User: Doctor Ming, I’ve been reading about gene-edited mice lately and keep coming across “ROSA26 mice.” What exactly are they? And why do so many studies prefer the ROSA26 locus? Dr. Ming: ROSA26 mice generally refer to mice in which…
Humanized immune system (HIS) mice are the key bridge connecting in vitro experiments to clinical validation. Their core purpose is to let drugs undergo a “dress rehearsal” in an animal body equipped with human immune responses. Currently, three main construction…
In patients with advanced cancer, cachexia — a state characterised by severe weight loss, appetite loss and rapid muscle wasting — has long posed a major clinical challenge. Once regarded as a mere side effect, cachexia is now understood to…
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…
