A therapy using cells derived from a donor to treat a different patient. Often intended to become an ‘off the shelf’ cell therapy product which uses cells derived from a single donor to treat many unrelated patients.
The development of antibodies in response to foreign antigens.
An antibody, also known as an immunoglobulin, is a large Y-shaped protein produced by B lymphocytes that circulate in the blood and body fluids and can bind to and neutralise antigens as part of the body’s immune response.
A foreign substance, usually a protein, which is capable of producing an immune response in the body.
A therapy using cells derived from a patient’s own body. This often involves the extraction of cells and an ‘ex vivo’ (outside the body) step of growing and multiplying them before transplanting them back into the patient.
An antibody-producing cell that develops from stem cells in the bone marrow, circulates in the blood and matures in lymph nodes.
Beta thalassemia is a blood disorder that reduces the production of haemoglobin, which is the iron-containing protein in red blood cells that carries oxygen to cells throughout the body. This leads to a lack of oxygen in many parts of the body as well as an increased risk of developing abnormal blood clots.
The growth of cells in a laboratory dish for experimental research. The cells are grown in a solution, or medium, that contains nutrients and growth factors. Different factors can be added to the culture medium to initiate changes in cell behaviour.
A population of cells all carrying the same genes, grown in the laboratory through many cycles of growth and division over many generations of cells.
The process of turning scientific knowledge into approved medical treatments, through a series of carefully controlled research and approval steps.
A research study in human subjects to answer specific questions about vaccines or new therapies or new ways of using known treatments. Clinical trials are used to determine whether new drugs or treatments are both safe and effective.
When cells become “committed” they are bound towards a programme of differentiation, becoming specialised to perform particular tasks. For a stem cell, this means it no longer retains the ability to self-renew.
One of the two or more cells formed in the division of a single cell.
The process by which cells become specialised to perform particular tasks.
A self-replicating molecule that carries genetic material in all organisms except RNA viruses.
Pluripotent stem-cell lines derived from the inner cell mass of early embryos no later than 14 days after fertilisation, before formation of the tissue germ layers. Embryonic stem cells are both pluripotent – so have the potential to become different cell types in the body - and have an ability to replicate indefinitely.
When the nucleus is expelled from the cell in the final stages of red blood cell (erythrocyte) maturation.
Red blood cells.
This is concerned with both production and quality control. It ensures that medicinal products are consistently produced and controlled to the quality standards appropriate for their intended use. Products need to meet good manufacturing practice guidelines before they can be used in clinical trials and are also required by marketing authorisation for the medicinal products to be sold or prescribed.
Stem cells that give rise to all the blood cell types and are described as multipotent.
The iron-containing protein in red blood cells that carries oxygen to cells throughout the body.
A type of pluripotent stem cell derived from a non-pluripotent cell, typically an adult specialised cell such as skin cells. The adult specialised cell is manipulated in the laboratory, so that it becomes pluripotent and can make virtually any cell type in the body. These iPS cells also have the ability to self-renew, so are able to divide and produce copies of themselves indefinitely.
This is when iron accumulates in the body and can lead to organ damage. It is a concern among patients who receive frequent transfusions due to the amount of iron that builds up.
Multipotent cells that are able form multiple mature cell types that constitute an entire tissue or tissues. An example of this would be haematopoietic (blood) stem cells, which give rise to all the different blood cell types.
Pluripotent cells can give rise to all of the cell types that make up the body.
Reconstruction of diseased or injured tissue by activation of resident cells or by cell transplantation
Reprogramming allows us to turn already specialised adult cells of the body into stem cells. The cells are manipulated in a laboratory using genes to become induced pluripotent stem cells.
One of the two types of nucleic acid that exist in nature, the other being DNA. It forms the genetic material of some viruses.
The ability of a stem cell to divide and produce copies of itself for an indefinite period of time. This is the defining property of stem cells. Such stem cells have been described as "eternal" in the sense that they can continue growing indefinitely if kept in the right conditions.
Sickle cell anaemia is an inherited blood disorder where the red blood cells, which carry oxygen around the body, develop abnormally. Red blood cells in sickle cell anaemia are can become rigid and shaped like a crescent (or sickle). The sickle-shaped cells contain defective haemoglobin and can also block vessels, causing tissue and organ damage.
Stem cells are found throughout our bodies and help us replenish cells that cells that have been used up or damaged. They are able to replicate and produce copies of themselves – daughter cells – as well as possessing the ability to differentiate into more specialised cells for different cell types each time they divide.
Totipotent cells can form all the cell types in a body, plus the extra embryonic, or placental, cells.
A protein that binds to specific sequences of DNA and thereby activates or represses the production of messenger RNA (known as 'transcription' of DNA to RNA). Messenger RNA carries the code for the production of new protein