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Long-term safety of lentiviral or gammaretroviral gene-modified T cell therapies.
Nat Med
January 2025
Center for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Long-term risks of gene therapy are not fully understood. In this study, we evaluated safety outcomes in 783 patients over more than 2,200 total patient-years of observation from 38 T cell therapy trials. The trials employed integrating gammaretroviral or lentiviral vectors to deliver engineered receptors to target HIV-1 infection or cancer.
View Article and Find Full Text PDFCAR-T cell targeting three receptors on autoreactive B cells for systemic lupus erythematosus therapy.
J Autoimmun
January 2025
Division of Haematology/Oncology, Department of Medicine, Case Western Reserve University, Cleveland, OH, USA; Department of Pathology, Case Western Reserve University, Cleveland, OH, USA; Pediatric Haematology and Oncology, The Angie Fowler Adolescent & Young Adult Cancer Institute, University Hospitals Rainbow Babies & Children's Hospital, Cleveland, OH, USA; The Case Comprehensive Cancer Center, Case Western Reserve University School of Medicine, Cleveland, OH, USA. Electronic address:
Systemic lupus erythematosus (SLE) is an autoimmune disease characterized by dysregulated B cell activation, autoantibody production, and nephritis. B cell activating factor (BAFF) overexpression enhances autoreactive B-cell survival, driving autoimmunity. BAFF specific belimumab and CD20 specific rituximab antibodies are used for SLE therapy but are not curative, highlighting the need for alternative B cell depletion therapies.
View Article and Find Full Text PDFElectroencephalogram-Guided General Anesthesia in a Pediatric Patient With Alexander's Disease: A Case Report.
A A Pract
January 2025
From the Department of Anesthesiology, Perioperative and Pain Medicine, Stanford University School of Medicine, Palo Alto, California.
In this case, the electroencephalogram (EEG) was used to guide anesthesia care for a pediatric patient with Alexander's Disease undergoing serial intrathecal injections. Previous procedures using a standard maintenance propofol dose of up to 225 µg/kg/min led to postanesthetic recovery times of over 6 hours, requiring a neurology consult for noncoherence. The EEG assisted in guiding maintenance propofol dosing to 75 µg/kg/min, decreasing postanesthetic wash-off and postanesthesia care unit (PACU) recovery time by 50%.
View Article and Find Full Text PDFVitiligo Exchange: An Expert Panel Discussion of Two Clinical Cases.
J Clin Aesthet Dermatol
December 2024
Dr. Grimes is the Founder and Director at the Vitiligo & Pigmentation Institute of Southern California in Los Angeles, California; Clinical Professor of Dermatology at the University of California, Los Angeles. Current President of the Global Vitiligo Foundation.
This article is based on a roundtable discussion in which three panelists review clinically relevant insights about vitiligo and discuss two cases that illustrate the multiple challenges faced by both patients and clinicians in managing this complex disease. The first is a 32-year-old White female patient with Fitzpatrick Skin Type III/IV with extensive depigmentation in the trunk area. The patient achieved 90-percent repigmentation with a combination therapy approach.
View Article and Find Full Text PDFIncomplete human reference genomes can drive false sex biases and expose patient-identifying information in metagenomic data.
Nat Commun
January 2025
Department of Pediatrics, University of California San Diego, La Jolla, CA, USA.
As next-generation sequencing technologies produce deeper genome coverages at lower costs, there is a critical need for reliable computational host DNA removal in metagenomic data. We find that insufficient host filtration using prior human genome references can introduce false sex biases and inadvertently permit flow-through of host-specific DNA during bioinformatic analyses, which could be exploited for individual identification. To address these issues, we introduce and benchmark three host filtration methods of varying throughput, with concomitant applications across low biomass samples such as skin and high microbial biomass datasets including fecal samples.
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