Their AAV progranulin included a C-terminal Myc tag for tracking the exogenous protein in the brain. This tag also happened to block sortilin binding. Regardless, the protein made its way into neuronal and microglial lysosomes, where it restored normal function.
According to Julia Hunt, a parenting and homemaking content creator, there are tons of unexpected places in our homes where mold is likely growing without us realizing it, and ending up directly in our mouths! Jakob Berg | Shutterstock When's the…
TGM-148 is designed to combine hemostatic efficacy with a favorable safety profile and low treatment burden Tangram’s second asset to enter the clinic in 2026 Phase 1 trial will evaluate TGM-148 in healthy volunteers, with data anticipated in 2027 TGM-148 is a first-on-target…
Cancer cells (green) are innervated with nerve cells (red). The cell nuclei (blue) are also visible. (Credit: Institute of Oral Biology/UZH) Different stem cell populations connect to nerves in distinct ways, new research shows. Innervation may therefore be crucial…
Peer-reviewed study demonstrates potent, multi-month extrahepatic delivery via both systemic administration—targeting skeletal muscle, heart, and adipose... - Peer-reviewed study demonstrates potent, multi-month extrahepatic delivery via both systemic administration—targeting skeletal muscle, heart, and adipose tissue—and localized routes across ocular,…
The testing protocol was deliberately field-like. Researchers diluted each biocide serially, incubated log-phase bacteria with the compound for ten minutes or longer, and then transferred the treated bacteria into fresh growth media to see whether any cells survived and regrew.
Dec 14, 2022, 07:00 ET AOC 1001 delivered siRNA to skeletal muscle and produced meaningful DMPK reduction in 100% of participants with a 45% mean reduction in DMPK after a single dose of 1 mg/kg or two doses of 2 mg/kg AOC 1001 produced splicing improvement of…
The landscape of CD122 therapy (targeting the beta subunit of the IL-2 and IL-15 receptors) has demonstrated growing clinical validation and increasing strategic interest for the treatment of autoimmune and inflammatory conditions such as celiac disease, eosinophilic esophagitis, graft-versus-host-disease, and vitiligo. MDNA209 is an…
AASLD The Liver Meeting® 2026 The AX-0810 Phase 1 study will be featured in two poster presentations at The Liver Meeting® 2026, the annual meeting of the American Association for the Study of Liver Diseases (AASLD), taking place November 5–9, 2026. The presentations will focus…
A preclinical nanoplatform that blocks the TRPA1 ion channel while delivering glucose starvation, chemodynamic, and sonodynamic therapy suppressed tumor growth and lung metastasis in a mouse model of triple-negative breast cancer. Article Title: TRPA1 blockade potentiates a ferroptosis-driven in situ vaccine nanoplatform…
Targets, assays, animal models, and early safety/tox studies before human trials.
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