic engineering of stem cells can be useful for increasing cell survival when transplanted, particularly into a hostile environment. They can be modified to deliver proteins to neighboring cells, kill cancer cells or reduce graft-host rejection.

What is the current research on thalassemia?

Can I get the Covid vaccine if I have thalassemia?

Currently, there are no serious warnings or precautions associated with the mRNA (COMIRNATY [Pfizer-BioNTech] or SPIKEVAX [Moderna]) vaccines in persons with thalassemia beyond those of the general population.

How long is the average lifespan of a person with thalassemia?

“Most thalassaemia patients would live up to the age of 25 to 30 years. Improved facilities will help them live up to the age of 60,” said Dr Mamata Manglani, head of pediatrics, Sion hospital.

Is hydroxyurea taken for life?

Take as directed Hydroxyurea oral capsule is used for long-term treatment. It comes with risks if you don’t take it as prescribed. If you stop taking the drug suddenly or don’t take it at all: Your cancer cells may divide more rapidly or your sickle cells may change back to their sickle shape.

How long should hydroxyurea be taken?

There are a few people who won’t respond, so the medication doesn’t help. But, you should try the hydroxyurea for at least six months before stopping It.

What disease did CRISPR cure?

Scientists are studying CRISPR for many conditions, including high cholesterol, HIV, and Huntington’s disease. Researchers have also used CRISPR to cure muscular dystrophy in mice. Most likely, the first disease CRISPR helps cure will be caused by just one flaw in a single gene, like sickle cell disease.

Is there a gene therapy for thalassemia?

Gene therapies for transfusion dependent β-Thalassemia: current status and critical criteria for success Thalassemia is one of the most prevalent monogenic diseases most frequently caused by quantitative defects in the production of β-globin leading to severe anemia.

Is thalassemia monogenic?

Thalassemia is one of the most prevalent monogenic diseases most frequently caused by quantitative defects in the production of β-globin leading to severe anemia. Technological advances in genome sequencing, stem cell selection, viral vector development, transduction and gene-editing strategies now …

Can gene therapy reduce need for red-cell transfusions in β-thalassemia?

Gene therapy with autologous CD34+ cells transduced with the BB305 vector reduced or eliminated the need for long-term red-cell transfusions in 22 patients with severe β-thalassemia without serious adverse events related to the drug product. (Funded by Bluebird Bio and others; HGB-204 and HGB-205 numbers, NCT01745120 .)

Can gene therapy overcome the limitations of allogeneic hematopoietic-cell transplantation?

In conclusion, we found that gene therapy with LentiGlobin drug product succeeded in overcoming a principal limitation of allogeneic hematopoietic-cell transplantation, which is a lack of a histocompatible donor. The safety profile after infusion was consistent with that associated with myeloablative conditioning with single-agent busulfan.