Results 71 to 80 of about 200,281 (336)

High‐Throughput Strategies for Streamlining Lipid Nanoparticle Development Pipeline

Advanced Science, EarlyView.
This review highlights emerging state‐of‐the‐art high‐throughput strategies for optimising lipid nanoparticle formulation. By integrating combinatorial design, characterization, in vitro/in vivo screening, automation, and machine learning into a closed‐loop framework, it provides a roadmap to streamline discovery and accelerate the translation of ...
Lois Lam, Stephanie Watson, Yogambha Ramaswamy, Gurvinder Singh   +3 more
wiley   +1 more source

Real-world treatment management in hereditary transthyretin amyloidosis – an experience report and proposal for therapy switch decision criteria

Neurological Research and Practice
Background Hereditary transthyretin amyloidosis is a rapidly progressive and lethal disease. Thanks to the increasing number of disease-modifying treatments, prognosis has improved significantly.
Duc Chu Dieu, Helena F. Pernice, Harisa Muratovic, Paul J. Wetzel, Gina Barzen, Nicolas W. Wieder, Stefanie M. Werhahn, Bettina Heidecker, Sebastian Spethmann, Katrin Hahn   +9 more
doaj   +1 more source

Alternative Isotope Options for Amyloidosis Imaging: A Technologist’s Perspective [PDF]

, 2023
Jaime Warren
openalex   +1 more source

Nanoscopic Mapping of the Extracellular Space in Amyloid Plaque‐rich Cortex

Advanced Science, EarlyView.
The extracellular space and diffusion around amyloid plaques are examined using shadow imaging and single‐particle tracking. Increased diffusivity is found near plaques, extracellular matrix alterations, and plaque core penetrability that varies with amyloid phenotype.
Juan Estaún‐Panzano, Yulia Dembitskaya, Ivo Calaresu, Somen Nandi, Quentin Gresil, Evelyne Doudnikoff, Thierry Leste‐Laserre, Thierry Amédée, Laurent Cognet, Laurent Groc, Urs Valentin Nägerl, Erwan Bezard   +11 more
wiley   +1 more source

Localised Laryngeal Amyloidosis Endoscopic Excision -A Case Report [PDF]

, 2013
:We report a case of primary laryngeal amyloidosis in a 35 year old adult patient who presented with hoarseness of voice for 6 months duration with no other symptoms.
Sundhar Krishnan, Gurunathan, Varma, Srinivasa, Vikram, Venakata Subramanian Jagannathan   +2 more
core   +1 more source

Prediction of peptide and protein propensity for amyloid formation [PDF]

, 2014
Understanding which peptides and proteins have the potential to undergo amyloid formation and what driving forces are responsible for amyloid-like fiber formation and stabilization remains limited.
A Quintas, A Trovato, A Trovato, AC Davison, AC Tsolis, Alexandre Quintas, AM Fernandez-Escamilla, AP Pawar, AV Finkelstein, B Rost, C Nerelius, Carlos Família, CM Dobson, D Eisenberg, David A. Phoenix, DJ Selkoe, DM Fowler, Eugene A. Permyakov, F Chiti, F Chiti, F Sasagawa, GG Tartaglia, GG Tartaglia, H Hu, I Cherny, I Walsh, IV Baskakov, J Palau, J Tian, JC Rochet, JD Sipe, JM Zimmerman, JW Kelly, JW Kelly, K Rajagopal, KF DuBay, KK Frousios, KT O’Neil, L Goldschmidt, LO Jimenez, M Belli, M Emily, M Hollander, M Kuhn, M López de la Paz, M Oliveberg, M Stefani, M Sunde, M Sunde, M Zamani, MB Kursa, MJ Thompson, MT Pastor, N Becker, N Qian, O Conchillo-Solé, PK Teng, PY Chou, RS Harrison, S Idicula-thomas, S Kawashima, S Kawashima, S Maurer-Stroh, S Ventura, S Yoon, S Yoon, Sarah R. Dennison, SJ Hamodrakas, SJ Hamodrakas, SK Maji, SO Garbuzynskiy, T Hothorn, T Hothorn, T Hothorn, T Scheibel, TPJ Knowles, VS Mathura, WH DePas, WT Astbury, Y Kallberg, Ž Eva   +80 more
core   +5 more sources

Engineered RNA Devices for In Vivo Targeted Therapeutics via Advanced Delivery Systems

Aggregate, EarlyView.
Schematic illustration of engineered RNA devices for in vivo targeted therapeutics via advanced delivery systems. ABSTRACT Engineered RNA devices can identify disease‐specific markers and precisely regulate gene expression, which is of great significance to the development of precision medicine.
Wei Luo, Xia Liu, Ying Han, Yijing Duan, Chuao Yu, Na Kong, Tian Xie   +6 more
wiley   +1 more source

Natural history and outcome in systemic AA amyloidosis [PDF]

, 2007
BACKGROUND:Deposition of amyloid fibrils derived from circulating acute-phase reactant serum amyloid A protein (SAA) causes systemic AA amyloidosis, a serious complication of many chronic inflammatory disorders.
Gallimore, JR, Gilbertson, JA, Gillmore, JD, Goodman, HJB, Hawkins, PN, Lachmann, HJ, Sabin, CA   +6 more
core   +1 more source

Genomics Review of Selective RET Inhibitors Sensitivity in Thyroid Cancer Clinical Trials

American Journal of Medical Genetics Part C: Seminars in Medical Genetics, EarlyView.
ABSTRACT RET gene is a driver of thyroid cancer (TC) tumorigenesis. The incidence of TC has increased worldwide in the last few decades, both in medullary and follicular‐derived subtypes. Several drugs, including multikinase and selective inhibitors, have been explored.
Sara Gil‐Bernabé, Lucía García‐DeLaFuente, Alejandro García‐Álvarez, Ginesa García‐Rostán, Jaume Capdevila, Jorge Hernando   +5 more
wiley   +1 more source

Co‐Opting MBNL‐Dependent Alternative Splicing Cassette Exons to Control Gene Therapy in Myotonic Dystrophy

Annals of Neurology, EarlyView.
Objective Myotonic dystrophy type 1 (DM1) is a highly variable, multisystemic genetic disorder caused by a CTG repeat expansion in the 3′ untranslated region of DMPK. Toxicity is exerted by repeat‐containing DMPK transcripts that sequester muscleblind‐like (MBNL) proteins and lead to deleterious yet predictable changes in alternative splicing.
Samuel T. Carrell, Ellie M. Carrell, Ryan Giovenco, Beverly L. Davidson   +3 more
wiley   +1 more source