Results 131 to 140 of about 72,117 (255)

ClinASO: An open-source platform for rapid drug discovery of gapmer antisense oligonucleotides. [PDF]

Mol Ther Nucleic Acids
Chen S, Liu H, Cong D, Dong A, Wang Y, Bi J, Guo S, Yang J, Wang X, Ren G, Zhang K, Wang H, Lai F, Dang Y.   +13 more
europepmc   +1 more source

International Guideline on the Diagnosis and Management of Pediatric Patients With Hereditary Angioedema

Allergy, EarlyView.
ABSTRACT Hereditary angioedema (HAE) with C1 inhibitor deficiency is a rare disease characterized by unpredictable episodes of tissue swelling (angioedema), which, in most cases, occur first under the age of 18 years, and entail a significant burden of disease not only for the patients but also for their families.
Henriette Farkas, Inmaculada Martinez‐Saguer, Konrad Bork, Anastasios E. Germenis, Anete S. Grumach, Hanga Réka Horváth, Andrea Luczay, Andrea Zanichelli, Markus Magerl, Stephen Betschel, Emel Aygören‐Pürsün, Jonathan A. Bernstein, Isabelle Boccon‐Gibod, Teresa Caballero, Mauro Cancian, Sandra Christiansen, Danny M. Cohn, Francisco Contreras, Sansanee Craig, Camelia Isaic, Ankur Jindal, Constance H. Katelaris, Hilary J. Longhurst, Andrew MacGinnitie, Jonny Peter, Grzegorz Porebski, Avner Reshef, Dinh Van Nguyen, Bruce Zuraw, Anthony J. Castaldo, Henrik Balle Boysen, Timothy Craig, the Hereditary Angioedema Working Group (HAWK Group), Adil Adatia, Fiorella Adrianzen, Shimalee Andarawewa, Sladjana Andrejevic, Gabriel Emmanuel Arce‐Estrada, Ecem Ay, Adil Bahadir, Noemi Anna Bara, Marko Barešić, Krasimira Baynova, Shira Benor, Juliette Besson, Dharmagat Bhattarai, Patricia Bigas, Alexis Bocquet, Laurence Bouillet, Nicholas Brodszki, Thomas Buttgereit, Rosario Cabañas, Regis Campos, Asuman Çamyar, Orlane Chol, Stefan Cimbollek, Monica Colque Bayona, Cascia Day, Mats de Lange, Alex Fam, Davide Firinu, Tomas Freiberger, Johana Gil‐Serrano, Delphine Gobert, Dawn Goodyear, Maria del Mar Guilarte Clavero, Svetlana Hadvabova, David Hagin, Roman Hakl, George Harmat, Mensuda Hasanhodzic, Gocki Jacek, Joshua Jacobs, Rashmi Jain, Milos Jesenak, Amin Kanani, Daniela Kapustová, Boris Karanovic, Paul Keith, Tamar Kinaciyan, Pavlina Kralickova, Marcin Kurowski, Krzysztof Kuziemski, Rolando Laurel‐Laurel, Iris Leibovich‐Nassi, Gabriela Leon Zambrana, Ramon Lleonart, Lorena Lorenzo, Ferhat Maksudov, Ania Manson, Dusanka Markovic, Jayne McGucken, Nihal Mete Gokmen, Radovan Mijanovic, Vania Maria Miranda Saavedra, Irene Modestou, Sandra Nieto, Nora Nilsson, Patrik Nordenfelt, Francesca Perego, Angelica Petraroli, Elsa Phillips‐Angles, Alicia Prieto‐García, Michel Raguet, Marc Riedl, Matija Rijavec, Solange Rodrigues Valle, Yaryna Romanyshyn, Antoine Saut, Riccardo Senter, Branislav Šlenker, Marta Sobotkova, Peter J. Spaeth, Marcin Stobiecki, Linda Sundler Björkman, Mireille‐Maria Suttle, Agnes Szilágyi, Paola Triggianese, Kassiani Tzeli, Martina Vachová, Anna Valerieva, Solange Valle, Lilian Varga, Walter A. Wuillemin, Patrick Yong, Zhi Yuxiang, Liudmyla Zabrodska, Radana Zachova, Julia Zharankova   +128 more
wiley   +1 more source

