• Understanding Genetics and Your Body
    • Overview of Cells
    • Overview of DNA
    • What is a Gene?
    • Overview of Chromosomes
    • What is a Gene Mutation?
    • Understanding Genetic Disorders
    • Monogenic Disorders
  • Understanding Cell Therapy
    • What is Cell Therapy?
    • Overview of Stem Cells
    • Overview of Hematopoietic Stem Cells (HSCs)
    • What is Hematopoietic Stem Cell Therapy?
  • Understanding Gene Therapy
    • What is Gene Therapy?
    • How is Gene Therapy Delivered?
    • What are the Potential Risks of Gene Therapy?
  • Understanding HSC Gene Therapy
    • The HSC Approach to Gene Therapy
    • How is HSC Gene Therapy Made?
    • What is Conditioning?
    • What is the HSC Gene Therapy Process?
  • Resources
    • Online Resources
    • Glossary
  • Keep Me Informed
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AVROBIO Gene Therapy Education

AVROBIO Gene Therapy Education

AVROBIO Gene Therapy Reference

  • Online Resources
  • Glossary
  • Keep Me Informed

The Basics

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.

Start here

Overview of Cells

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. 

Understanding Genetics
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.

Start Topic 1 ›› Read Section 1

  1. Overview of Cells
  2. Overview of DNA
  3. What is a Gene?
  4. Overview of Chromosomes
  5. What is a Gene Mutation?
  6. Understanding Genetic Disorders
  7. Monogenic Disorders

Understanding
Cell Therapy

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.

Start Topic 2 ›› Read Section 1

  1. What is Cell Therapy?
  2. Overview of Stem Cells
  3. Overview of Hematopoietic Stem Cells (HSCs)
  4. What is Hematopoietic Stem Cell Therapy?

Understanding
Gene Therapy

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.

Start Topic 3 ›› Read Section 1

  1. What is Gene Therapy?
  2. How is Gene Therapy Delivered?
  3. What are the Potential Risks of Gene Therapy?

Understanding HSC
Gene Therapy

An approach to gene therapy

Hematopoietic stem cell (HSC) gene therapy aims to treat or prevent genetic disorders by modifying the patient’s own HSCs.

Start Topic 4 ›› Read Section 1

  1. The HSC Approach to Gene Therapy
  2. How is HSC Gene Therapy Made?
  3. What is Conditioning?
  4. What is the HSC Gene Therapy Process?

Resources

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. 

Start Topic 5 ›› Read Section 1

  1. Online Resources
  2. Glossary

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