Results 81 to 90 of about 5,426,861 (329)

Lipid Nanoparticles for the Delivery of CRISPR/Cas9 Machinery to Enable Site‐Specific Integration of CFTR and Mutation‐Agnostic Disease Rescue

Advanced Functional Materials, EarlyView.
Lipid nanoparticles (LNPs) are optimized to co‐deliver Cas9‐encoding messenger RNA (mRNA), a single guide RNA (sgRNA) targeting the endogenous cystic fibrosis transmembrane conductance regulator (CFTR) gene, and homologous linear double‐stranded donor DNA (ldsDNA) templates encoding CFTR.
Ruth A. Foley, Paul G. Ayoub, Vrishti Sinha, Colin Juett, Alicia Sanoyca, Emily C. Duggan, Lindsay E. Lathrop, Priyanka Bhatt, Kevin Coote, Beate Illek, Brigitte N. Gomperts, Donald B. Kohn, Steven J. Jonas   +12 more
wiley   +1 more source

Ikaros family zinc finger 1 regulates dendritic cell development and function in humans

Nature Communications, 2018
IKZF1 is a transcription factor known to regulate mammalian B-cell development. Here the authors show that IKZF1 is required for human pDC development and regulation of DC cytokine production in patients with IKZF1 haploinsufficiency, findings which are ...
Urszula Cytlak, Anastasia Resteu, Delfien Bogaert, Hye Sun Kuehn, Thomas Altmann, Andrew Gennery, Graham Jackson, Attila Kumanovics, Karl V. Voelkerding, Seraina Prader, Melissa Dullaers, Janine Reichenbach, Harry Hill, Filomeen Haerynck, Sergio D. Rosenzweig, Matthew Collin, Venetia Bigley   +16 more
doaj   +1 more source

Plasma‐Treated Hydrogel for Combined RONS and Chemotherapy Delivery: A Proof‐of‐Concept In Ovo

Advanced Functional Materials, EarlyView.
This study explores plasma‐treated hydrogels (PTH) as a new way to deliver both reactive oxygen and nitrogen species and chemotherapy (Doxorubicin) directly to tumors. In ovo tests show effects after a single dose, especially in osteosarcoma tumors. Tumor weight decrease, and a resistance‐related protein is reduced.
Milica Živanić, Albert Espona‐Noguera, Angela Privat Maldonado, Patrik Matušů, Francesco Tampieri, Maria‐Pau Ginebra, Abraham Lin, Evelien Smits, Annemie Bogaerts, Cristina Canal   +9 more
wiley   +1 more source

14 Years after Discovery: Clinical Follow-up on 15 Patients with Inducible Co-Stimulator Deficiency

Frontiers in Immunology, 2017
BackgroundInducible co-stimulator (ICOS) deficiency was the first monogenic defect reported to cause common variable immunodeficiency (CVID)-like disease in 2003.
Johanna Schepp, Janet Chou, Andrea Skrabl-Baumgartner, Peter D. Arkwright, Karin R. Engelhardt, Sophie Hambleton, Sophie Hambleton, Tomohiro Morio, Ekkehard Röther, Klaus Warnatz, Raif Geha, Bodo Grimbacher, Bodo Grimbacher   +12 more
doaj   +1 more source

Epstein–Barr Virus+ Smooth Muscle Tumors as Manifestation of Primary Immunodeficiency Disorders

Frontiers in Immunology, 2018
Epstein–Barr virus positive (EBV+) smooth muscle tumors (SMTs) constitute a very rare oncological entity. They usually develop in the context of secondary immunodeficiency caused by human immunodeficiency virus infection or immunosuppressive treatment ...
T. Magg, T. Schober, C. Walz, J. Ley-Zaporozhan, F. Facchetti, C. Klein, F. Hauck   +6 more
semanticscholar   +1 more source

Cold Quad‐Modal Nanocomplex for Precise and Quantitative In Vivo Stem Cell Tracking

Advanced Functional Materials, EarlyView.
Multimodal albumin–bismuth sulfide–superparamagnetic iron oxide (ABS) nanocomplexes are developed for stem cell tracking across four different imaging modalities: MRI, MPI, MSOT, and CT. Combining its flexibility with high sensitivity, this quad‐modal imaging agent enables a robust quantification of ABS‐labeled stem cells in vivo.
Ali Shakeri‐Zadeh, Chao Wang, Shreyas Kuddannaya, Saleem Yousf, Jeff W. M. Bulte   +4 more
wiley   +1 more source

Early-onset primary antibody deficiency resembling common variable immunodeficiency challenges the diagnosis of Wiedeman-Steiner and Roifman syndromes

Scientific Reports, 2017
Syndromic primary immunodeficiencies are rare genetic disorders that affect both the immune system and other organ systems. More often, the immune defect is not the major clinical problem and is sometimes only recognized after a diagnosis has been made ...
Delfien J. Bogaert, Melissa Dullaers, Hye Sun Kuehn, Bart P. Leroy, Julie E. Niemela, Hans De Wilde, Sarah De Schryver, Marieke De Bruyne, Frauke Coppieters, Bart N. Lambrecht, Frans De Baets, Sergio D. Rosenzweig, Elfride De Baere, Filomeen Haerynck   +13 more
doaj   +1 more source

First Year of Israeli Newborn Screening for Severe Combined Immunodeficiency—Clinical Achievements and Insights

Frontiers in Immunology, 2017
Severe combined immunodeficiency (SCID), the most severe form of T cell immunodeficiency, is detectable through quantification of T cell receptor excision circles (TRECs) in dried blood spots obtained at birth.
Erez Rechavi, Atar Lev, Amos J. Simon, Tali Stauber, Suha Daas, Talia Saraf-Levy, Arnon Broides, Arnon Broides, Amit Nahum, Amit Nahum, Nufar Marcus, Nufar Marcus, Suhair Hanna, Suhair Hanna, Polina Stepensky, Polina Stepensky, Ori Toker, Ori Toker, Ilan Dalal, Ilan Dalal, Ilan Dalal, Ilan Dalal, Amos Etzioni, Amos Etzioni, Shlomo Almashanu, Raz Somech, Raz Somech, Raz Somech   +27 more
doaj   +1 more source

Nano‐ and Micro‐Sized Solid Materials Used as Antiviral Agents

Advanced Functional Materials, EarlyView.
Due to the rise of viral infections in humans and possible viral outbreaks, the use of nano‐ or micro‐sized materials as antiviral agents is rapidly increasing. This review explores their antiviral properties against RNA and DNA viruses, either as a prevention or a treatment tool, by delving into their mechanisms of action and how to properly assess ...
Orfeas‐Evangelos Plastiras, Imelda Raczkiewicz, Peggy Bouquet, Jean‐Philippe Dacquin, Jeremy Dhainaut, Anne Goffard, Christophe Volkringer   +6 more
wiley   +1 more source

Inborn errors of human IKAROS: LOF and GOF variants associated with primary immunodeficiency.

Clinical and Experimental Immunology, 2022
H. Kuehn, Brigette Boast, S. Rosenzweig
semanticscholar   +1 more source