Gene therapy for hereditary hematological disorders: From clinical breakthroughs to future horizons.

Gene therapy has achieved clinical breakthroughs for hereditary hematological disorders, with approved treatments for hemoglobinopathies and hemophilia, moving toward curative potential.

• Why it matters: Hereditary hematological disorders cause significant health burdens and lack fully curative treatments, creating a critical need for innovative therapies that target genetic roots rather than supportive care.
• What they did: The review covers current gene therapy strategies—including gene addition, editing, and silencing—using ex vivo and in vivo delivery methods, highlighting recent clinical successes and emerging technologies like base and prime editing.
• The result: Advances in gene therapy are shifting from experimental to durable, potentially curative options, though challenges like immune responses and manufacturing hurdles remain, with future efforts focusing on improving safety, efficiency, and accessibility.