Guanine base modifications in antisense oligonucleotides mitigate acute central nervous system toxicity. [PDF]

RSC Chem Biol
Katsuyama M, Matsubayashi T, Ying Y, Mon SSL, Kuroda T, Yoshida-Tanaka K, Hara RI, Yamada T, Ui-Tei K, Yoshioka K.   +9 more
europepmc   +1 more source

Update on Non‐Biological and RNA‐Based Therapeutics in Chronic Inflammatory Diseases: Precision Medicine Through Small Molecules: An EAACI Position Paper

Allergy, EarlyView.
ABSTRACT In the last decades, critical advancements in research technology and knowledge on disease mechanisms steered therapeutic approaches for chronic inflammatory diseases towards unprecedented target specificity. For allergic and chronic lung diseases, biologic drugs pioneered this goal, acquiring on the way—through the clinical use of monoclonal ...
F. Roth‐Walter, I. M. Adcock, C. Benito‐Villalvilla, R. Bianchini, L. Bjermer, G. Caramori, L. Cari, K. F. Chung, Z. Diamant, I. Eguiluz‐Gracia, E. F. Knol, M. Jesenak, F. Levi‐Schaffer, G. Nocentini, L. O'Mahony, O. Palomares, F. Redegeld, M. Sokolowska, B. Van Esch, M. Zurlo, C. Stellato   +20 more
wiley   +1 more source

Characterisation of Antisense Oligonucleotides by Ion-Pair Reversed-Phase UHPLC-HRMS: Method development using Design of Experiments. [PDF]

J Mass Spectrom
Triolo A, Tavani F, Barnini P, Furlanetto S, Orlandini S.   +4 more
europepmc   +1 more source

Building a precision therapeutics program at a tertiary care children's hospital

Pediatric Investigation, EarlyView.
Luke Hamilton, Heidi Trinkman, Diana Carrasco, Kaitlin Smith, Kelly Vallance, Alejandra Dominguez, Danielle Miller, Megan Gibbs, Anish Ray   +8 more
wiley   +1 more source

Switching Long‐Term Prophylaxis to Donidalorsen for Hereditary Angioedema: 1‐Year OASISplus Results

Allergy, EarlyView.
This study evaluated the long‐term safety and efficacy of donidalorsen in patients who switched from a long‐term prophylactic treatment (LTP) to donidalorsen with 1‐year outcomes. Patients who switched from LTP to donidalorsen experienced a 67.6% reduction in HAE attack rates over 52 weeks.
Marc A. Riedl, Jonathan A. Bernstein, Joshua S. Jacobs, Timothy Craig, William R. Lumry, H. James Wedner, Aleena Banerji, Selina Gierer, Andrew Smith, Michael E. Manning, Francesca Perego, Laura Bordone, Sabrina Treadwell, Tao Lin, Kenneth B. Newman, Aaron Yarlas, Danny M. Cohn   +16 more
wiley   +1 more source

A xenotransplantation model for reactivation of paternal UBE3A using human-specific antisense oligonucleotides. [PDF]

Sci Rep
Smeenk H, Lendemeijer B, Buurma MG, Forgione MA, Slump DE, Monshouwer RA, Wallaard I, Mientjes EJ, Hoogendijk WJG, Elgersma Y, de Vrij FMS, Kushner SA.   +11 more
europepmc   +1 more source

Inhibition of HMGA2 Leads to Reduced Cell Proliferation and Increased Apoptosis in Human Embryonal Carcinoma Cell Lines

Andrology, EarlyView.
ABSTRACT Background The most prevalent solid tumors in young men are testicular germ cell tumors (TGCTs), and embryonal carcinoma is the most common subtype among non‐seminomatous germ cell tumors (NSGCTs). Despite the excellent cure rates of cisplatin‐based chemotherapy, resistance develops in 15%–30% of patients with metastatic cancer, which results ...
Marco De Martino, Matteo Lampitto, Alfredo Fusco, Marco Barchi, Francesco Esposito, Paolo Chieffi   +5 more
wiley   +1 more source

Chemically inducible antisense oligonucleotides for cell-specific gene silencing. [PDF]

RSC Chem Biol
Xun Z, Hai Y, Tang LJ, Jiang JH, Wu Z.
europepmc   +1 more source