Cell and Gene Therapy

Cell and gene therapy are rapidly evolving disciplines of precision medicine that have shown promise in treating various diseases or medical needs in humans. Despite both existing in the field of precision medicine they are functionally different techniques.
Current examples of cell and gene therapy are:

Haematopoietic stem cells
Skin grafts
Expanded autologous cells
Recombinant oncolytic viruses
Novel gene editing technologies
Engineered tissues

[1]

Cell therapy aims to repair or replace damaged cells via the implantation of healthy cells into the body. Cell therapy has existed for some time in forms such as blood or marrow transfusions, however, recent advancements in the field have provided promising additional treatments utilizing cell therapy.

In contrast, gene therapy relies on the modification or manipulation of a gene expression or biological process to achieve a therapeutic outcome.[2] Gene therapy can work in several ways including:

Utilizing a new or modified gene to treat a specific disease
Deactivating a gene that is not functioning correctly and causing a disease
Replacing a disease-causing gene with a new healthy copy of the gene

[3]
The United States Food and Drug Administration has approved certain gene transfer therapies for clinical use however due to the lack of clinical studies genome editing therapies are currently undergoing risk evaluation.[4]

Cell and Gene Therapy Regulations

In the United States, The Center for Biologics Evaluation and Research (CBER) utilizes the Public Health Service Act and the Federal Food Drug and Cosmetic Act to regulate cell and gene products as well as devices related to cell and gene therapy.[5] Currently, CBER has approved over 30 cell and gene therapy products.[6]

In the United Kingdom, cell and gene therapies (also called advanced therapy medicinal products, or ATMP) are regulated by The Medicines and Healthcare Products Regulatory Agency (MHRA). MHRA is the governing body responsible for both the clinical trial authorization as well as overseeing the importation or manufacture of ATMP.[7]

What Instruments are used for Cell and Gene Therapy?

Cell and gene therapy is a diverse field requiring an equally diverse array of laboratory instrumentation. Instruments that are currently used throughout cell and gene therapy research and development include:

Incubators
Biological safety cabinets
Laboratory freezers
Laboratory microscopes
Laboratory centrifuge
Flow cytometer
Fluorescence microscopes

[1] "Cell, Tissue and Gene Therapy Products", World Health Organization, https://www.who. int/teams/health-product-policy-and-standards/standards-and-specifications/cell--tissue-and-gene-therapy-products
[2] "Long Term Follow-Up After Administration of Human Gene Therapy Products Guidance for Industry", U.S. Department of Health and Human Services Food and Drug Administration Center for Biologics Evaluation and Research, https://www.fda.gov/media/113768/download?attachment
[3] "What is Gene Therapy?", U.S. Food and Drug Administration, https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/what-gene-therapy
[4] "GENETIC THERAPIES Benefits and Risks", National Heart, Lung, and Blood Institute, https://www.nhlbi.nih.gov/health/genetic-therapies/benefits-risks
[5] "Cellular & Gene Therapy Products", Center for Biologics Evaluation and Research Food and Drug Administration, https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products
[6] "Approved Cellular and Gene Therapy Products", Center for Biologics Evaluation and Research Food and Drug Administration, https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/ approved-cellular-and-gene-therapy-products
[7] "Advanced therapy medicinal products: regulation and licensing", Medicines and Healthcare products Regulatory Agency, https://www.gov.uk/guidance/advanced-therapy-medicinal-products-regulation-and-licensing