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Why are some conditions more suitable for gene and cell therapies than others?

Depictions of gene and cell therapies in the news can make it seem like a ‘miracle cure’. Science fiction narratives often suggest that, in the future, they will be used to treat all illness and injury. 

The idea of treating a condition or illness at its root may certainly seem more appealing than the idea of invasive surgery, or lifelong regimen of medication or physical therapy. 

But to assess whether this is possible, or desirable, we need to consider: what makes a condition suitable for treatment by gene and cell therapies? 

This explainer page is currently undergoing final review by members of the EuroGCT partnership.

Understanding the ‘root cause’ of a disease or condition

Gene and cell therapy work by correcting something which has gone wrong at a genetic or cellular level. This means that scientists need to understand what has gone wrong, and where it has gone wrong.

Recreating tissues for transplant 

The idea of treating tissue that is not functioning correctly, or which has been severely damaged, with a ‘lab-grown’ replacement is appealing. However, the difficulty of this task depends on the region of the body affected.

This is not to say that single-cell transplants are not technically challenging, or that they are easy to develop. However, transplants requiring multiple cell types must overcome additional challenges. 

Delivering and integrating therapies into the body

Gene and cell therapies need to be delivered to the right area of the body and activated at the right time, for the right length of time. This need for precision presents many challenges.

Risk and benefit

Any new therapy comes with unknowns, risks, and costs. With limited funding, and limited researchers to investigate different avenues, funders and researchers must consider where their efforts are most likely to be successful. Researchers and funders have to consider several questions.

Where does this leave us?

As with all therapies, there are challenges in developing gene and cell therapies. These challenges may be biological, technical, or logistical. Some may be overcome as researchers progresses; others will take political effort.

It is important to remember that for all the promise of gene and cell therapies, they are not the gold standard for all conditions. Traditional therapies can greatly improve the quality of life for people living with hard-to-treat conditions. For some conditions and some individuals, they may present a more suitable solution than gene and cell therapies. Gene and cell therapies are one tool among many for treating congenital and difficult-to-treat conditions.

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