HEMATOPOIETIC STEM CELL GENE THERAPY FOR INHERITED MONOGENIC DISEASES AND ITS IMPLICATIONS FOR FUTURE GENE THERAPY TRIALS IN TURKEY

Year 2019, , 17 - 32, 01.02.2019

Fatima Aerts-kaya , Gülen Güney Esken Özgür Doğuş Erol

https://doi.org/10.23902/trkjnat.483402

Abstract

Stem cell therapy
offers a great advantage for the development of new treatments in the field of
regenerative and restorative medicine. However, the use of stem cell therapies
and their clinical indications can even be further improved using genetic modification
of the cells. Due to the high level of consanguineous marriages in Turkey, the
country suffers from an increased frequency of inborn genetically inherited diseases.
Treatment of these diseases is difficult, since 1) diagnosis is often delayed
in rural areas, 2) distance to specialized centers may be considerable, 3)
treatment may require frequent hospital visits and 4) treatment procedures are
often both invasive and expensive.

Here, we discuss the current status of gene therapy of hematopoietic stem cells
(HSCs) for rare, inherited monogenic diseases and the advantages to use these
cells as an alternative treatment option for patients in Turkey. We discuss
results of clinical trials using retroviral and lentiviral gene therapy for the
treatment of immune deficiencies, hemoglobinopathies and several enzyme
deficiencies, new developments in the field of the HSC gene therapy to improve
safety and efficacy and recommendations for the future.

Keywords

Gene Therapy, hematopoietic stem cells, lentiviral vectors, biosafety, inherited monogenic diseases

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