HARNESSING CRISPR-CAS TECHNOLOGIES FOR PAEDIATRIC IMMUNODEFICIENCIES: A NEW FRONTIER
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Keywords
CRISPER-Cas, Genetic defect, Paediatric, Immunodeficiencies
Abstract
Aim: This research examines the safety and effectiveness of CRISPR-Cas technologies in correcting genetic defects and restoring immunological function.
Method: This study reviewed CRISPR-Cas research on treating paediatric immunodeficiencies using PRISMA criteria.
Databases: The searches conducted in PubMed, Web of Science, and Embase primarily focused on scholarly papers published in English
Findings: Five of seventeen papers matched the requirements for more complete research after a thorough examination. Numerous studies have demonstrated that CRISPR-Cas may correct genetic abnormalities in SCID and Wiskott - Aldrich syndrome. Thus, animal models have showed considerable immune function improvements and disease amelioration. Due to a lack of clinical application data, laboratory results differ from paediatric patient outcomes.
Conclusion: In conclusion, CRISPR-Cas technology allows precise genetic changes, promising therapy for paediatric immunodeficiencies. Despite promising preclinical results, longer-term clinical trials are needed to determine safety and effectiveness. To maximise the utility of CRISPR-Cas for paediatric immunodeficiency therapies, laboratory discoveries must be translated into clinical practice.
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Humera Khatoon
Professor, Department of Pharmacology, Jinnah University for Women, Karachi, Pakistan
Javeria Javed
PhD fellow, Department of Pharmacology, Jinnah University for Women, Karachi, Pakistan
Sana Waqar
Lecturer, Institute of Pharmaceutical Sciences, Jinnah Sindh Medical University, Karachi, Pakistan
Farah Owais
Assistant Professor, Department of Pharmacology, Federal Urdu University, Karachi, Pakistan
Shomaiza Andleeb
Lecturer, Department of Pharmacy, Iqra University, Karachi, Pakistan
Javeria Khan
MPhil Fellow, Department of Pharmacology, Jinnah University for Women, Karachi, Pakistan
Maham Siddiqui
MPhil Fellow, Department of Pharmacology, Jinnah University for Women, Karachi, Pakistan
Rida Islam
MPhil Fellow, Department of Pharmacology, Jinnah University for Women, Karachi, Pakistan
Syed Fozail Sarmad
MBBS, Jinnah Postgraduate Medical Centre, Karachi, Pakistan
Wajeeha Ishrat
Lecturer, Department of Pharmacology, Federal Urdu University, Karachi, Pakistan