Wolfgang Miesbach: How Safe Is AAV Gene Therapy?
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared a post on LinkedIn about a recent article by Lindsey A George et al., published in Molecular Therapy, adding:
“6,000+ people treated. Nine tumors reported.
When a cancer shows up after AAV gene therapy, was the treatment to blame, or just chance?
That’s the new Opinion piece in Molecular Therapy by Lindsey George, Rebecca Ahrens-Nicklas and Frederic Bushman.
First, the reassuring part.
AAV gene therapy has a strong safety record. Across an estimated 6,000 plus people treated, only nine tumors have ever been reported afterward – and just one carries clear molecular proof that the vector itself caused the cancer.
That one case is sobering.
A child treated for MPS I at 13 months developed a brain tumor four years later.
The vector had landed inside a gene called PLAG1 and switched it on – using its own built-in regulatory elements to do it.
Encouragingly, the child remains neurologically intact.
The deeper lesson is about proof.
Vector DNA often inserts in a scrambled, rearranged mess, tumor tissue is scarce, and routine clinical labs simply don’t look for it.
In this case the team’s first four methods couldn’t reconstruct what had happened. It took long-read sequencing to finally map it.
Rare doesn’t mean impossible.
The authors make a strong case for rigor, transparency and long-term follow-up as these therapies reach more patients.”
Title: Evaluating cases of possible insertional mutagenesis after AAV gene therapy
Authors: Lindsey A George, Rebecca C Ahrens-Nicklas, Frederic D Bushman
Other posts featuring Wolfgang Miesbach on Hemostasis Today.
-
Sep 20, 2026, 13:29Factor XI Deficiency and the Challenge of Unpredictable Bleeding – EHC
-
Sep 20, 2026, 13:18Cindy Howry: FDA Approves First Treatment for Ataxia-Telangiectasia
-
Sep 20, 2026, 13:08Mohammad Salar Amoli: When Routine Coagulation Tests Miss von Willebrand Disease
-
Sep 20, 2026, 12:55Stelios Andreadis։ Human Salivary Gland Organoids Offer New Potential for Regenerative Medicine
-
Sep 20, 2026, 12:43Obi Light Ogbonnia: The Power of Lived Experience in Sickle Cell Care
-
Sep 20, 2026, 12:34Sabrine Mekni: Advancing HSCT for Children With Fanconi Anemia
-
Sep 20, 2026, 12:11Giacomo Buso: Silent Atherosclerosis Begins Earlier Than Expected
-
Sep 20, 2026, 12:0310 Posts Not to Miss This Week
-
Sep 20, 2026, 11:27Edward Lee Carter: Prasugrel Dosing Matters in P2Y12 Inhibitor Comparisons After PCI