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Papers, explained in our own words

Every entry summarises what the study set out to test, what it found and why it changes how we design delivery systems. Browse research and reviews or search the collection.

586 articles

Showing 1–3 of 3 articles · Keyword: CRISPR/Cas9 Clear keyword

Current drug targets2026ResearchNon-viral Gene Delivery

1. CRISPR/Cas9-based Genome Editing in Inherited Diseases: Mechanisms, Therapeutic Advances, Safety and Clinical Translation

Fatima J, Siddique Yh

Gene therapy has transformed modern biomedical research by enabling curative strategies that target the molecular basis of disease. Among genome-editing technologies, the CRISPR/Cas9 system has emerged as a versatile and efficient platform due to its programmability, simplicity, and precision. This review summarizes…

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Applied microbiology and biotechnology2026ResearchNon-viral Gene DeliveryDrug Delivery

2. T cell engineering by targeted small extracellular vesicles delivering CRISPR/Cas9 system for PD-1 knock-out

Farahani Ms, Shamsara M, Moazzeni Sm, Khalili S, Johnson B, Darzi L, Et Al.

Among the emerging cancer therapies, immunotherapy using T cell engineering stands out as one of the most promising approaches. While the clustered regularly interspaced palindromic repeats (CRISPR/Cas) system is widely recognized as one of the most common gene-editing system tools, the main challenge for in vivo…

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Genome medicine2026ResearchNon-viral Gene Delivery

3. Phenotype rescue through the restoration of full-length dystrophin using CRISPR/Cas9 genome editing in Duchenne muscular dystrophy patient-derived iPSCs carrying the deletion of two exons

Rovina D, Milasi S, Di Giuseppe G, Pioner Jm, Langione M, Giammarino L, Et Al.

Duchenne muscular dystrophy (DMD) is a X-linked disease affecting skeletal and cardiac muscle and is caused by mutations in the dystrophin gene (DMD). Patient-derived induced pluripotent stem cells (iPSCs) serve as reliable in vitro disease models. Their genetic correction by CRISPR/Cas9 allows the generation of…

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