چکیده مقاله
Sickle cell disease SCD is a non inherited disease caused by a point mutation of the globin gene HBB , resulting in abnormal hemoglobin S production HbS The CRISPR Cas9 system has been developed as an innovative tool for precise genome editing and offers potential therapeutic strategies for SCA This technology allows targeted correction of the HBB mutation or the induction of fetal hemoglobin HbF to compensate for the failure of the HbS Recent studies have shown that CRISPR Cas9 can efficiently edit the hematopoietic stem cells HSCs to restore normal globin expression or to disrupt HbF suppressors such as BCL11A Preclinical studies have shown promising results, with the HSC successfully engrafted and producing healthy red cells in animal models Furthermore, CRISPR based ex vivo therapies reduce risks beyond target compared to in vitro approaches Despite challenges such as delivery efficiency and long term safety, clinical advances such as ongoing CRISPR SCA studies highlight its transformative potential This review discusses the molecular mechanisms of CRISPR Cas9 in SCA therapy, current research progress and future directions to translate this technology into therapeutic therapies
کلیدواژهها
نویسندگان
Pouria Jalali
Faculty of Medicine, Shahid Sadoughi University of Medical Sciences, Yazd, Iran
Mohammad Mahdi Amini
Department of Medical Laboratory Science, Kashan Branch, Islamic Azad University, Kashan, Iran
شیوه ارجاع
Jalali, Pouria and Amini, Mohammad Mahdi,1404,The Application of CRISPR-Cas9 in Sickle Cell Disease Treatment: From Molecular Mechanisms to Clinical and Ethical Challenges,5th World Congress of new findings in Health ,Health Sciences and Educational Sciences
ارائهشده در
مجموعه مقالات پنجمین کنگره جهانی یافته های نوین در سلامت، علوم بهداشتی و علوم تربیتی31 تیر 1404