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Gene Therapy in Hematology

Gene therapy and gene-editing approaches for hematologic disease

Written by: Saygı Hospital Health Guide Editorial Board
Last updated:

This content has been compiled by the Saygı Hospital Health Guide Editorial Board and is periodically reviewed by a specialist physician.

This content is for informational purposes only and does not constitute medical advice. You can book an appointment at our Hematoloji department. Book Appointment →

What is Gene Therapy in Hematology?

Gene therapy in hematology covers innovative approaches that aim to correct inherited blood diseases at the genetic level. Lentiviral vector-based gene addition and CRISPR-Cas9 gene editing are the two principal technology platforms in this field.

Exagamglogene autotemcel (exa-cel, CASGEVY) is the first gene-editing therapy approved by the FDA. It uses CRISPR-Cas9 to modify the BCL11A erythroid enhancer, increasing fetal hemoglobin in sickle cell disease and transfusion-dependent beta thalassemia. Lovotibeglogene autotemcel (lovo-cel, LYFGENIA) is a lentiviral vector-based gene therapy approved for sickle cell disease.

In hemophilia A, valoctocogene roxaparvovec uses an AAV5 vector to deliver the FVIII gene, and in hemophilia B, etranacogene dezaparvovec delivers the FIX gene with AAV5. These therapies require myeloablative conditioning and an autologous transplant process, and long-term safety follow-up is ongoing.

Symptoms

Gene therapy is a treatment modality, not a symptom-driven indication
Recurrent pain crises in sickle cell disease
Chronic transfusion dependence in thalassemia
Recurrent bleeding episodes in hemophilia A or B
Inadequate response to conventional therapy

Risk Factors

Infertility risk from myeloablative conditioning
Insertional mutagenesis risk with lentiviral vectors
Pre-existing neutralizing antibodies against AAV vectors
Long-term safety data are still limited

When to See a Doctor?

If you experience any of the following symptoms, seek medical attention promptly:

  • Severe sickle cell disease or beta thalassemia major
  • Severe hemophilia A or B
  • Insufficient benefit from conventional therapy

Treatment Methods

01
CASGEVY (exagamglogene autotemcel) for sickle cell and beta thalassemia
02
LYFGENIA (lovotibeglogene autotemcel) for sickle cell disease
03
Valoctocogene roxaparvovec for hemophilia A
04
Etranacogene dezaparvovec for hemophilia B
05
Myeloablative conditioning, often busulfan-based
06
Long-term monitoring for insertional mutagenesis and hematologic malignancy

Which Department to Visit?

You can visit our Hematoloji department for these complaints. Our specialist physicians will create the most suitable treatment plan for you.

Learn About Hematoloji Department

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You can make an appointment with our specialists or contact us for your concerns.

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Health Disclaimer: The information on this page is prepared for general informational purposes only. It does not replace medical diagnosis and treatment. Please consult your physician for your complaints. Saygı Hospital does not accept responsibility for actions taken based on the information on this page.