Scientific advances have helped us better understand human genetics and spurred development of therapies designed to address the root cause of genetic disorders: variations in a gene.
GTReference intends to provide comprehensive information to help you understand how changes in a gene can cause a genetic disorder and to provide an overview of how cell and gene therapies aim to treat these disorders.
There are many different types of cell therapy and gene therapy, and the science is rapidly evolving. To help focus the information and resources provided, GTReference provides information needed to understand how hematopoietic stem and progenitor cell (HSPC) gene therapy, sometimes referred to as HSC gene therapy, works.
and Your Body
The key to understanding genetic disorders
Many human diseases and disorders have an inherited genetic component that may have a direct effect on how the body functions, or its effect may be influenced by environmental and lifestyle factors. It is therefore important to learn about the basic concepts of human genetics and the key roles that cells, DNA, genes, and chromosomes play in regulating our health.
Our goal is to help you understand how changes in a gene can cause a genetic disorder and how cell therapy and gene therapy might work to restore the lost function of the affected gene.
Harnessing the power of the cell to treat disease
Enhanced understanding of how cells function and replicate has led to the development of new therapies that utilize cells to treat various diseases.
Potential solutions to genetic disorders
Scientific advances in the understanding of human genetics have spurred the development of numerous gene therapies designed to address a disease or condition at its root cause: a change or variation in a gene.
More information to help you learn about gene therapy
Gene therapy is complex and reading through this website is just the beginning! Explore these tools and resources to learn more about gene therapy.