Original title: Gene therapy for hereditary hematological disorders: From clinical breakthroughs to future horizons
Zhenghe Chen, Wei Wang, Youlan Wu, Yawen Qiang +2 · August 2026
Inherited blood disorders like sickle cell disease, β-thalassemia, and hemophilia cause severe illness and require lifelong care. Standard treatments manage symptoms but rarely cure them. To fix this, researchers reviewed gene therapy methods that target the root genetic problems. These methods include adding working genes, directly editing DNA, or turning off faulty genes. Doctors deliver these fixes either by treating cells outside the body and returning them, or by injecting treatments directly inside patients. Gene therapies have now gained approval for hemoglobinopathiesBlood disorders affecting hemoglobin, like sickle cell disease. and hemophilia. Emerging tools like base editing, prime editing, and lipid nanoparticle delivery are also advancing. However, obstacles remain, including unwanted immune reactions, unintended genetic changes, toxic conditioning treatments, complex manufacturing, and high costs that restrict patient access to these treatments.
Imagine a factory that produces red blood cells, but the blueprint for making them has a typo. The factory keeps producing faulty cells. Normally, you'd just keep patching up the faulty cells. In this paper, the researchers describe a way to fix the typo in the blueprint itself, so the factory makes perfect cells from then on. Here, the blueprint is the patient's DNA, the factory is their blood stem cells, and fixing the typo is the gene therapy that corrects the genetic error.
For millions of people with inherited blood disorders like sickle cell disease and hemophilia, gene therapy offers a one-time treatment that could replace decades of transfusions, injections, and hospital visits. This shifts the goal from managing symptoms to fixing the root genetic cause, potentially transforming lives and reducing healthcare burdens.
The abstract notes that gene therapy still faces significant hurdles: immune reactions, unintended genetic changes, toxicity from pre-treatment conditioning, complex manufacturing, high costs, and limited access. These challenges prevent widespread adoption and mean that many patients cannot yet benefit.
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Gene therapy for hereditary hematological disorders: From clinical breakthroughs to future horizons — Zhenghe Chen, Wei Wang, Youlan Wu et al. — Molecular therapy. Nucleic acids — doi:10.1016/j.omtn.2026.103075
Abstract licence: CC BY
Explanation generated by Synthesis from the abstract — it is not the original paper.