Gene therapy

About the Road to Gene Therapy for GNE Myopathy 

About PIGA-CDG: is a rare and potentially devastating metabolic disorder that affects development across many systems of the body. Watch the video below and learn more about the function of PIGA in the human body, the symptoms of the disorder and potential areas of research for treatment. 

About SRD5A3-CDG: is an inherited condition that causes neurological and vision problems and other signs and symptoms. The pattern and severity of this condition's features vary widely among affected individuals.

How can you help our CDG community? Is easy! Share among your social media and other channels the information we make available within this section and website. This raises awareness, accelerates diagnosis, and secures better care and management for our CDG children and adults!

Gene therapy can potentially help all CDG, as it is capable of correcting the faulty gene copies that cause CDG.

What is gene therapy?

Gene therapy is a type of treatment that “corrects” patients’ genes to potentially treat or prevent diseases. The faulty gene is replaced with a copy of a healthy gene using a transport vehicle (also called a vector, which is usually a virus) to transport the healthy gene and introduce it into the cells. The goal of gene therapy is to treat a genetic disease by correcting the defect within a person’s DNA (read more here). It is a very promising and challenging therapy.

Watch the video released by the U.S. Food and Drug Administration entitled Gene Therapy Inside Out:

The American Society of Gene & Cell Therapy (ACGCT) has released a series of videos that helps to understand Gene therapy.

Watch below the Basics about Gene therapy. And learn more here.

National Organisation for Rare Diseases RareEDU™ released the video below, Gene Therapy: Your Questions Answered, in order to address a vital topic to today's rare disease community. The goal of this video is to address the questions, hopes and concerns that patients and caregivers, across many different diseases, have about gene therapy. Since more than 80% of rare diseases are believed to be genetic, this video will serve as a helpful resource for the rare disease community.

Watch the video below about vectors that are essentially vehicles designed to deliver therapeutic genetic material, such as a working gene, directly into a cell.

During the educational session below, experts in the field reviewed different gene therapy approaches, disease applications, and research processes, and described the current state of the field.

During the forum below, key stakeholders in the space presented a variety of novel models for the development of gene therapy for people with ultra-rare diseases and those living in countries with lower income economies.

The following website here, the video and the infographic below from American Society of Gene & Cell Therapy (ACGCT) explain the difference between gene therapy, cell therapy, and gene editing.

Complete your knowledge with available resources to help you better understand this therapeutic approach, including 

• What is gene therapy? here and here 
• Visit NORD section about Understanding the Importance of Gene Therapy for Rare Diseases