Results 211 to 220 of about 159,025 (352)

NANO GEL BASED CO-DELIVERY OF DICLOFENAC SODIUM AND LUTEOLIN: A PROMISING THERAPEUTIC STRATEGY FOR SPINAL MUSCULAR ATROPHY

Dr. Saravanan V. S., Karthiraja A. S., Sankaranarayanan A., Gowtham Prakash U., Gunavathy V., Guna V., Muniyan N., Metilda Stella Rani G.*
openalex   +1 more source

The systemic complexity of a monogenic disease: the molecular network of spinal muscular atrophy [PDF]

Ines Tapken, Theresa Schweitzer, Martina Paganin, Tobias Schüning, Nora Tula Detering, Gaurav Sharma, Moritz Niesert, Afshin Saffari, D. Kühn, Amy Glynn, Federica Cieri, Pamela Santonicola, Claire Cannet, Florian Gerstner, Kiterie M. E. Faller, Yu-Ting Huang, Rashmi Kothary, Thomas H. Gillingwater, Elia Di Schiavi, Christian M. Simon, Niko Hensel, Andreas Ziegler, Gabriella Viero, Andreas Pich, Peter Claus   +24 more
openalex   +1 more source

Survival Motor Neuron Protein is Released from Cells in Exosomes: A Potential Biomarker for Spinal Muscular Atrophy [PDF]

, 2017
Leslie A. Nash, Emily McFall, Amanda M. Perozzo, Maddison Turner, Kathy L. Poulin, Yves De Repentigny, Joseph K. Burns, Hugh J. McMillan, Jodi Warman‐Chardon, Dylan Burger, Rashmi Kothary, Robin J. Parks   +11 more
openalex   +1 more source

A Systemic Selective Modified mRNA Delivery Platform for Preventing Chemotherapy‐Induced Cardiotoxicity

Advanced Science, EarlyView.
In Yoo and Mainkar et al., we present a minimally invasive, CM‐selective modRNA delivery system encapsulated in lipid nanoparticles for intravenous (IV) administration. This platform enables selective cardiac translation of therapeutic modRNA but suppresses expression in off‐target tissues, including tumors.
Jimeen Yoo, Gayatri Mainkar, Matteo Ghiringhelli, Bernardo Gindri Dos Santos, Magdalena M. Żak, Keerat Kaur, Ann Anu Kurian, Matthew Adjmi, Segev Sharon, Eftychia Markopoulou, Lucia Žigová, Daniela Hirsch, Sydney Pessin, Rachel Hanan, Juan Luis Escano Abreu, Niki Brisnovali, Nicole Dubois, Leigh Goedeke, Mengcheng Shen, Lior Zangi   +19 more
wiley   +1 more source

Newborn Screening for Spinal Muscular Atrophy in the UK: Use of Modelling to Identify Priorities for Ongoing Evaluation. [PDF]

Int J Neonatal Screen
Thokala P, Bessey A, Knowles R, Marshall J, Visintin C, Lawton M, Lombardo S.   +6 more
europepmc   +1 more source

Relationship between muscular atrophy of the tongue and impairment of bolus transport during swallowing in patients with amyotrophic lateral sclerosis

, 2008
Hiroshige Taniguchi, Sachiko Ootaki, Yuka Kajii, Yoshiaki Yamada, Makoto Inoue   +4 more
openalex   +2 more sources

A Hydrodynamic Bioreactor for High‐Yield Production of Extracellular Vesicles from Stem Cell Spheroids with Defined Cargo Profiling

Advanced Science, EarlyView.
This study harnesses hydrodynamic flows to generate, confine and stimulate stem cell spheroids, enabling the large‐scale production of extracellular vesicles (EVs). This innovative method not only streamlines spheroid formation and subsequent EV release in a single, integrated process, but also ensures the generation of EVs with enhanced biological ...
Solène Lenoir, Elliot Thouvenot, Giacomo Gropplero, Léonie Dec, Damarys Loew, Clotilde Théry, Jose E Perez, Claire Wilhelm   +7 more
wiley   +1 more source

Targeting the mutant androgen receptor with PROTACs in spinal and bulbar muscular atrophy. [PDF]

Neurotherapeutics
Lee C, Lim J.
europepmc   +1 more source

P863: Additional haplotype in NIPD for spinal muscular atrophy: Triploid of a twin gestation [PDF]

Lingrong Kong
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RUNX2 Activation in Fibro/Adipogenic Progenitors Promotes Muscle Fibrosis in Muscular Dystrophy

Advanced Science, EarlyView.
This study revealed a novel role of the chemokine‐TGF‐β1‐RUNX2 axis in determining the fate of FAP differentiation and modulating muscle fibrosis in patients and mice with muscular dystrophies. ABSTRACT Clinical evidence indicates concurrent muscle inflammation and fibrosis in muscular dystrophies (MDs); however, the molecular mechanisms underlying ...
Pengkai Wu, Zehua Zhang, Kai Zheng, Ziqi Zhu, Yong Zhu, Abdukahar Kiram, Lei Zhao, Huihui Chen, Zhu Xu, Xihua Li, Beicheng Sun, Zhijian Li, Dengqiu Xu   +12 more
wiley   +1 more source