CRISPR/Cas9 – 바이오노믹스

CRISPR/Cas9 is an cas9-guided genome-editing system that uses an sgRNA to direct precise DNA modification, and it is widely used as a programmable nuclease platform for gene knockout, correction, and knock-in. It is applied across hematology, cancer, neurodegeneration, inherited disease, reproductive biology, infectious disease, and delivery engineering, including studies in hematopoietic stem and progenitor cells, oral squamous cell carcinoma, alzheimers disease, type 1 diabetes, and parkinsons disease. Key advances include successful genome editing in 30% of HEK293 clones and 23.91% of hematopoietic stem cell clones, plus improved delivery with LuT lipid nanoparticles showing a 9.2-fold increase in editing efficiency over benchmark DOTAP SORT LNPs. Recent literature also highlights thermally gated and dual-responsive nanoplatforms for spatiotemporal control, ribonucleoprotein delivery for multiplex editing in primary human T cells, and dCas9/nCas9-derived systems for epigenetic or regulated base editing. In disease models, CRISPR/Cas9 has been used to correct atp7b H1069Q in patient-specific iPSCs, disrupt repressor sites to upregulate utrn in Duchenne muscular dystrophy, and target the hbb FSC 36/37 (-T) locus in hematopoietic stem cells. Overall, the literature emphasizes both therapeutic promise and delivery constraints, especially poor cellular uptake, off-target effects, and immune activation, while expanding applications to precision medicine and large-fragment DNA insertion.

Hematology and inherited disease

CRISPR/Cas9 was evaluated for targeting the hbb FSC 36/37 (-T) mutation locus in hematopoietic stem cells. (PMID:40676395)
Gene correction in human hematopoietic stem and progenitor cells produced comparable correction rates in NSG and NBSGW mice. (PMID:41987334)
Hemophilia gene therapy reviews describe CRISPR/Cas9 as part of genome-editing strategies integrated into treatment and targeted gene insertion. (PMID:41863244)
CRISPR/Cas9 corrected the atp7b H1069Q point mutation in patient-specific induced pluripotent stem cells. (PMID:41981235)

Cancer and immuno-oncology

CRISPR/Cas9 was discussed as a basis for precision cancer treatment and direct tumor targeting strategies. (PMID:41833894)
In oral squamous cell carcinoma, CRISPR-Cas9-mediated editing was assessed for reversing oncogenic mutations. (PMID:41837831)
CRISPR/Cas9 ribonucleoproteins were used for single and multiplex knockout in primary human T cells, including pdcd 1 and trac. (PMID:41640336)
Biomimetic nanoparticle integration was reported to enhance specificity and reduce systemic toxicity in preclinical gene-editing models. (PMID:41926291)

Neurology and neurodegeneration

CRISPR/Cas9 was reviewed as a therapeutic strategy for alzheimers disease, including modulation of apoe4, app, mapt, psen1, psen2, and trem2. (PMID:41931258)
In parkinsons disease, CRISPR/Cas systems were described as enabling precise genetic modification and disease-model generation. (PMID:41905621)
CRISPR/Cas9 was highlighted as a potentially powerful therapeutic strategy for htt in Huntington’s disease. (PMID:41849610)
Gene-editing technologies were also discussed as promising for enhancing stem cell therapy in Alzheimer’s disease. (PMID:41926312)

Delivery and platform engineering

Delivery remains a major bottleneck because of poor cellular uptake, off-target effects, and immune activation. (PMID:41863246)
LuT lipid nanoparticles improved CRISPR/Cas9 delivery, with a 9.2-fold increase in editing efficiency for lead 1A7B13 LNPs versus DOTAP SORT LNPs. (PMID:41845088)
Thermally gated and NIR-activated nanoplatforms enabled spatiotemporal control of editing, including mitochondrial DNA editing. (PMID:41774834; PMID:41584446)
Non-viral and ribonucleoprotein-based delivery approaches were emphasized for transient yet lasting therapeutic effects and in vivo engineering. (PMID:41543272; PMID:41851456)

Other disease and translational applications

CRISPR/Cas9 was used to disrupt repressor binding sites and upregulate utrn, ameliorating Duchenne muscular dystrophy. (PMID:41877484)
In type 1 diabetes, CRISPR-based β-cell replacement and immune-evasive islet engineering were highlighted as emerging strategies. (PMID:42023429; PMID:41995155)
CRISPR/Cas9 was used to generate disease models and functional knockouts, including cftr, claudin 1, alpha fetoprotein, and pdcd10. (PMID:41814120; PMID:41978949; PMID:41651174; PMID:41865434)
The technology was also discussed for infertility research, germline correction, and future vaccine applications. (PMID:41620000; PMID:41840308)