gene editing

Mapping immunocompetent tissues for safer vaccines and gene therapy: A miRNA-based strategy for mRNA-LNP platforms

Emerging Therapies in Diabetes: New Drugs and Innovative Remedies

Diabetes mellitus is a chronic metabolic disorder with a rapidly increasing global prevalence, posing significant health and economic challenges. More than 500 million adults are currently affected worldwide, with projections indicating further increases, particularly in low- and middle-income countries. The disease ma...

Genotoxicity Evaluation of Pharmaceuticals

This chapter provides a comprehensive overview of genomic safety considerations for both small molecules and cell and gene therapies. For small chemical entities, testing is essential to identify a possible genotoxic potential early in drug development. Mutations in somatic and germline cells can lead to cancer or heri...

Post-transcriptional dysregulation precedes morphological transformation in chemically induced liver cancer

DNA lesions impede RNA polymerase II, yet the transcriptional consequences of that stress are less understood than the lesions themselves. We reanalysed total RNA and matched whole genomes from 438 liver lesions across four divergent mouse backgrounds, one of them the separate species M. caroli, 428 of them induced by...

Transposon end recognition and pairing by I-F3 CRISPR-associated transposase

To develop gene therapy tools based on CRISPR-associated transposons (CASTs), it is essential to define how transposon ends are recognized and paired during transposition. Transposon 7 (Tn7)–like transposons typically contain asymmetric left- and right-end sequences that flank and define DNA cargo. However, how the tra...

CRISPR/CAS9-based gene editing in cancer therapy

Single-allele nanoscale mapping of regulatory variants.

Millions of genetic variants are linked to human disease but identifying underlying mechanisms is challenging because most variants are noncausal and lie within the noncoding genome. We developed a Micro Capture-C variant-to-function platform (MCCv) based on analysis of single-allele chromatin structure. This can ident...

Pioneering the next generation therapies in Parkinson's disease

Gene dosage effect and reduced core fucosylation in B lymphocytes of FUT8 heterozygous mice

Core fucosylation (alpha1,6-fucosylation) of N-glycans, catalyzed solely by fucosyltransferase 8 (FUT8), plays important roles in development, immunity, and antibody effector functions. Here we report the generation of Fut8 floxed mice using a CRISPR/Cas9-based Extreme Genome Editing (EGE) system, in which loxP sites f...

A naturally evolved mutation in cavefish mc3r reshapes energy homeostasis across development in a nutrient-limited environment

Food availability in natural habitats is often unpredictable and drives the selection of adaptive strategies in organisms to survive food scarcity. However, the genetic and molecular mechanisms that enable animals to survive under such nutritional extremes are poorly understood. Naturally adapted organisms therefore pr...

Introduction: Metals in Health and Environment

Metals occupy a unique and often paradoxical position at the interface between human health and the environment. Essential metals such as iron, zinc, copper, magnesium, and molybdenum are indispensable for numerous biological processes, yet their deficiency or excess can lead to serious health consequences. In contrast...

Biohacking Our Way to Health and Transhumanism

Biohacking emerged as a grassroots movement that seeks to democratize biology, biotechnology, and healthcare. Initially known as garage biology, biohacking developed from the convergence of synthetic biology, citizen and open-source science, and advances in biotechnology. What began as amateur experimentation in home l...