Advocacy   |   Education

ASGCT Debuts Patient Education Program

ASGCT Staff - January 18, 2019

Gene Therapy 101 Collection Now Live on

The American Society of Gene & Cell Therapy (ASGCT) is proud to announce the first release of the Society's Patient Education program, a new initiative for 2019. Designed by ASGCT committee volunteers in coordination with patient advocacy groups, the new patient-centered portal is designed to educate and inform patients, families, and the public on the status and promise of gene and cell therapies.

Gene Therapy 101, a collection of three high-level gene and cell therapy explainers, is now live on Five disease-specific topics will release through February, ultimately providing patients and patient advocates with video, text, and infographic resources to share with various stakeholders. ASGCT encourages people to share these resources with their friends and family.

Gene Therapy 101

Gene Therapy Basics

Gene therapy has been studied for more than 40 years and can help stop or slow the effects of disease on the most basic level of the human body—our genes. And to understand how it works, we’ll start at the basics.

Different Approaches

Gene therapy, cell therapy and gene editing are fields of biomedical research with a similar goal in mind: To treat disease by changing our bodies at a microscopic level. Specifically, these approaches aim to change our genetic makeup. However, each one changes our genetic material in slightly different ways. What do these approaches have in common, and what makes them different?

Developing a Treatment

Let’s learn how gene therapies go from concepts to actual treatments that can improve the lives of patients. First, the therapy spends a lot of time in preclinical studies, meaning that it is not yet ready for humans and is being researched in labs and animals to learn if it could be successful. By the time the therapy enters clinical trials for humans, it is better understood and ready for further development. Here’s more information about the process of developing a treatment.

Disease Treatments

Over the course of the next month, ASGCT will release five disease-specific resources. The first disease groups were selected for the promising statuses of their gene and cell therapy diseases.

  • Spinal Muscular Atrophy—February 1

  • X-Linked Myotubular Myopathy—February 12

  • Leukodystrophy—February 15

  • Blood Disorders—February 22

  • Inherited Retinal Disorders—February 28

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