gene editing

Programmable Enzymes for Large-Cargo Genome Engineering: From Targeted DNA Integration to Therapeutic Genome Writing

Genome engineering is undergoing a conceptual transition from the correction of individual sequence variants toward the programmable installation of larger functional genetic elements. CRISPR-associated nucleases established RNA-guided genome targeting as a broadly accessible technology, while base and prime editing su...

Retrotransposons: A New Frontier in In Vivo Genome Engineering

Genome engineering has transformed cell therapy, but clinical implementation remains largely confined to ex vivo workflows followed by adoptive cell transfer. These approaches require costly manufacturing, often rely on viral vectors, and carry risks associated with unwanted genomic alterations. Recent advances in lipi...

High-Throughput Screening of a Lipid Nanoparticle (LNP) Library Identifies Lead Formulations for CRISPR-Cas9 Ribonucleoprotein Delivery and Gene Editing.

Delivery of CRISPR-Cas9 ribonucleoprotein (RNP) using synthetic materials can bypass the host translational machinery and limit the duration of genomic exposure, minimizing the risk of off-target mutations. Despite their advantages, lipid nanoparticle (LNP) platforms for CRISPR-Cas9 RNP delivery that achieve highly eff...

Ocular Gene Therapy in China: Technological Framework, Clinical Translation, and Future Perspectives.

This review summarizes the technological landscape and representative clinical advances of ocular gene therapy in China, highlighting the evolution from established gene-replacement approaches toward increasingly differentiated therapeutic strategies and key challenges in interpreting clinical benefit and translating e...

Emerging stem cell strategies for neurodegenerative diseases.

Degenerative diseases continue to impose a substantial global health burden with limited curative options. Neurodegenerative diseases, the principal focus of this review, are among the largest contributors to this burden. Stem cell therapies have emerged as a promising approach, offering the potential to replace lost c...

Female iPSC X chromosome inactivation erosion and its transcriptomic effects during CRISPR gene editing and neural differentiation.

Variable XCI is found in CRISPR-edited female iPSCs, which was largely preserved in iPSC-derived neurons and predominantly influenced X-linked genes, which has significant implications in hiPSC-based modeling of neurodevelopmental disorders.

Technology of the future: a call for co‐development of gene editing tools in wildlife management

Precision gene editing in infant neuroblastoma: a review of nanoparticle: enabled CRISPR/Cas9 therapies

Emerging preclinical findings support the feasibility of nanoparticle-mediated CRISPR/Cas9 delivery, although direct evidence in infant neuroblastoma remains limited and the evidence for nanoparticle-mediated CRISPR/Cas9 therapy remains largely preclinical.

Molecular prevalence and genetic diversity of Blastocystis spp. in patients with irritable bowel syndrome and inflammatory bowel disease: a case–control study in southwestern Iran

The clinical significance of Blastocystis spp. in irritable bowel syndrome (IBS) and inflammatory bowel disease (IBD) remains uncertain. This study investigated its molecular prevalence, subtype (ST) distribution, and phylogenetic characteristics among patients with IBS or IBD and healthy controls in southwestern Iran....

Bioethics in assisted reproduction and embryology

The use of assisted reproductive therapies, advances in embryo research, and developments in scientific fields such as gene editing and in vitro gametogenesis have attracted the attention of bioethicists for years. On one side, the 14-day rule has faced criticism from embryo researchers advocating for its extension. On...

PolyFab: an efficient in vitro pathway assembly tool for natural product synthesis in Saccharomyces cerevisiae

Saccharomyces cerevisiae is a widely used industrial chassis for the heterologous biosynthesis of natural products. However, achieving high-level expression of heterologous genes and developing efficient metabolic engineering strategies remain major challenges. Here, based on the polycistronic expression system and the...

Comparative mitochondrial genomics of the medicinal mushroom Phellinus tuberculosus reveals conserved core genes and dynamic genome evolution within Hymenochaetaceae

Phellinus species are well-known white-rot fungi belonging to the Hymenochaetaceae family. Phellinus species contain medicinally functional compounds and nutrients. However, a comprehensive genus-level analysis of their mitochondrial genomes remains unavailable to date. The complete mitochondrial genome of P. tuberculo...