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    Abstract Final

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    Abstract Final

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    Title: CRISPR-based Drug Development: Revolutionizing Personalized Medicine

    Author(s): Nitesh Kumar , Abhishek Verma , Ayushi Gupta , Naveen Maurya


    Affiliation(s): Department of Biotechnology , Seth Vishambhar Nath Institute of Engineering
    & Technology , Safedabad, Barabanki , Lucknow , Uttar Pradesh , 225003
    Abstract:
    The CRISPR-Cas9 system, originally a bacterial defense mechanism, has
    emerged as a revolutionary tool in genetic engineering, offering unprecedented
    precision in genome editing. This study presents real-world experimental
    findings on using CRISPR-Cas9 for drug development targeting specific genetic
    mutations in cancer and monogenic disorders.
    Our research focused on creating CRISPR-based therapeutic models for Sickle
    Cell Anemia and KRAS-mutated cancers. Using patient-derived induced
    pluripotent stem cells (iPSCs), we performed targeted edits on the HBB gene,
    correcting the SCD mutation. Post-editing, differentiation into erythroid cells
    confirmed restored hemoglobin functionality via high-performance liquid
    chromatography (HPLC).
    For KRAS-mutated cancers, CRISPR-Cas9-mediated knockouts of the mutant
    KRAS allele in pancreatic cancer cell lines reduced tumorigenicity. Functional
    assays, including colony formation and apoptosis detection, demonstrated a
    45% decrease in oncogenic activity. Whole-genome sequencing validated
    minimal off-target effects, while lipid nanoparticles (LNPs) facilitated efficient
    delivery in vivo.
    To enhance CRISPR precision, our study employed prime editing for single-base
    corrections, achieving a 70% editing efficiency in vitro. These approaches
    underscore the potential of CRISPR in accelerating drug discovery pipelines by
    directly addressing disease-causing mutations.
    This research highlights the transformative potential of CRISPR-based therapies
    in personalized medicine. Challenges such as delivery mechanisms, immune
    responses, and regulatory hurdles are discussed alongside future prospects of
    integrating CRISPR with AI-driven drug design. Our findings support CRISPR as a
    cornerstone technology in shaping the future of targeted therapies and
    precision healthcare.
    Keywords: CRISPR, Sickle Cell Anemia, KRAS, Gene Editing, Precision Medicine

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