Here, the authors discuss how patient-derived organoids should be grown and how advanced genome-editing tools may be applied to research on modeling of cancer and infectious diseases. They also highlight practical applications of organoids ranging from basic studies to drug screening and precision medicine.
In this sutdy, the authors describe an easy, quick and cost-effective fluorescent polymerase chain reaction (PCR)-based method, CRISPR Somatic Tissue Activity Test (CRISPR-STAT), to determine target-specific efficiency of sgRNA. They validated the method using CRISPR/Cas9 and zebrafish which could be applied to other model systems and other genome targeting nucleases.
Recently, a simple and inexpensive liver-specific transgenic approach was developed, in which a hydrodynamics-based transfection (HT) method was coupled with the Sleeping Beauty transposase system. Various HT models in which different oncogenic pathways are activated and/or tumor-suppressing pathways inactivated have been developed in recent years. The applicability of HT models in liver cancer research is expected to broaden and ultimately elucidate the cooperation between oncogenic signaling pathways and aid in designing molecular therapy to target altered pathways.
The Sleeping Beauty Transposon Vector System for Treatment of Rare Genetic Diseases: An Unrealized Hope?
BigField GEG Tech's insight:
In this review, diseases affecting the blood system, the connective tissue, the immune system, the metabolism, and the nervous system and their treatment utilizing the SB transposase system will be discussed. Moreover, advantages and disadvantages of SB transposase-based gene therapeutic approaches will be mentioned.
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Here, the authors discuss how patient-derived organoids should be grown and how advanced genome-editing tools may be applied to research on modeling of cancer and infectious diseases. They also highlight practical applications of organoids ranging from basic studies to drug screening and precision medicine.