The editing in human cells and in mice of the survival motor neuron 1 gene (SMN1) restored the levels of SMN protein that the mutation of the SMN2 gene produces in spinal muscular atrophy (SMA). Scientists from the Broad Institute in Boston and The Ohio State University reversed the mutation using the base editing technique. “This base editing approach to treating SMA should be applicable to all SMA patients, regardless of the specific mutation that caused their SMN1 loss,” the lead author David Liu, a professor and director of the Merkin Institute of Transformative Technologies in Healthcare at the Broad Institute of Harvard and MIT, told BioWorld.
A live-attenuated vaccine targeting SARS-CoV-2 infection, which can be administered through the nose, has shown promise in preclinical animal studies carried out by researchers in Berlin. In an article published April 3, 2023, in Nature Microbiology, the authors reported that the COVID-19 vaccine candidate – sCPD9 – triggered the most robust immune response in a hamster model when compared with Biontech/Pfizer’s mRNA vaccine BNT162b2 and Ad2-Spike.
Astrazeneca has described spirocyclic compounds acting as protein arginine N-methyltransferase 5 (PRMT5) inhibitors reported to be useful for the treatment of cancer.
Researchers at Dana-Farber Cancer Institute Inc., Memorial Hospital for Cancer and Allied Diseases, Memorial Sloan Kettering Cancer Center and Stevens Institute of Technology have divulged proteolysis targeting chimeras (PROTACs) comprising an E3 ubiquitin ligase-binding moiety linked to cyclin-dependent kinase 4 (CDK4) and/or 6 (CDK6)-targeting moiety through a linker.
Nutshell Biotech (Shanghai) Co. Ltd. has identified fused ring compounds acting as fibroblast growth factor receptor 4 (FGFR4) inhibitors reported to be useful for the treatment of cancer and myeloproliferative diseases.
Medshine Discovery Inc. has synthesized PROTAC compounds comprising an E3 ubiquitin ligase binding moiety coupled to IRAK-4-targeting moiety through a linker.
Shanghai Haihe Biopharma Co. Ltd. has disclosed hydroxamic acid compounds acting as ENPP1 inhibitors reported to be useful for the treatment of cancer and infections.
Gliotransmitter release by astrocytes depends on calcium dynamics and is an essential regulator of brain functions. Alterations of calcium homeostasis have been observed in neurons and astrocytes from mouse models of Alzheimer’s disease (AD). A new longitudinal study in the PS2APP mouse model of AD suggests a possible link between calcium dynamics in astrocytes and memory loss.
Congenital anomalies of the kidneys and urinary tract (CAKUT) remain the main cause of chronic kidney disease before the age of 25 years, and account for about 40% of childhood end-stage renal diseases. Studies in Xenopus species have shown ENPP6 knockdown to lead to impaired pronephros development, and mutations in the ENPP6 paralogue PIGN gene have been tied to CAKUT.
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