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

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dc.contributor.authorPANDA, ARen_US
dc.contributor.authorDAS, S.en_US
dc.date.accessioned2025-05-12T07:20:51Z
dc.date.available2025-05-12T07:20:51Z
dc.date.issued2024-09
dc.description.abstractGlobal 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.en_US
dc.identifier.affiliationsJSS College of Pharmacy, M. Pharm Department of Pharmaceutics, JSS Academy of Higher Education and Research, Mysuru-570015, Karnataka, Indiaen_US
dc.identifier.affiliationsDepartment of Biotechnology, University Institute of Biotechnology, Chandigarh University, Mohali, Punjab, Indiaen_US
dc.identifier.citationPANDA AR, DAS S.. CRISPR-CAS9-MEDIATED EX VIVO GENE EDITING FOR INHERITED HEMATOLOGICAL DISORDERS: ADVANCEMENTS, CHALLENGES, AND CLINICAL POTENTIAL . International Journal of Pharmacy and Pharmaceutical Sciences . 2024 Sep; 16(9): 1-7en_US
dc.identifier.issn0975-1491
dc.identifier.issn2656-0097
dc.identifier.placeIndiaen_US
dc.identifier.urihttps://imsear.searo.who.int/handle/123456789/246463
dc.languageenen_US
dc.publisherInnovare Academic Sciences Pvt. Ltd.en_US
dc.relation.issuenumber9en_US
dc.relation.volume16en_US
dc.source.urihttps://doi.org/10.22159/ijpps.2024v16i9.51048en_US
dc.subjectCRISPR-Cas9en_US
dc.subjectGene editingen_US
dc.subjectInherited hematological disordersen_US
dc.subjectPrecision medicineen_US
dc.subjectEx vivo interventionsen_US
dc.subjectGenetic mutationsen_US
dc.subjectClinical trialsen_US
dc.subjectOff-target effectsen_US
dc.subjectImmune responsesen_US
dc.subjectTherapeutic advancementsen_US
dc.titleCRISPR-CAS9-MEDIATED EX VIVO GENE EDITING FOR INHERITED HEMATOLOGICAL DISORDERS: ADVANCEMENTS, CHALLENGES, AND CLINICAL POTENTIALen_US
dc.typeJournal Articleen_US
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