Resources to educate on gene therapy research


Checklist Resource Guide

Designed with your teams in mind, HemDifferently created the following checklists as tools in supporting their understanding of gene therapy research. Each individual resource guide can benefit your team by highlighting gene therapy research and how it applies to different team structures.

Gene Transfer 101

A simple one page diagram helping patients and caregivers to understand the 5 steps involved in gene transfer research.

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HemDifferently Brochure

A comprehensive brochure for patients and caregivers that goes into the science and research behind gene therapy.

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What Is a Therapeutic Vector?

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Demystifying Gene Therapy Research

Getting to Know Gene Therapy Research:
Behind the Science and Practical Considerations

Annette von Drygalski, MD, PharmD, RMSK
Director, Hemophilia and Thrombosis Treatment Center at University of California San Diego Health
Professor of Clinical Medicine, UC San Diego, California

A background on gene therapy research

With 2,500 studies completed, ongoing, or initiated worldwide, gene therapy is being evaluated to determine the benefits and risks for a range of genetic conditions, including hemophilia A and B. Some gene therapies have already been approved for treatment of certain conditions, including a genetic eye disease and spinal muscular atrophy. No gene therapies for hemophilia A or B have been approved for use or determined to be safe or effective.

There are three different types of gene therapy being researched, including gene editing, cell therapy, and gene transfer. At HemDifferently, we’re particularly interested in the method of gene transfer.

How is gene transfer designed to work?

In gene transfer research, a working copy of a mutated gene is designed in a laboratory to provide instructions for the body to make the proteins it needs. That working gene is placed in a protective viral shell and delivered through one-time infusion. Once inside the body, the functional gene is designed to provide instructions for the body to produce the missing or mutated gene. Ongoing research monitors clinical trial participants to gather data to better understand short as well as long term safety and efficacy.

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