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Journal Club: Anti-CD19 Chimeric Antigen Receptor T Cell Therapy for Refractory Systemic Lupus Erythematosus.
ACR Open Rheumatol
November 2023
Beth Israel Deaconess Medical Center and Harvard Medical School, Boston, Massachusetts.
Objective: Despite substantial advances in the treatment of systemic lupus erythematosus (SLE), some patients do not respond to the current state-of-the art therapies. This study assessed the tolerability and efficacy of CD19 chimeric antigen receptor (CAR) T cells in a small series of seriously ill and treatment-resistant patients with SLE.
Methods: Five patients with SLE (four female patients and one male patient) with a median age of 22 (range 18-24) years, a median disease duration of 4 (range 1-9) years, and active disease (median Systemic Lupus Erythematosus Disease Activity Index score of 16 [range 8-16]) refractory to several immunosuppressive drug treatments were enrolled in a compassionate-use CAR-T cell program.
Normalization of clonal diversity in gene therapy studies using shape constrained splines.
Sci Rep
March 2022
University of Groningen - Bernoulli Institute for Mathematics, Computer Science and Artificial Intelligence, Groningen, Netherlands.
Viral vectors are used to insert genetic material into semirandom genomic positions of hematopoietic stem cells which, after reinfusion into patients, regenerate the entire hematopoietic system. Hematopoietic cells originating from genetically modified stem cells will harbor insertions in specific genomic positions called integration sites, which represent unique genetic marks of clonal identity. Therefore, the analysis of vector integration sites present in the genomic DNA of circulating cells allows to determine the number of clones in the blood ecosystem.
View Article and Find Full Text PDFSite-specific host gene modification by zinc finger nucleases: pointing the way to drug free control of HIV-1?
Clin Transl Immunology
July 2014
The Kirby Institute of Infection and Immunity in Society, University of New South Wales , Kensington, New South Wales, Australia ; The Centre for Applied Medical Research, St Vincent's Hospital, Sydney, New South Wales, Australia.
Anti-retroviral therapy (ART) for human immunodeficiency virus-1 (HIV-1) infection has transformed its clinical course with spectacular reductions in morbidity and mortality, turning this once fatal diagnosis into a manageable chronic infection. However, ART has its limitations. Current ART does not eliminate the virus.
View Article and Find Full Text PDFCell-based therapy for myocardial repair in patients with acute myocardial infarction: rationale and study design of the SWiss multicenter Intracoronary Stem cells Study in Acute Myocardial Infarction (SWISS-AMI).
Am Heart J
July 2010
Department of Cardiology, University Hospital Zurich, Zurich, Switzerland.
Article Synopsis
- Recent studies suggest that giving bone marrow mononucleated cells (BM-MNCs) intracoronarily may improve heart remodeling after a heart attack, especially when administered between days 4 to 7 post-incident.
- A multicenter trial involving 192 patients with acute myocardial infarction (AMI) will assess the effectiveness of BM-MNC treatment compared to a control group receiving standard medical care, with evaluation using cardiac magnetic resonance imaging over a year.
- The primary goal is to measure changes in left ventricular ejection fraction at 4 months, while the study will also investigate the best timing for treatment and identify which patients might benefit most from cellular therapy.
The art of reinfusing intestinal secretions.
J Support Oncol
June 2010
Division of Gastroenterology and Hepatology University of Virginia Health System, Charlottesville, VA 22908 , USA.
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