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A 15-Item modification of the PSP rating scale to improve clinical meaningfulness and statistical performance.
Nat Commun
January 2025
Biogen Inc, Cambridge, MA, USA.
Progressive supranuclear palsy (PSP) is a rare neurodegenerative disorder characterized by physical, cognitive, and behavioral impairments. The PSP Rating Scale (PSPRS) is a widely used and validated, clinical scale to monitor disease progression. Here we show the modification of PSPRS to improve clinical meaningfulness and sensitivity.
View Article and Find Full Text PDFDelivery of Prime editing in human stem cells using pseudoviral NanoScribes particles.
Nat Commun
January 2025
CIRI, Centre International de Recherche en Infectiologie Univ Lyon, Inserm, U1111, Université Claude Bernard Lyon 1, CNRS, UMR5308, ENS de Lyon, F-69007, Lyon, France.
Prime Editing can rewrite genes in living cells by allowing point mutations, deletions, or insertion of small DNA sequences with high precision. However, its safe and efficient delivery into human stem cells remains a technical challenge. In this report, we engineer Nanoscribes, virus-like particles that encapsidate ribonucleoprotein complexes of the Prime Editing system and allow their delivery into recipient cells.
View Article and Find Full Text PDFOrthogonal and multiplexable genetic perturbations with an engineered prime editor and a diverse RNA array.
Nat Commun
December 2024
Department of Chemical and Biomolecular Engineering, Rice University, Houston, TX, USA.
Programmable and modular systems capable of orthogonal genomic and transcriptomic perturbations are crucial for biological research and treating human genetic diseases. Here, we present the minimal versatile genetic perturbation technology (mvGPT), a flexible toolkit designed for simultaneous and orthogonal gene editing, activation, and repression in human cells. The mvGPT combines an engineered compact prime editor (PE), a fusion activator MS2-p65-HSF1 (MPH), and a drive-and-process multiplex array that produces RNAs tailored to different types of genetic perturbation.
View Article and Find Full Text PDFEnhanced fetal hemoglobin production via dual-beneficial mutation editing of the HBG promoter in hematopoietic stem and progenitor cells for β-hemoglobinopathies.
Stem Cell Res Ther
December 2024
Centre for Stem Cell Research (CSCR), A Unit of InStem Bengaluru, Christian Medical College Campus, Vellore, Tamil Nadu, 632002, India.
Background: Sickle cell disease (SCD) and β-thalassemia patients with elevated gamma globin (HBG1/G2) levels exhibit mild or no symptoms. To recapitulate this natural phenomenon, the most coveted gene therapy approach is to edit the regulatory sequences of HBG1/G2 to reactivate them. By editing more than one regulatory sequence in the HBG promoter, the production of fetal hemoglobin (HbF) can be significantly increased.
View Article and Find Full Text PDFSurgical Complications Following Appendectomy in Obese Patients: A Single Tertiary Care Center Study.
Cureus
November 2024
General Surgery, King Abdulaziz Medical City, Riyadh, SAU.
Background The escalating global prevalence of obesity raises concerns about its implications for health outcomes. While obesity is acknowledged as a major risk factor for various diseases, its impact on appendicitis and appendectomy outcomes remains less explored. Methods Data on overweight and obese adults aged 18 to 65 treated for appendicitis in King Abdulaziz Medical City in Riyadh, Saudi Arabia were collected retrospectively.
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