Results 141 to 150 of about 70,372 (263)

Establishing Sensory Neurons as Therapeutic Targets in Peripheral Neuropathy Driven by Polyglutamine Expanded Murine ATXN3

Annals of Neurology, EarlyView.
Repeat expansion disorders frequently involve peripheral neuropathy, yet mechanisms remain unclear. Using a spinocerebellar ataxia type 3 (SCA3) Knock‐In Atxn3Q300/Q6, we identify progressive sensorimotor deficits, peripheral nerve pathology, and dorsal root ganglia RNA splicing dysregulation.
Juan P. Mato, John M. Hayes, Jacen Emerson, Maeha Sridharan, Toluwani Adelaja, Arsal Naeem, Alexandra F. Putka, Hayley S. McLoughlin   +7 more
wiley   +1 more source

The adeno-associated virus Rep proteins target PP4:SMEK1 by preventing substrate recruitment. [PDF]

PLoS Pathog
Vandewinkel B, Torrekens S, Claes Z, Bollen M, Henckaerts E.   +4 more
europepmc   +1 more source

Entdeckung eines antiviralen Elektronentransferprozesses zur Erzeugung katalytisch selbstversorgender viraler Restriktionsfaktoren

Angewandte Chemie, EarlyView.
CYB5R3 wird als mutmaßlicher Elektronentransferpartner von Viperin (RSAD2) identifiziert, was erklärt, warum die ER‐Lokalisation für die antivirale Aktivität erforderlich ist. Dieses Wissen ermöglichte die Entwicklung immunstiller, katalytisch selbstversorgender antiviraler Restriktionsfaktor‐Enzyme (iCAREs), die ein antivirales Nukleosidtriphosphat ...
Mengdi Wu, Nghi Thao Hoang, Deborah Grifagni, Meritxell Wu‐Lu, Yujie Sheng, Theo Situmorang, Pei‐Hsin Tai, Zheng Chen, Astrid Maluta, Mohammed Hakil, Bianca Susini, Hannah Florance, Peter‐Leon Hagedoorn, John Mark Sutton, Maria‐Andrea Mroginski, Simone Ciofi‐Baffoni, Kourosh H. Ebrahimi   +16 more
wiley   +1 more source

Improvement of Adeno-Associated Virus (AAV)-Based Technologies by Cell-Penetrating Penta-Peptides (CPP5s). [PDF]

Pharmaceutics
Guo CW, Diener A, Matsuyama S.
europepmc   +1 more source

Discovery of an Antiviral Electron Transfer Process to Create Catalytically Self‐Sufficient Viral Restriction Factors

Angewandte Chemie International Edition, EarlyView.
CYB5R3 is identified as a putative electron‐transfer partner of viperin (RSAD2), explaining why ER localisation is required for antiviral activity. Its utilisation enabled the engineering of immune‐silent, catalytically self‐sufficient antiviral restriction factor enzymes (iCAREs) that produce an antiviral nucleoside triphosphate analogue (ANTA ...
Mengdi Wu, Nghi Thao Hoang, Deborah Grifagni, Meritxell Wu‐Lu, Yujie Sheng, Theo Situmorang, Pei‐Hsin Tai, Zheng Chen, Astrid Maluta, Mohammed Hakil, Bianca Susini, Hannah Florance, Peter‐Leon Hagedoorn, John Mark Sutton, Maria‐Andrea Mroginski, Simone Ciofi‐Baffoni, Kourosh H. Ebrahimi   +16 more
wiley   +1 more source

Gene Therapy Using Recombinant Adeno-Associated Virus for Leber Congenital Amaurosis Induced by RPE65 Mutation. [PDF]

Clin Ophthalmol
Owliaee I, Teimoori A, Shoushtari M, Shojaeian A.   +3 more
europepmc   +1 more source

Improved Detection of Myositis‐Specific Autoantibodies Using Luciferase Immunoprecipitation Systems Assay: Comparison With Line Blot and Conventional Immunoprecipitation

Arthritis &Rheumatology, EarlyView.
Objective Myositis‐specific autoantibodies guide the diagnosis and classification of idiopathic inflammatory myopathies, but current immunoassays vary in accuracy, particularly for autoantibodies associated with interstitial lung disease. To compare the performance of the luciferase immunoprecipitation systems (LIPS) assay with line blot and ...
Peter D. Burbelo, Iago Pinal‐Fernandez, Maria Casal‐Dominguez, Xin Tian, Shijinqiu Gao, Neha Gupta, Eric W. Robbins, John A. Chiorini, Christopher A. Mecoli, Lisa Christopher‐Stine, Livia Casciola‐Rosen, Andrew L. Mammen, Sonye K. Danoff, Anthony F. Suffredini, Julio A. Huapaya   +14 more
wiley   +1 more source

Rapid multiplex liver gene-editing in mice using adeno-associated virus 8 or lipid nanoparticles. [PDF]

Mol Ther Adv
Wu D, Bolt I, Tolenaars DW, Duijst S, In Het Panhuis W, Hofstraat SRJ, Zwolsman R, van der Meel R, Paulusma CC, van de Graaf SFJ.   +9 more
europepmc   +1 more source

Precision medicine in paediatrics: Progress and priorities

British Journal of Clinical Pharmacology, EarlyView.
Precision medicine is revolutionizing personalized healthcare, advancing both diagnostics and therapeutics at an unprecedented pace. Reviewing the paediatric applications of pharmacometrics, pharmacogenomics and advanced therapy medicinal products highlights not only the relevance of these exciting innovations to frontline care but also the significant
Nicola Husain, Claire Booth, Joseph F. Standing, Charlotte I. S. Barker   +3 more
wiley   +1 more source

Rewriting Renal Fate: The Evolving Landscape of Adeno-Associated Virus-Mediated Kidney Gene Therapies. [PDF]

Kidney360
Hickman PG, Asokan A, Wilson MH.
europepmc   +1 more source