Results 251 to 260 of about 7,473,287 (361)

Plasma microRNA Signature as Predictive Marker of Clinical Response to Therapy During Multiple Sclerosis

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Despite the availability of effective therapies for Multiple Sclerosis (MS), the unpredictable nature of disease progression and the variability in individual treatment outcomes call for reliable biomarkers. This pilot study aims to investigate the potential of plasma circulating microRNAs (miRNAs) as predictive biomarkers for ...
Fortunata Carbone, Alessandra Colamatteo, Teresa Micillo, Gianmarco Abbadessa, Silvia Garavelli, Clorinda Fusco, Claudia Russo, Francesco Perna, Federica Garziano, Claudia La Rocca, Maria Mottola, Giorgia Teresa Maniscalco, Simona Bonavita, Antonio Luca Spiezia, Alessia Castiello, Roberta Lanzillo, Vincenzo Brescia Morra, Claudio Procaccini, Paola de Candia, Giuseppe Matarese   +19 more
wiley   +1 more source

PP 6.3 – 00178 Targeting the human and macaque CCR5 genes using the CRISPR-SaCas9 gene-editing platform

Journal of Virus Eradication, 2022
M. Caocci, T. Burdo, R. Kaminski, P. Mancuso   +3 more
doaj  

Development of a novel gene editing lexicon for hemophilia: methodology and results. [PDF]

Res Pract Thromb Haemost
Kessler CM, Valentino LA, Thornburg CD, Unzu C, Kay MA, Peyvandi F, Smith P, Miesbach W, McKeown W, Pierce GF, Khair K, Starcevic K, Pillai M, Jones M, Sindhurakar A, Whyte L, Delwart V, Chiao M, Gutstein DE, Antonino I, Hermans C, Pipe SW.   +21 more
europepmc   +1 more source

Oligophrenin-1 (OPHN1), a Gene Involved in X-Linked Intellectual Disability, Undergoes RNA Editing and Alternative Splicing during Human Brain Development

, 2014
Sabina Barresi, Sara Tomaselli, A. Athanasiadis, Federica Galeano, Franco Locatelli, Enrico Bertini, Ginevra Zanni, Angela Gallo   +7 more
openalex   +2 more sources

RNA editing differently affects protein-coding genes in D. melanogaster and H. sapiens [PDF]

, 2015
Luigi Grassi, Guido Leoni, Anna Tramontano   +2 more
openalex   +1 more source

Molecular Screening of SCN1A‐Related Seizures in Children With Febrile Seizures: Diagnostic Yield and Variant Distribution

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective SCN1A‐related seizures first present as febrile seizures (FS). Definitive features emerge later, making early diagnosis challenging. We evaluated the diagnostic yield and clinical characteristics of pathogenic SCN1A variants in FS, and analyzed the effect of variant characteristics on clinical phenotypes required for early ...
Jia Wang, Yang‐Yang Wang, Lin‐Yan Hu, Xiu‐Yu Shi, Wen He, Dan Sun, Baoguang Li, Xiaoming Liu, Sanqing Xu, Lijun Du, Chunhui Tang, Li‐Ping Zou   +11 more
wiley   +1 more source

Fighting RNA viruses with a gold nanoparticle Cas13d gene-editing armor. [PDF]

Mol Ther Nucleic Acids
De Carli A, Favaro D, Filipponi C, Filippini F, Fonnesu R, Plicanti E, Nottoli S, Barski P, Lindstaedt A, Witt D, Falleni A, Frenzilli G, Alcalá-Lalinde A, Herrera-Carrillo E, Raffa V, Freer G, Pistello M, Lai M.   +17 more
europepmc   +1 more source

54. Genome Editing of Primary Human CD34+ Hematopoietic Stem Cells Enables a Safe Harbor Targeted Gene Addition Therapeutic Strategy for Chronic Granulomatous Disease [PDF]

, 2015
Suk See De Ravin, Andreas Reik, Peiqi Liu, Linhong Li, Madhusudan V. Peshwa, Narda Theobald, Uimook Choi, Janet Lee, Sherry Koontz, Gary Lee, Philip D. Gregory, Fyodor D. Urnov, Harry L. Malech   +12 more
openalex   +1 more source

Association Between Metabolic Syndrome and Radiographic Changes in Psoriatic Arthritis: A Cohort Study

Arthritis Care &Research, EarlyView.
Objective Metabolic syndrome (MetS) is a known comorbidity of psoriatic arthritis (PsA) and is associated with PsA disease activity. We aimed to explore the association between MetS and radiographic features (peripheral and axial) in PsA. Methods We included patients with PsA followed at our prospective observational cohort for the period between 1978 ...
Fadi Kharouf, Shangyi Gao, S. Ercan Tunc, Justine Y. Ye, Daniel Pereira, Dafna D. Gladman, Vinod Chandran   +6 more
wiley   +1 more source

Gene Editing for Duchenne Muscular Dystrophy: From Experimental Models to Emerging Therapies. [PDF]

Degener Neurol Neuromuscul Dis
Haque US, Yokota T.
europepmc   +1 more source