Gene therapy for sickle cell and thalassaemia
CRISPR-based and lentiviral gene therapies for haemoglobin disorders.
Papers per year
Each bar is 12 months; the last one ends 17 Oct 2026. New trials in the same years: 10, 10, 16, 5, 8.
New guidelines and consensus this year
- Conditioning regimen for hematopoietic cell transplant in transfusion dependent thalassemia: Recommendations from the EBMT practice harmonisation and guidelines committeeBone Marrow Transplant2026 Oct
- Gene Therapy for Sickle Cell Disease: Practice Recommendations from the American Society for Transplantation and Cellular Therapy and the International Society for Cell & Gene TherapyTransplant Cell Ther2026 Jul
All papers from the last 12 months
238 papers (18 Oct 2025 – 17 Oct 2026), newest first. Loading the full list…
- Gene therapy for hereditary hematological disorders: From clinical breakthroughs to future horizonsMol Ther Nucleic Acids2026 Dec 8PMID 42761173Review
- Cost-effectiveness of exagamglogene autotemcel gene-edited therapy in patients with transfusion-dependent β-thalassemia in the United StatesJ Med Econ2026 DecPMID 41955126
- Cost-effectiveness of exagamglogene autotemcel gene-edited therapy in patients with sickle cell disease with recurrent vaso-occlusive crises in the United StatesJ Med Econ2026 DecPMID 41730016
- Epigenetic strategies for fetal hemoglobin induction in sickle cell diseaseEur J Med Chem2026 Nov 15PMID 42551134Review
- Sickle cell disease and hematopoietic stem cell transplantation: donor expansion, gene-modified grafts and prenatal horizonsCurr Opin Hematol2026 Nov 1PMID 42710047Review
- 2026 Update on Clinical Trials in β-ThalassemiaAm J Hematol2026 NovPMID 42584024Review
- Efficacy, Safety, and Treatment-Delivery Feasibility of Autologous Gene Therapy for Sickle Cell Disease: A Systematic Review With Descriptive Synthesis of Clinical TrialsEur J Haematol2026 NovPMID 42400217Systematic review
- Translating base editors: a decade of progress from the bench to the clinicNat Rev Drug Discov2026 Oct 16PMID 42888260Review
- Precision Medicine in Sickle Cell Disease: From Molecular Pathways to Curative StrategiesHemoglobin2026 Oct 15PMID 42876272Review
- Bibliometric analysis of the research trends of thalassemia in the past decadeExpert Rev Hematol2026 Oct 15PMID 42865314
More hematology trends
All specialties →Bispecific antibodies in myeloma
Teclistamab, elranatamab and talquetamab in multiple myeloma.
Measurable residual disease
Detecting residual disease after treatment of blood cancers.
CAR-T in blood cancers
Chimeric antigen receptor T-cell therapy in lymphoma, myeloma and leukaemia.
Key point: CAR T-cell therapy has emerged as an effective option for refractory or progressive mantle cell lymphoma after prior covalent BTK inhibitor treatment.
Immune thrombocytopenia
ITP and its newer treatments.
Key point: Adult ITP care incorporates newer thrombopoietin receptor agonists and small molecule kinase inhibitors, emphasizing thrombotic risk and quality of life.
DOACs in cancer-associated thrombosis
Direct oral anticoagulants for venous thromboembolism in people with cancer.
Myelofibrosis treatment
JAK inhibitors and newer drugs in myelofibrosis.
Key point: Ruxolitinib-based combination regimens showed improved spleen and symptom responses with acceptable safety, with efficacy significantly influenced by prior JAK inhibitor exposure.
Too many papers to read?
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How these numbers are counted
PubMed query, searched in titles and abstracts by publication date:
("sickle cell"[tiab] OR thalassemia[tiab] OR thalassaemia[tiab]) AND ("gene therapy"[tiab] OR "gene editing"[tiab] OR CRISPR[tiab] OR exagamglogene[tiab] OR lovotibeglogene[tiab])Trials: ClinicalTrials.gov studies first submitted in each 12-month window that match the same terms. A query can miss synonyms or catch unrelated papers, so treat counts as approximate.