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Different Approaches

Gene therapy, cell therapy, and gene editing are fields of biomedical research with a similar goal in mind: To treat disease by targeting the cause of the disease. Learn more below about these different approaches, along with common challenges, risks, and benefits.

Key Concepts

Biomedical Research is a field of science that aims to understand biological processes with the goal of curing or treating disease in humans and animals. Biomedical research includes both basic research (in a lab) and clinical research (with patients). Understanding how living things operate on their most basic level can give us clues to achieve better health.

Genetic Material typically means DNA and RNA. DNA and RNA are strings of molecules that help instruct cells to create proteins. DNA helps store the genetic information, like a blueprint. RNA’s function is to then convert the genetic instructions contained within DNA into a format that helps cells build the correct proteins.

Gene Therapy is the introduction, removal or change in genetic material in the cells of a patient to treat an inherited or developed disease. Typically, genetic material, such as a working copy of a gene, is delivered to cells using a vector. A vector is often derived from a virus. For safety, all viral genes are removed and the vector is modified to only deliver therapeutic genes into the cells. Once in the cell, a working copy of the gene will help make proteins despite the presence of a faulty gene. Achieving the normal expression and function of proteins makes a big impact on our overall health. Learn more about Gene Therapy Basics or Vectors 101.  

Cell Therapy is the transfer of cells into a patient with the goal of improving a disease. Some cell therapies are routine, like blood transfusions. One approach is gene-modified cell therapy, which removes the cells from the patient’s body, then a new gene can be introduced or a faulty gene can be corrected. The modified cells are then put back into the body. An example of this approach is CAR-T cell therapy.  

Gene Editing is a type of gene therapy. The goal of gene editing is to remove or correct pieces of DNA within the gene rather than replace the gene as regular gene therapy would. Gene editing uses technology that is highly precise to make this change inside the cell. The cells can be from the patient or donor.

Challenges

  • No one-size-fits all. Each disease with a certain gene mutation requires a specific gene therapy approach. There is no one-size-fits all. Currently, most gene and cell therapy options are limited to treating only the diseases that are caused by a single (monogenic) gene mutation. Some genetic diseases are caused by mutations (changes) in a single gene, while other diseases are caused by mutations in multiple genes, for example, cancer. Environmental factors, such as UV radiation from the sun, can even play a role in causing disease. Diseases can be complex, making them difficult to develop and test treatments.

  • Small number of patients. Gene therapy often targets rare diseases, meaning there is are a small number of people with the disease. This means it can take longer or be more difficult to gather enough research data or to find people that are eligible to participate in a clinical trial. 

  • Funding. Testing therapies in clinical trials requires funding for the studies, preparation of regulatory documents and ensuring safety for humans. There is a lot of cost that goes into the work it takes to prepare a clinical trial, make a gene therapy, and ensure it is carried out at the highest standard. Having enough funding can help speed up the development process of promising therapies. 

  • Right place, right time. Gene and cell therapies need to express the gene in the right tissue, at the right level, for the right amount of time. This means that a lot of research goes into the best way to deliver the genetic material. The response of the patient’s immune system also needs to be considered based on the therapy. 

Learn more about the clinical trials process.

Risks

  • Informed consent. Before participating in a clinical trial, a member of the research team should review any potential risks and benefits with the patient or caregiver.  

  • No guarantees. Therapies being studied in clinical trials are not a guaranteed cure and cannot guarantee beneficial results. There is always a chance that the investigational treatment may not work.  

  • Cannot be given another dose. In the event a person is not satisfied with the outcome, the person cannot receive another dose of the gene therapy. Participating in a clinical trial may also prevent future participation in other trials or from receiving other types of treatments.  

  • Long-term effects unknown. Gene therapy can be an alteration for the lifetime, so people should be aware that there could be long term effects (both good or bad) that are unknown at this time. 

Benefits  

  • Hope for rare and fatal disease. Gene and cell therapy can help treat rare and serious diseases that have limited treatment options. Many of these rare inherited diseases would end in disability or premature death. But with gene and cell therapy, early studies show that these rare diseases have been slowed or completely stopped. 

  • Earlier is better. If gene therapy is received earlier in the course of disease, it has the potential to stop any damage before it occurs. But, it is still being researched to what extent a gene therapy might reverse any damage.  

  • One-time treatment. Gene and cell therapy aim to be given one time and have the potential to be maintained over a long period of time without needing to be given again.  

  • Targets the cause. Gene and cell therapies make it possible to design treatments that can target any of the thousands of genes in the body. Targeting specific genes targets the cause of the disease.  

Explore more resources on our website about gene therapy for specific diseases, helpful insights on the patient journey, and clinical trials information.  

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Last Updated: 07/26/2021

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