DIY Gene-Therapy Claims

What the claim usually sounds like

Online posts, videos or sellers may say that gene therapy can now be done at home, by a "biohacker", for longevity, muscle, appearance or a disease. They may use real scientific terms such as CRISPR or "gene delivery" to sound credible. This page checks the claim, not a product, and names no brand or seller.

What the FDA says

• Gene therapy is a regulated medical product. The FDA says gene therapy products are biological products regulated by its Center for Biologics Evaluation and Research. Human studies require an investigational new drug application before they begin, and marketing a product requires approval of a biologics license application. [3]
• CRISPR in humans counts as gene therapy. The FDA considers any use of CRISPR/Cas9 gene editing in humans to be gene therapy. [1]
• What to look for. The FDA cautions consumers to make sure that any gene therapy they are considering has either been approved by FDA or is being studied under appropriate regulatory oversight. [1]
• Registration is not approval. The FDA says that inclusion of a product on ClinicalTrials.gov, or a firm registering with FDA, does not mean the product is legally marketed. [2]

Why this is not a do-it-yourself technology

Approved and trial gene therapies involve manufacturing controls, safety testing and monitoring that individuals cannot reproduce. The reasons below come from what regulators and published cases describe.

• Immune reactions can be severe. In a 2003 case report, an 18-year-old with partial ornithine transcarbamylase deficiency died 98 hours after receiving an adenovirus-based gene transfer in a supervised safety study. The authors pointed to the steep toxicity curve for this type of vector and to large differences in how individuals respond. [4] A review reports that fatalities have also been observed with high-dose systemic AAV gene therapy due to liver, kidney, heart or lung failure. [5] These happened in medical research settings with monitoring, and people outside such settings would have no comparable safeguards.
• Delayed risks. The FDA says gene therapy products are often designed to produce permanent or long-acting changes, and people may be at increased risk of undesirable and unpredictable outcomes, so those in trials may be monitored for extended periods. [6] Cancers have been reported years after some approved or trial gene therapies. [7]
• Unintended DNA changes. Gene-editing tools can cause off-target changes, and methods to detect them are not standardized. [8] See off-target effects, durability and long-term monitoring.
• Quality and identity. The FDA warns that illegally marketed regenerative products can cause infections including life-threatening blood infections, unwanted immune responses and cross-contamination related to how they were prepared. [2]
• Even approved products have serious risks. For example, the FDA lists a boxed warning for acute serious liver injury and acute liver failure for the approved gene therapy Elevidys, with its use limited by the label. [9]

Claim check

Where real access exists

Approved products are prescribed by specialists for specific diseases. See approved gene and RNA therapies. Supervised trials are listed on ClinicalTrials.gov. [1] See clinical trials explained for participants and expanded access. Ethics and oversight of human genome editing are discussed in somatic vs germline interventions.

Who should be especially careful

Anyone with a serious illness who is tempted by an unapproved offer, parents considering anything for a child, and anyone taking medicines or with liver, immune or blood conditions. Talk with a doctor first.