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chromatin remodeling in pericentral hepatocytes modulates mash through cyp450 activity
Original title: “Chromatin remodeling in pericentral hepatocytes modulates MASH through CYP450 activity.”
epidemiological characteristics and treatment patterns of alport syndrome in korea
Original title: “Epidemiological characteristics and treatment patterns of Alport syndrome in Korea.”
surgical management of necrotizing pneumonia in pediatric patients a retrospective analysis in a third level center
Original title: “Surgical Management of Necrotizing Pneumonia in Pediatric Patients. A Retrospective Analysis in a Third-Level Center.”
of hoofbeats horses and zebras a conversation with drs william dock theodore woodward and alvan feinstein
Original title: “Of Hoofbeats, Horses, and Zebras: A "Conversation" with Drs. William Dock, Theodore Woodward, and Alvan Feinstein.”
Convergent Therapeutics' Dr. Philip Kantoff and Plus Therapeutics' Dr. Marc Hedrick discuss how unmet medical needs, maturing isotope supply chains and innovative delivery are positioning targeted radiation as oncology's next big platform.
Original title: “The Brave New World of Radiotherapeutics”
Durable expression of anti-sickling fetal hemoglobin and reduction in sickle hemoglobin observed In patients with sickle cell disease treated with ristoglogene autogetemcel, mean anti-sickling fetal hemoglobin levels were above 60% with a mean durable reduction in corresponding sickle hemoglobin below 40%. The median time to neutrophil and platelet engraftment was relatively short -- 17.5 days and 19 days, respectively. No patients had an investigator-reported severe vaso-occlusive crisis after engraftment. Treatment with the investigational base-editing gene therapy ristoglogene autogetemcel (risto-cel) showed promise in patients with sickle cell disease, according to an interim analysis of the phase I/II BEACON study. As described by Heeney and colleagues, unlike other gene therapy strategies, risto-cel introduces precise edits at the HBG1 and HBG2 promoter regions to inhibit BCL11A binding and promote the expression of anti-sickling fetal hemoglobin. "This targeted approach preserves overall BCL11A expression, avoiding disruption of its broader biologic functions," they pointed out. Gene-editing strategies are designed to directly address the underlying genetic mutation responsible for the disease or induce the production of fetal hemoglobin. This approach was endorsed by the FDA with the approval of exagamglogene autotemcel (exa-cel; Casgevy) -- a CRISPR/Cas9 gene-edited therapy -- for use in patients ages 12 and older with sickle cell disease and transfusion-dependent beta thalassemia. Locatelli pointed out that ex vivo gene therapy is resource-intensive and ill-suited to treat large numbers of patients in countries with limited resources, where most patients with sickle cell disease and transfusion-dependent beta thalassemia live.
Original title: “Base-Editing Gene Therapy Shows Promise in Sickle Cell Disease | MedPage Today”
cardio obstetric breakthrough beyond routine pregnancy care unmasking hodgkin s lymphoma
Original title: “A Cardio-Obstetric Breakthrough Beyond Routine Pregnancy Care: Unmasking Hodgkin's Lymphoma.”
the 516th case hyperinflammatory state acute renal dysfunction hepatosplenomegaly with lymphadenopathy and recurrent cerebral infarctions
Original title: “[The 516th case: hyperinflammatory state, acute renal dysfunction, hepatosplenomegaly with lymphadenopathy, and recurrent cerebral infarctions].”
silent myocardial infarction in pregnancy uncovered during routine antenatal echocardiographic screening
Original title: “Silent Myocardial Infarction in Pregnancy Uncovered During Routine Antenatal Echocardiographic Screening.”
toxic epidermal necrolysis mimicking linear iga bullous dermatosis without mucosal involvement a rare case report
Original title: “Toxic Epidermal Necrolysis Mimicking Linear IgA Bullous Dermatosis Without Mucosal Involvement: A Rare Case Report.”
treatment related benefit and satisfaction in patients with fabry disease in france insight into patients expectations and preferences from the prospective non interventional satis fab study
Original title: “Treatment-related benefit and satisfaction in patients with Fabry disease in France: insight into patients' expectations and preferences from the prospective, non-interventional SATIS-Fab study.”
pkc activators improve the function of biogenesis deficient cftr channels
Original title: “PKC activators improve the function of biogenesis-deficient CFTR channels.”
six novel sacs mutations expand the autosomal recessive spastic ataxia of charlevoix saguenay spectrum
Original title: “Six novel SACS mutations expand the autosomal recessive spastic ataxia of Charlevoix-Saguenay spectrum.”
distinct genetic variants from the whole exome sequencing of syndromic anorectal malformations a cross sectional study
Original title: “Distinct genetic variants from the whole-exome sequencing of syndromic anorectal malformations: A cross-sectional study.”
tirzepatide in metabolic diseases clinical efficacy and safety beyond diabetes and obesity
Original title: “Tirzepatide in Metabolic Diseases: Clinical Efficacy and Safety Beyond Diabetes and Obesity.”
increased glycogenin exposed residual glycogen in lysosomes is the early pathological finding in asymptomatic pompe disease
Original title: “Increased Glycogenin-Exposed Residual Glycogen in Lysosomes Is the Early Pathological Finding in Asymptomatic Pompe Disease.”
patient derived intestinal organoids in the global cystic fibrosis landscape
Original title: “Patient-Derived Intestinal Organoids in the Global Cystic Fibrosis Landscape.”
kikuchi fujimoto disease a differential diagnosis of febrile lymphadenopathy
Original title: “[Kikuchi-Fujimoto disease : a differential diagnosis of febrile lymphadenopathy].”
artificial intelligence personalized education dunning kruger effect
Original title: “Artificial intelligence, personalized education, Dunning-Kruger effect.”
roxadustat in the treatment of pure red cell aplasia associated with anti epo antibodies
Original title: “Roxadustat in the Treatment of Pure Red Cell Aplasia Associated With Anti-EPO Antibodies.”