This study investigates the safety and efficacy of lentivirus-modified hematopoietic stem cell gene therapy (HSCGT) for treating advanced symptomatic juvenile metachromatic leukodystrophy (MLD). MLD is a genetic lysosomal storage disease characterized by the dysfunction of arylsulfatase A (ARSA) or its activator prosaposin (PSAP), leading to the accumulation of sulfatides in the nervous system. The study found that HSCGT is safe and provides clinical benefits to patients with post-symptomatic juvenile MLD.

To infer gene regulatory networks (GRNs) of mixed populations in scRNA-seq data, the research team utilized the mixture Poisson lognormal (MPLN) model to analyze scRNA-seq count expression data (Figure 1).

A recent study in Engineering presents a revolutionary cotransporter system for rice harvesting and transportation. Developed by a team led by Wenyu Zhang, it features a dual-unloading mode and a novel HUT strategy, enhancing efficiency and autonomy in farming operations.

In a pivotal study, scientists have unveiled the intricate relationship between China's territorial functions and its CO₂ emissions, offering a fresh perspective on the environmental implications of regional development.