gene editing

Retinitis Pigmentosa: A Review of Its Pathogenesis and Emerging Molecular and Cellular Therapies

Retinitis pigmentosa (RP) is the most common inherited retinal dystrophy and remains largely untreatable. This review summarizes RP pathogenesis and critically appraises emerging therapeutic strategies, including gene augmentation, gene editing, RNA-based therapies, gene-agnostic and neuroprotective approaches, stem-ce...

Evolutionary Genetics of Insect Mimicry and Masquerade.

This review provides a comprehensive overview of recent progress, encompassing key insect lineages such as Lepidoptera, Hymenoptera, Odonata, and Mantodea, and highlights the pivotal roles of evolutionary hot spot genes, noncoding sequences, and structural variations in driving phenotypic diversification and adaptation...

Edited HSPCs rescue frataxin deficiency and inflammation-driven multisystemic pathology in Friedreich’s ataxia mice

Friedreich’s ataxia (FRDA) is a multisystemic neurodegenerative disorder caused by GAA repeat expansion in intron 1 of frataxin (FXN), resulting in FXN deficiency and mitochondrial dysfunction. Current therapies do not correct the underlying genetic defect. Here, we demonstrate that ex vivo CRISPR/Cas9 excision of the...

A Standalone Flavoprotein Monooxygenase Directs Divergent Tropolone Skeletal Editing through Baeyer–Villiger-Triggered Rearrangement

Late-stage oxidative remodeling of tropolone cores underpins the remarkable scaffold diversity in fungal meroterpenoid biosynthesis. However, the mechanistic basis by which a single enzymatic catalyst diverts a common substrate toward carbon-excising ring contraction versus carbon-retentive skeletal rearrangement rem...

LNP Delivery of Prime Editors Enables Durable Correction of PAH R408W in Phenylketonuria Models

Phenylketonuria (PKU) is a monogenic metabolic disorder caused by mutations in the phenylalanine hydroxylase (PAH) gene, the most common of which is c.1222C>T (p.R408W). Current treatment options, including dietary restriction and enzyme substitution, can be effective but are often challenging for patients to maintain...

Generation of Elite Ideal Rice Germplasm via CRISPR/Cas9-Mediated Multiplex Targeted Gene Editing.

Gene editing of five key negative regulatory genes, GS3 , OsBADH2 , Xa13 , Bsr‐d1 and OsSULTR3;6 , generated an elite ideal‐type rice variety mk‐10 , which exhibits a longer grain shape, enhanced tolerance to heat and alkali stress, increased 2AP content, and simultaneous resistance to Xoo , rice blas...

The Role of Immunotherapy and Gene Therapy in The Treatment of Breast Cancer in Middle-Aged Women: Integration with Pathomorphological Markers and Organ-Preserving Surgery

This article analyses modern, biologically targeted approaches to the treatment of breast cancer in middle-aged women. The main focus is on determining the pathomorphological and molecular characteristics of the tumour, the role of immunotherapy, particularly in triple-negative breast cancer, the current level of clini...

Precision Gene-Based Therapy for PFIC: Matching Platform to Disease Biology

Progressive familial intrahepatic cholestasis (PFIC) encompasses genetically heterogeneous disorders affecting bile transport, membrane organisation, epithelial polarity and bile-acid signalling. Current medical and surgical treatments can improve symptoms and disease course in selected patients, but substantial unmet...

Loss of nono impairs cardiac morphogenesis and excitation–contraction coupling via the PI3K/Akt pathway in zebrafish

Pathogenic variants in NONO are clinically linked to congenital heart disease (CHD), but in vivo mechanisms and gene-targeted interventions remain poorly explored. Using CRISPR/Cas9 gene editing and morpholino silencing, we established knockout (KO) and knockdown (KD) zebrafish models to study nono in cardiogenesis. Ad...

Has it been a crime or should it be criminalised? A case study on genome-edited babies and the improvement of criminal statutes in China

The 2018 “gene-edited babies” incident involving He Jiankui elicited widespread global condemnation, and he was subsequently convicted of and sentenced for illegal medical practice. Although several years have passed, impulses and initiatives aimed at advancing the clinical application of human germline genome editing...

The emerging role of ARE1 in the molecular regulation of nitrogen use efficiency: opportunities for genome editing

Inhibition of MHC class II CLIP binding by HLA gene editing