[Nature Medicine] Pediatric ambulatory male participants with a genetic Duchenne muscular dystrophy diagnosis and receiving stable glucocorticoids received a single intravenous low-dose or high-dose fordadistrogene movaparvovec.

[Cancer Gene Therapy] Researchers proposed an original ovarian cancer treatment through the use of two recently developed modified hTERT promoters: the mutated hTERT and the chimeric hTERT-CMV.

[Nature Biomedical Engineering] Scientists demonstrated the synthesis and enzymatic stabilization of telomerase RNA component, a therapeutically relevant long non-coding RNA (lncRNA) that extends telomere length and replicative lifespan in human stem cells.

[Nature Biomedical Engineering] The authors developed a scalable cerebral cortical organoid platform by screening biocompatible polymers that prevent the fusion of organoids cultured in suspension.

[Nature Medicine] Investigators critically review the scientific advances, the clinical trials, and the various regulatory considerations that will need to be overcome for human stem cell-derived pancreatic islet-like cells to become the next cell therapy breakthrough for diabetes treatment.

[Oncogene] The authors employed an epigenetic siRNA library to identify a crucial regulator, ZMYND8, which is involved in glycolysis in pancreatic cancer cells.

[ACS Biomaterials Science & Engineering] Researchers presented a novel approach to enhance the re-endothelialization of decellularized rat liver scaffolds by seeding HUVECs in the presence of S1P, aiming to bioengineer a fully endothelialized liver.

[Madrigal Pharmaceuticals, Inc.] Madrigal Pharmaceuticals, Inc. announced that the Committee for Medicinal Products for Human Use of the European Medicines Agency has adopted a positive opinion recommending approval of resmetirom for the treatment of adults with noncirrhotic metabolic dysfunction-associated steatohepatitis with moderate to advanced liver fibrosis.

[Journal for Immunotherapy of Cancer] Scientists identified the role of Annexin A2 in the tumor-associated antigens presentation and immune evasion, which could be an actionable target in cancer immunotherapy.

[Signal Transduction and Targeted Therapy] Scientists showed that sorafenib disrupts mitochondrial function by impairing the assembly of electron transport chain supercomplexes, thereby forcing cells to shift their metabolism toward glucose as the dominant energy source.

[Nature Genetics] Investigators pinpointed the loss of an expression quantitative trait locus (eQTL) in EFHD1 during hepatocyte maladaptation associated with genotype-specific regulation by FOXO1, further contributing to the risk of metabolic dysfunction-associated steatotic liver disease.

[Autophagy] Researchers demonstrated that the natural compound acacetin (ACA) alleviated metabolic dysfunction-associated fatty liver disease (MAFLD by regulating macroautophagy/autophagy in a mouse model of rapidly induced steatohepatitis.