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Researchers from HuidaGene Therapeutics Co. Ltd. have evaluated the effects of adenine base editing (ABE)-induced exon skipping of exon 50 in a humanized mouse model of Duchenne muscular dystrophy (DMD).
Neuronal ceroid lipofuscinosis, commonly known as Batten disease, is an inherited pediatric neurodegenerative lysosomal storage disease caused by mutations in the CLN5 gene. The disease is incurable and there is an urgent medical need for novel therapies. A murine model of Batten disease was developed to study a novel AAV vector that expresses CLN5, AAV9-CLN5. In the study by University College London investigators, the gene therapy, driven by the synapsin promoter, was intracerebroventricularly administered into neonatal Cln5-knockout mice.
Researchers have developed a new highly effective therapeutic for pain relief by altering the chemical properties of an antinausea drug, netupitant. The modified drug is able to enter the intracellular membrane-bound endosome and target the GPCRs therein, rather than at the cell surface, that leads to optimal pain relief. The study, published in the Proceedings of the National Academy of Sciences (PNAS) on May 22,2023, was led by Nigel Bunnett, Professor and Chair of Molecular Pathobiology at NYU College of Dentistry, and illustrates how GPCR-mediated pain signaling occurs inside the endosomes rather than at the surface, highlighting the need for drugs that can reach receptors within the cells itself.
Mixed phase III study results have Bioxcel Therapeutics Inc. moving ahead to complete the clinical trial while withstanding a hammering from investors. BXCL-501 (dexmedetomidine), a sublingual film being developed to treat bipolar disorders- or schizophrenia-associated agitation, produced clinically meaningful efficacy results in part 1 of the pivotal study with half of the approved dose, but the primary efficacy endpoint was not statistically significant at two hours (p=0.077). BXCL-501 separated from placebo at four hours (p=0.049).
The researchers who enabled patients with spinal cord injuries to walk independently after implanting programmable electrodes below their lesions have now taken things one step further, restoring direct communication from the brain to the spinal cord, enabling the brain rather than an external computer to direct leg movements.
The researchers who enabled patients with spinal cord injuries to walk independently after implanting programmable electrodes below their lesions have now taken things one step further, restoring direct communication from the brain to the spinal cord, enabling the brain rather than an external computer to direct leg movements.
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