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Introduction to cell and gene medicine

Dig Deeper

Sometimes, a disease or debilitating health condition is caused by one or more genetic changes that occur inside the body.

Many diseases or conditions caused by defective genetic code have few treatment options. Conventional medicine often treats the symptoms of the disease or slows down the disease. Doctors use cell and gene medicine in an attempt to resolve the underlying genetic defect.

Illustration Credit: Monica Schroeder/Science Source/SCIENCE PHOTO LIBRARY​

What causes genes to mutate?

  1. Inherited mutations can be passed from parents to their children.
  2. Other gene mutations can occur when a piece of genetic code is missing, defective, or unfortunately duplicated in error during pregnancy.
  3. Mutations can occur because of damage to cells. (Skin cancer is one disease caused by long-term changes to cells after too much exposure to sunlight’s ultraviolet radiation.)
  4. There also are genetic mutations that cause significant illness only in adulthood, such as delayed-onset Type 1 Diabetes.

Not all mutations cause a need for cell or gene medicine. Some simply create unique and wonderful distinctiveness. However, when a mutation causes excruciating pain, is life-threatening, or may impact the quality of life, doctors may recommend cell or gene medicine.

Example

This is what a physician scientist may see when searching for a mutation. This is an autoradiogram (X-ray film) of DNA showing a ras gene which has mutated, and which is associated with bowel cancer. The pattern of black bands represents the coded structure of the gene. A segment of the gene (see the arrow drawn on the image) has mutated to an abnormal code “CAC”. As a result of the gene mutation, an abnormal protein is produced. Mutated sequences of the ras gene cause almost half of human bowel cancers. This gene mutation was identified using a technique called ARMS (Amplification Refractory Mutation System). The photograph was taken at ICRF Applied Development Laboratory, UK.

Location: ICRF Applied Development Laboratory, UK Photo Credit: James King-Holmes/Science Photo Library​

You're Full of Genes

Especially for Children (Ages 5-9)

Author: Dr. Claudia Zylberberg, CEO of Akron Biotech

The book takes children on a journey with games and activities that help them learn about how trillions of cells with trillions of genes work to make each child unique.

Buy it on Amazon:

https://www.amazon.com/Youre-Full-Genes-Claudia-Zylberberg/dp/0578620669

The Gene: An Intimate History

General Education

This short television series, from Ken Burns and Barak Goodman, is adapted from a book Dr. Siddhartha Mukherjee.  It explores the ethics and breakthroughs of gene-based medicine. 

Watch it on PBS:

https://www.pbs.org/kenburns/the-gene/

Add Resources

These videos and websites provide basic information on the science of cell and gene therapy when you want to dig deeper. If you ever find a great resource that describes cell and gene medicine clearly – and you really like the resource – please let us know here. We want to ensure great resources are easy to access. Also, if you ever find any information you believe may contain an error or allow a visitor to infer something that’s not accurate, please let us know immediately here. We care deeply about the integrity of our website. Note: We have a team of volunteers who screen content before we add it to Healing Genes. If you send a great recommendation and don’t see it right away, that’s because our healthcare volunteers are making sure it’s accurate.