Durable reprogramming of human T cells may now be possible thanks to a new technique based on the CRISPRoff and CRISPRon methodology. Researchers from the Arc Institute, Gladstone Institutes, and the University of California San Francisco (UCSF) have stably silenced or activated genes in this type of immune cell without cutting or altering its DNA, making T cells more resistant, active, and effective against tumors.
Genescience Pharmaceuticals Co. Ltd. has discovered new tyrosinase (TYR) inhibitors acting as melanin inhibitors potentially useful for the treatment of skin hyperpigmentation.
Alterome Therapeutics Inc. has identified new RAC-α serine/threonine-protein kinase (AKT1; PKB-α) E17K mutant)inhibitors reported to be useful for the treatment of cancer.
Onkure Therapeutics Inc. has disclosed new phosphatidylinositol 3-kinase (PI3K) inhibitors reported to be useful for the treatment of cancer, congenital lipomatous overgrowth, vascular malformations, epidermal naevi and skeletal abnormalities and scoliosis (CLOVES syndrome) and PIK3CA-related overgrowth spectrum.
Excessive signaling by platelet-derived growth factor receptor (PDGFR)-α, a transmembrane tyrosine kinase, helps drive the rapid angiogenesis that many types of tumor cells need in order to proliferate and metastasize.
H. Lundbeck A/S and Contera Pharma A/S have entered into a strategic research collaboration designed to accelerate the discovery and development of innovative oligonucleotide-based medicines for serious neurological conditions with unmet needs.
Proqr Therapeutics NV has received clinical trial application (CTA) authorization under the EMA’s new centralized review process for a phase I study of AX-0810, which is being developed for the treatment of cholestatic diseases such as primary sclerosing cholangitis and biliary atresia.
Aniridia-associated keratopathy (AAK) is a rare genetic eye condition caused by PAX6 haploinsufficiency, which leads to chronic inflammation, neovascularization and vision loss. Currently, there are limited therapeutic options for the treatment of AAK, which has been linked to limbal stem cell deficiency.
Hypoxia-inducible factors (HIFs) play a central role in how retinal cells respond to low oxygen, but their chronic activation has been linked to the development of age-related macular degeneration (AMD) and related retinal disorders.
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