Understanding Cell & Gene Therapy

Cell and gene therapies are the result of decades of scientific research and innovation from around the world. These therapies are unlocking new ways to revolutionize medicine and standards of care, leading to better health outcomes for more people.

Cell Therapy vs. Gene Therapy

Cell therapy involves the transfer of cells with a certain function into a patient to fix an underlying problem.13 Gene therapy has the same goal but improves function by transferring genetic material (usually in a carrier or vector) into the appropriate cells of the body.14

Both cell and gene therapies differ from traditional pharmaceutical treatments. They target the underlying cause of genetic and acquired diseases with the aim of treating, preventing, and potentially curing disease.

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The Power of Cell & Gene Therapies

Precision

Cell and gene therapies are highly precise, targeting the underlying causes of diseases and disorders, and not just the symptoms.

Potential

Since these therapies focus on underlying causes, they have the potential to cure disease, from cancers to rare and chronic diseases. This includes genetic and acquired diseases and disorders that currently do not have effective treatments.15

Prevention

Cell and gene therapies have the potential to prevent damage before it happens because they can potentially slow or stop disease progression.

What is Cell Therapy?

Cells are the basic building blocks of the tissues and organs of our bodies. Each cell carries genetic instructions for what type of cell it will be, and the role that cell plays in how the body works. For example, some cells support our blood, delivering oxygen throughout our bodies, while others are important for muscle development or bone function.

Healthy cells are essential for healthy bodies. Unfortunately, they don’t always function as they should, which can lead to illness. In some cases, gene mutations create cell function problems from birth. In others, cells can become damaged over time. This can happen as we age or because of environmental factors.16

Cell therapies aim to replace or repair the impaired cells or tissues, by transferring other human cells into the body.17

How Cell Therapy Works

Cell therapy involves transplanting a specific type or types of cells into the body to treat or prevent a disease.18

Depending on the therapy, cells can:

  • Be taken from the patient’s own body, then modified and returned to the body (autologous cell therapy), or
  • Be taken from a compatible donor (allogeneic cell therapy).

Cell Therapy Types

Cells used for cell therapy are classified based on their potential to transform into different cell types. Depending on the treatment, different types may be used.

Pluripotent cells can transform into any cell type in the body. Embryonic stem cells and umbilical cord stem cells are both pluripotent cell types.19

Multipotent cells can transform into a limited number of other cells. For example, hematopoietic (blood forming) stem cells can transform into any type of blood cell that the body needs. Another example is mesenchymal stem cells (commonly found in bone marrow or fat), which can transform into the same type of cell as those surrounding it or be used to deliver medicine to a specific targeted area of the body.

Differentiated or primary cells are of a fixed type. Epithelial cells that cover the skin, blood vessels and body cavities, as well as muscle cells, are two examples of primary cell types.

What is Gene Therapy?

Gene therapy uses genetic material to fix or replace genes that do not work properly, to prevent or treat diseases.20 It has the potential to improve the lives of people living with different diseases, including inherited disorders.21
 
Gene therapy involves modifying the genes present within certain cells of the body. For example, a faulty gene that causes disease could be replaced with a healthy copy of that gene to restore a cell’s function. Or a different gene could be introduced to provide instructions for the cell to function properly.22

How Gene Therapy Works23

To do this, genetic material (DNA or RNA) is delivered to cells to modify how those cells produce single proteins or groups of proteins that are necessary for a given cell to properly function. Essentially, the genetic material that is introduced gives the cells the instructions for what to do.
 
Typically, this genetic material is delivered directly to cells using vectors, which are often developed from modified viruses. Since viruses have proven to be good at making their way into cells, they’re useful for delivering genetic material. Viral genes are removed to make them safe to use as vectors, so that they don’t cause infection. 

Vectors can be delivered two ways:

  • Ex vivo: Removing a patient’s own cells, then delivering the genetic material to these cells outside the body before returning them to the patient
  • In vivo: Delivering the genetic material directly to the patient

What is Gene Editing?

Gene editing (also called genome editing) is a type of gene therapy that is showing promise for several diseases, from sickle cell disease to HIV.24
 
It involves directly editing the existing DNA in a cell – a one-time process that aims to treat or prevent disease.25
 
While gene therapy uses genetic material to change how a cell functions, gene editing involves actually changing the DNA within those cells, which then changes the instructions for what the cell should be doing to prevent or treat a disease (such as producing more of a certain type of protein). 
 
Like the vectors used in gene therapies, gene editing can be administered ex vivo (removing cells, editing the DNA within them and reintroducing them) or in vivo (editing cells directly within the patient’s body). 
 
Researchers are exploring different types of gene editing, with early clinical trials producing positive results.