Stem Cell Transplantation and Gene Therapy in Patients with Primary Immunodeficiency Diseases

immunoloji-17-1-2024

Taha Ulutan KARSa
aKonya City Hospital, Clinic of Hematology, Konya, Türkiye

Kars TU. Stem cell transplantation and gene therapy in patients with primary immunodeficiency diseases. Çölkesen F, ed. Primary Immunodeficiency Diseases in Adults. 1st ed. Ankara: Türkiye Klinikleri; 2024. p.125-8.

ABSTRACT
Hematopoietic stem cell transplantation (HSCT) and gene therapy (GT) have become important in the treatment of patients with primary immunodeficiency (PID), which has an increased incidence in countries where consanguineous marriages are common. Today, with advancing biotechnology, new genetic mutations can be identified and diagnosed at an early age. Current treatment approaches such as HSCT and GT aim to increase both life expectancy and quality of life in patients with PID. HSCT and GT performed at an early age, before disease symptoms progress and comorbidity develops, are mostly successful.

Keywords: Primary immunodeficency disease; hematopoietic stem cell transplantation; gene therapy

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