CRISPR-CAS9-MEDIATED EX VIVO GENE EDITING FOR INHERITED HEMATOLOGICAL DISORDERS: ADVANCEMENTS, CHALLENGES, AND CLINICAL POTENTIAL

ATASI RANJAN PANDA; SHREEYA DAS

doi:10.22159/ijpps.2024v16i9.51048

Authors

ATASI RANJAN PANDA JSS College of Pharmacy, M. Pharm Department of Pharmaceutics, JSS Academy of Higher Education and Research, Mysuru-570015, Karnataka, India https://orcid.org/0009-0008-2369-5673
SHREEYA DAS Department of Biotechnology, University Institute of Biotechnology, Chandigarh University, Mohali, Punjab, India https://orcid.org/0009-0005-5951-6487

DOI:

https://doi.org/10.22159/ijpps.2024v16i9.51048

Keywords:

CRISPR-Cas9, Gene editing, Inherited hematological disorders, Precision medicine, Ex vivo interventions, Genetic mutations, Clinical trials, Off-target effects, Immune responses, Therapeutic advancements

Abstract

Global healthcare systems have a great challenge in the form of inherited hematological diseases, which necessitates the development of new remedial strategies. By precisely targeting inherited abnormalities, Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-CRISPR-associated protein 9 (Cas9)-mediated ex vivo gene editing has surfaced as a promising approach to treat these diseases. This review offers a comprehensive examination of the advancements, challenges, and clinical eventuality of CRISPR-Cas9-intermediated ex vivo gene editing for inherited hematological diseases. With advancements in CRISPR-Cas9 technology, the eventuality to correct inheritable mutations responsible for inherited hematological diseases is within reach. However, challenges such as off-target effects, immune responses, and ethical considerations need to be addressed for the safe and effective perpetration of this technology. A promising understanding of how CRISPR-Cas9-intermediated gene editing functions in practice is handed by ongoing clinical studies, giving rise to the possibility of advanced remedial approaches and bettered patient issues. By addressing these complications in a human-readable format, this review attempts to provide greater understanding and appreciation for the eventuality of CRISPR-Cas9 technology in revolutionizing the treatment landscape for these challenging disorders and contribute to the ongoing discussion in the field and facilitate further exploration towards effective treatments for these challenging disorders.

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