Publications by authors named "N J Thomsen"

Sickle cell disease (SCD) is a prevalent, life-threatening condition with few treatment options, attributed to a heritable mutation in β-hemoglobin. Therapeutic induction of fetal hemoglobin (HbF) with small molecules has been pursued as a treatment to ameliorate many disease complications but with limited success. Herein, we report the discovery of , a novel, potent, and selective molecular glue degrader of the transcription factor WIZ that robustly induces HbF expression as a potential treatment for SCD.

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  • - The study aimed to evaluate the long-term clinical and radiological outcomes of capitate fractures, with a cohort of 23 patients identified from a larger group suffering wrist pain, and a follow-up conducted after an average of 16 years.
  • - Out of the 16 eligible patients, the majority had healed capitate fractures with one case showing signs of osteoarthritis, although it did not cause any symptoms; none showed signs of avascular necrosis.
  • - Functional assessments revealed minimal impairments, with patients reporting good hand function, normal wrist movement, and strength, indicating a low risk of developing posttraumatic arthritis in the surrounding joints.
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  • - Sickle cell disease (SCD) is a serious inherited condition caused by a mutation in the β-hemoglobin gene, and increasing fetal hemoglobin (HbF) levels can help reduce complications.
  • - Researchers discovered two small molecules, dWIZ-1 and dWIZ-2, that act as molecular glue degraders to induce HbF by targeting a previously unrecognized repressor, the WIZ transcription factor.
  • - These compounds effectively triggered HbF production in animal models, suggesting that targeting WIZ for degradation offers a promising and accessible new treatment approach for SCD.
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Introduction: Diabetic peripheral neuropathy (DPN) is a common complication of both type 1 (T1D) and type 2 diabetes (T2D). No cure for DPN is available, but several potential targets have been proposed for treatment. Heat shock proteins (HSPs) are known to respond to both hyper- and hypoglycemia.

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The allosteric inhibitor of the mechanistic target of rapamycin (mTOR) everolimus reduces seizures in tuberous sclerosis complex (TSC) patients through partial inhibition of mTOR functions. Due to its limited brain permeability, we sought to develop a catalytic mTOR inhibitor optimized for central nervous system (CNS) indications. We recently reported an mTOR inhibitor () that is able to block mTOR functions in the mouse brain and extend the survival of mice with neuronal-specific ablation of the 1 gene.

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