Gene Therapy

Updated On Feb 25, 2022

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This information is meant to be used for educational purposes to inform providers, patients, and genetic service delivery stakeholders about genetics policy topics. Sharing of information, resources, or policy statements is no way an endorsement of stated positions by NCC.

Introduction

Genetic illnesses are caused by genetic changes in the structure of a gene or a region of the genome.  Gene therapy is an experimental procedure that uses genes to treat or prevent disease.  Using gene therapy, it may be possible to introduce working genes into an individual’s genetic code.  Or, it could be possible to simply “knock out” or inactivate a gene that isn’t working properly and is making incorrect proteins.

For more information on gene therapy, including a discussion of the ethics surrounding gene therapy, please visit the MedlinePlus website.  MedlinePlus is maintained by the National Institute of Health, U.S. National Library of Medicine.  

Genetic technologies, such as genome therapy, raise practical and ethical questions. Some worry that these technologies could cause more harm than good.  For example, if a gene is introduced in the wrong place in a person’s genetic code, it could cause other genes to not function anymore.  This could cause larger health problems than the one it was deployed to correct.  One such case occurred in 2003, when the FDA had to halt a clinical trial on X-linked severe combined immune deficiency disorder (SCID) due to it causing leukemia; more information on that trial can be found here.


As explained in the MedlinePlus article above, some worry that these technologies could be used for non-health related reasons.  For example, could people use gene therapy to increase height simply to make someone a better basketball player?  How does one draw a line between permissible vs impermissible gene therapy?  Whose right is it to make these decisions?

 

Legislation and Regulation

There are no laws or regulations that apply specifically to gene therapy.  At this time, gene therapy is still in the realm of research, and thus, test subjects would be covered by general research protections.  “The Web of Legal Protections for Participants in Genomic Research” is an article that summarizes and discusses these protections.  In general, this type of research is largely subject to local oversight by institutional biosafety committees (IBCs) and institutional review boards (IRBs), which develop policies for the researchers at their institutions.  Research that is funded through the NIH are subject to their regulations.  A good overview of regulation can be found in this article.

 

Are you interested in learning what your state’s government or the federal government are currently proposing for either legislation or regulation? Check out Legislative/Tracking system for up-to-date information and subscribe to our Twitter channel to get the latest updates in your pocket.

Policy Positions

Organizations working within the genetics community (national genetic organizations, advocacy organizations, etc.) have published positions on gene therapy. Explore these position statements below. 
Position Statements by Organizations
Genome editing in clinical genetics: points to consider—a statement of the American College of Medical Genetics and Genomics
Link
https://www.acmg.net/PDFLibrary/Genome-Editing-Clinical-Genetics.pdf
Organization
American College of Medical Genetics and Genomics (ACMG)
Subject
Gene Editing
Date
January 26, 2017
Encouraging Responsible Genome Editing Research
Link
https://www.cell.com/ajhg/fulltext/S0002-9297(17)30247-1
Organization
American Society of Human Genetics (ASHG)
Subject
Gene Editing Genetic Research
Date
August 3, 2017
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