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https://www.selleckchem.com/products/2-deoxy-d-glucose.html Two SCID-X1 neonatal canines treated with this approach achieved long-term therapeutic immune reconstitution with no prior conditioning. Therapeutic levels of gene-corrected CD3+ T cells were demonstrated for at least 16 months, and all other correlates of T cell functionality were within normal range. Retroviral integration-site analysis demonstrated polyclonal T cell reconstitution. Comparative analysis of integration profiles of foamy viral (FV) vector and cocal LV vector after in vivo gene therapy found distinct integration-site patterns. These data demonstrate that clinically relevant and durable correction of canine SCID-X1 can be achieved with in vivo delivery of cocal LV. Since manufacturing of cocal LV is similar to VSV-G LV, this approach is easily translatable to a clinical setting, thus providing for a highly portable and accessible gene therapy platform for SCID-X1. Atopic dermatitis (AD) is a common and debilitating dermatosis that often impacts the physical and psychological quality of life in children and adults. A limited number of treatment options are available for AD, and often these treatments result in an insufficient response or may be contraindicated for some patients. This treatment gap creates an increasing demand for alternative AD therapies. The Janus kinase (JAK)-signal transducers and activators of transcription (STAT) pathway is known to play a critical role in the dysregulation of immune responses in AD. Inhibition of the JAK enzymes in the JAK-STAT pathway has shown potential for the treatment of this chronic skin condition. We review the evolving efficacy and safety profile of abrocitinib, an oral JAK1 inhibitor, in the treatment of AD based on the data available from phase I, II, and III clinical trials. Evidence supports clinical efficacy, improved pruritus and an acceptable safety profile, making abrocitinib a viable alternative to conventional AD therapies. Pivotal phas
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