An introduction to Gene & Cell Therapy

Hudi Pharma Cell & Gene Therapy

We want to talk about Cell & Genes therapy due to their  role in treating diseases with a more profound impact on people and their lives. It is estimated that millions of people globally suffer from more than 10.000 rare diseases caused by mutations of single genes. For these patients, cell and gene therapies offer hope and potential cures for previously untreatable diseases and difficult-to-treat diseases across a wide array of medical fields.

 
What are cell & genes? 
Cells are the basic building blocks of all living things, and genes can be found deep within cells. Genes are small sections of DNA that carry genetic information and instructions for making proteins, which help build and maintain the body1.
 
 
What are genetic diseases? 
Genetic diseases happen when a critical piece or whole section of DNA is substituted, deleted or duplicated2. These changes are called genetic mutations3. Some serious genetic diseases caused by genetic mutations can be passed to future generations4. 
 
How do cell and gene therapies help treat genetic diseases? 

Cell therapy and gene therapy are overlapping fields of biomedical research and treatment6. Both therapies aim to treat, prevent, or potentially cure diseases, and both approaches have the potential to alleviate the underlying cause of genetic diseases and acquired diseases6. But, cell and gene therapies work differently.

The difference between cell therapy and gene therapy:


    • Cell therapy aims to treat diseases by restoring or altering certain sets of cells or by using cells to carry a therapy through the body5. With cell therapy, cells are cultivated or modified outside the body before being injected into the patient. The cells may originate from the patient (autologous cells) or a donor (allogeneic cells)6.
    • Gene therapy aims to treat diseases by replacing, inactivating or introducing genes into cells— either inside the body (in vivo) or outside of the body (ex vivo)6.
 

Some therapies are considered both cell and gene therapies. These therapies work by altering genes in specific types of cells and inserting them into the body.

References – Novartis 
  1. National Institutes of Health (NIH) U.S. National library of medicine. What is a gene? Available at: https://medlineplus.gov/genetics/. Last accessed April 2019.
  2. National Institutes of Health (NIH) U.S. National library of medicine. What kinds of gene mutations are possible? Available at: https://medlineplus.gov/genetics/. Last accessed April 2019.
  3. National Institutes of Health (NIH) U.S. National library of medicine. What is a gene mutation and how do mutations occur? Available at: https://medlineplus.gov/genetics/. Last accessed April 2019.
  4. World Health Organization. Genes and human diseases. Last accessed April 2019.
  5. Friedman T. A brief history of gene therapy. Nat Genet. 1992; 2: 93-98.
  6. American Society of Gene & Cell Therapy. Different approaches. Available at: https://patienteducation.asgct.org/gene-therapy-101/gene-therapy-approaches. Last accessed April 2019.& 
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Francisca Porter

Francisca Porter