I. From Gene Editing to Aging‑Related Phenotypes: Why Choose LMNA? Gene‑edited mice employ molecular‑biology, genetic and embryo‑manipulation techniques to precisely modify target genes, generating genetic‑altered animals with gene‑knockout (KO), gene‑knock‑in (KI), conditional knockout (cKO), point‑mutation or base‑editing modifications. Compared with…
Induced pluripotent stem cells (iPSCs) are generated by reprogramming differentiated somatic cells back into pluripotent stem cells. They open new avenues for drug development and supply valuable cellular resources for cell‑replacement therapy. Pluripotent stem cells (PSCs) are mainly derived from…
Apolipoprotein E (APOE) is a star target in lipid‑metabolism and neurodegenerative‑disease research. Located on human chromosome 19, it encodes a secreted glycoprotein that maintains two independent ApoE pools in the periphery (liver‑derived) and the central nervous system (brain‑derived) respectivelyFrontiers。 Three major…
Autoimmune Encephalitis (AE) is a rare neuro‑immune disorder characterised by central‑nervous‑system inflammation driven by dysregulated autoimmune responses. The immune system fails to distinguish self‑antigens from foreign threats and generates autoantibodies (alongside T‑cell responses) targeting neuronal surface or intracellular antigens. These…
On August 19, 2026, blockbuster news swept the biotech community: Moderna and Merck announced that Intismeran autogene (mRNA‑4157/V940), a personalized mRNA cancer vaccine, in combination with Keytruda for adjuvant treatment of high‑risk completely resected stage III/IV melanoma met the primary endpoint of recurrence‑free…
In recent years, gene‑editing and AAV‑based gene therapies have frequently made headlines, hailed as beacons of hope for curing genetic diseases. What many overlook, however, is that before these cutting‑edge therapies advance to human trials, they must repeatedly pass a…
Biotinidase Deficiency (BTD) is an autosomal‑recessive inherited metabolic disorder. It is caused by pathogenic variants in the BTD gene that reduce or abolish biotinidase activity, impairing the recycling of free biotin. Officially listed in China’s First National Rare Disease Catalogue…
IgG4-related disease (IgG4-RD) is a chronic progressive fibrosing inflammatory disorder that received a unified international nomenclature in 2011. Capable of mimicking malignant tumors and involving nearly all visceral organs, it has become a core research focus in rheumatology and pathology.…
In July 2026, Nature Reviews Drug Discovery published the article Novel drug targets in 2025. According to its data, among the 94 innovative medicines approved by the world’s four major drug regulatory authorities in 2025, 11 targets represented globally first-in-class…
The global population living with type 2 diabetes mellitus (T2DM) has reached 590 million, and the figure is projected to rise to 700 million by 2045. Meanwhile, obesity remains a prevalent pandemic; over 30% of the population will be overweight…
