Genome-wide CRISPR screening identifies cellular factors controlling nonviral genome editing efficiency

Genome-Wide CRISPR Screening Identifies Cellular Factors Controlling Nonviral Genome Editing Efficiency

A novel genome-wide CRISPR screening strategy that will facilitate the systematic engineering of novel nonviral genome editing delivery methods, where the identified novel gene hits can be further used to increase editing efficiency for other therapeutically relevant cell types.

In vivo delivery strategies for therapeutic CRISPR genome editing

A comprehensive review of delivery modalities of CRISPR systems solely in vivo that underpin their therapeutic translation and outlines the remaining barriers to durable, tissue-selective, and broadly deployable CRISPR therapeutics is provided.

CRISPR/Cas9-Based Genome Editing: Understanding Differences in DNA Repair Pathways, Profiles, and Outcomes

Over a decade of advances in Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) and CRISPR-associated protein 9 (Cas9)-based technologies have culminated in the first-ever FDA-approved CRISPR/Cas-based therapy. Aside from this approved therapy for sickle cell anemia, several CRISPR/Cas-based therapies are currently under development or testing for a range of chronic diseases, including viral diseases like human immunodeficiency virus type 1 (HIV-1) infection, genetic diseases like familial hypercholesterolemia, and cancer. The success of these therapies hinges on the effective delivery of CRISPR/Cas9 components to target regions, efficient Cas endonuclease editing, repair profiles generated, and their resulting outcomes. Here, we discuss the factors that influence the generation of CRISPR/Cas9-generated repair edits, the overall profiles, and outcome prediction(s), as well as the analytical tools that have been developed to date. Finally, how this technology has been used towards a functional HIV-1 cure is discussed.

A comprehensive review of CRISPR-Cas9-mediated genome editing in Leishmania strains: methodologies, applications, challenges and future directions

CRISPR-Cas9 Technology in Genetic Disorders: Genome Editing Approaches and Therapeutic Applications – A Review

The potential application of CRISPR technology for the possible management of geneticbased conditions, including sickle-cell anemia, β-thalassemia, cystic fibrosis, and Duchenne muscular dystrophy is described.

Analytical dissection of CRISPR-Cas9-mediated gene editing fidelity: integrating next-generation bioanalytical platforms for precision immunotherapeutics and rare disease correction

The clinical translation of CRISPR- Cas9 therapeutics requires rigorous and biologically grounded evaluation of genome editing fidelity that extends beyond conventional measures of on-target efficiency and canonical off-target detection. Existing fidelity assessment strategies often capture limited aspects of editing outcomes and do not fully account for context-dependent biochemical, epigenetic, and cellular heterogeneity that can influence therapeutic safety and efficacy. This review focuses on analytical and translational frameworks for CRISPR fidelity assessment, with emphasis on the strengths and limitations of current bioanalytical platforms. Recent advances integrating orthogonal technologies including digital droplet PCR, genome-wide off-target mapping approaches (e.g., CIRCLE-seq and GUIDE-seq), and single-cell proteogenomic profiling enable more comprehensive characterization of editing outcomes across molecular and functional dimensions. Applications in edited T cells targeting immune checkpoints and in hematopoietic stem cell correction models illustrate how multi-layered analyses can reveal context-dependent variability and previously undetected off-target effects. Emerging approaches, including integrative scoring frameworks and AI-assisted analytical models, are discussed in an exploratory context, with emphasis on current limitations, validation requirements, and challenges in standardization. In addition, ongoing efforts toward minimally invasive monitoring strategies are considered with respect to their potential, as well as their current constraints. Despite significant progress, major challenges remain in assay harmonization, cross-platform comparability, and translation to clinically actionable frameworks. Accordingly, CRISPR fidelity is best understood as a multi-dimensional biological phenotype that requires careful, context-specific evaluation. This perspective provides a balanced foundation for advancing genome editing toward safer and more reliable therapeutic applications.