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Cell and Gene Therapy Overview

Overview of Cell and Gene Therapy 

Cell and Gene Therapy involves extracting cells, protein or DNA from a patient or donor and altering the genetics to provide a targeted result once reinjected into the patient. Within the past 50 years significant advances in biopharmaceuticals have resulted in significant successes in the treatment of a wide range of haematological, neurological and oncological disorders. Overall, this method of therapy can offer a more effective and longer lasting effect than traditional medicines.

This technique of altering genetic material can greatly impact an individual’s quality of life and offer a sense of hope when more traditional methodologies may have failed them or are not available. There are a variety of innovative methods that can be utilised in order to obtain the desired results.

Cell Therapy

Cell Therapy can be either from a patient’s own body (autologous) or from a donor (allogenic) and have the potential for unlimited application when treating disease. A variety of cells can be utilised in the procedure of Cell Therapy ranging from lymphocytes, red and white blood cells, platelets and most notoriously stem cells.

Pluripotent stem cells are capable of the production of any human cell, operating as a potential source for inaccessible or scarce cells currently present in the patient. These cells can be divided into two forms, embryonic stem cells (ESCs) and induced pluripotent stem cells (iPCSs).

ESCs – Derived from early-stage embryos (blastocysts) and can develop into over 200 human cell types if coded to do so. Another key factor is their capability to replicate indefinitely.

iPCSs – Mature somatic cells, commonly skin or blood cells that have been reprogrammed into an ESC like condition, it additionally possesses the same, critical, infinite replication characteristic as EPCs.

Gene Therapy

Altering the genetic material can result in the prevention of disease via modifying how a single protein, or group of proteins, is synthesised by the cell. A primary example of this treatment is either substituting a mutated gene with a functional copy or introducing a required gene to the patient. These methods can result in reducing levels of a disease-causing version of a protein, increase effectiveness of the patient’s immune system and/or produce new or modified proteins.

Gene Therapy can be carried out both in vivo (the gene can be delivered directly to the patient’s targeted cells) or ex vivo (where the therapeutic gene is inserted into cells external from the body in a laboratory, before being introduced to the patient). Therapeutic, altered genes are administered via a deactivated virus, most commonly lentiviruses, retroviruses or adeno-associated viruses.

There are a plethora of methods and approaches that can be utilised to execute ex vivo Gene Therapy such as T-Cell Therapy, natural killer Cell Therapy and tumour infiltrating lymphocytes. However, this technique is most frequently applied to hematopoietic stem cells (HSCs) and is utilised to treat immunological and genetic diseases that influence tissues and organs reached by blood cells.

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