Results 181 to 190 of about 80,448 (304)

Systematic generation of <i>Drosophila</i> Wnt transgenes enables the characterization of canonical Wnt signaling. [PDF]

Fly (Austin)
Little JC, Debrunner M, de Hoog T, Tadjik F, Kehl CJ, Hausmann G, Brunner E, Basler K.   +7 more
europepmc   +1 more source

Seeing Invisible Oligomers: Rethinking α‐Synuclein Pathology Through Proximity Ligation Assay

Movement Disorders, EarlyView.
Abstract Parkinson's disease (PD) and multiple system atrophy are defined by α‐synuclein (αSYN)‐positive inclusions – Lewy bodies (LBs) and glial cytoplasmic inclusions – yet mounting evidence indicates that these inclusions represent only a fraction of disease‐relevant pathology.
Hiroaki Sekiya, Nanna Møller Jensen, Dennis W. Dickson, Poul Henning Jensen   +3 more
wiley   +1 more source

Pioneer Factor FOXA1 Boosts CHO Cell Productivity. [PDF]

Biotechnol J
Butterfield SP, Saunders FL, White RJ.
europepmc   +1 more source

Observations on an Open‐Label Phase 1/2 Dopamine Gene Therapy Trial (OXB‐102/Axo‐Lenti‐PD) in People with Parkinson's Disease

Movement Disorders, EarlyView.
Abstract Background SUNRISE‐PD was a dose‐escalating, phase 1/2 study investigating a second‐generation lentiviral vector gene therapy delivering the genes for dopamine synthesis (OXB‐102) to treat Parkinson's disease (PD). The trial was prematurely terminated due to insolvency of the sponsor.
Simon Rowe, Amy Evans, Saeed Kayhanian, Philip C. Buttery, Robert Morris, Katie Andresen, Laura Sherlock, Samuel Hewitt, Tagore Nakornchai, Marwan Hariz, Kyriacos Mitrophanous, Ludvic Zrinzo, Matthew Appleby, Phil Woodgate, Seema Maru, Patricia Limousin, Christine Girges, Thomas Foltynie, Stephane Palfi, Roger A. Barker   +19 more
wiley   +1 more source

Universal strategy to generate lentiviral vectors encoding lethal proteins. [PDF]

Mol Ther Adv
Versier B, Soundiramourty A, Manchon E, Ravassard P, Charron D, Christen Y, Mallet J, Al-Daccak R, Serguera C.   +8 more
europepmc   +1 more source

Gene Therapy for Amino Acid Decarboxylase Deficiency: Clinical and Imaging Outcomes in a French Cohort

Movement Disorders, EarlyView.
Abstract Background Intracerebral gene therapy is effective for amino acid decarboxylase (AADC) deficiency, but relationships between anatomical putaminal coverage, metabolic dynamics, and clinical recovery remain poorly understood. Objectives Assess safety, long‐term efficacy, and clinical–radiological correlations in a genetically diverse European ...
Clément Dunoyer, Gaëtan Poulen, Marie‐Céline François‐Heude, Eline Chauvet‐Piat, Bérénice Lecardonnel, Emmanuel Roze, Marie‐Ange Nguyen Morel, Domitille Gras, Adeline Quintard, Gaelle Baroux, Pierre Meyer, Philippe Coubes, Christophe Milesi, Julien Baleine, Chrystelle Sola, Vivien Szabo, Bénédicte Delye, Jana Haberlova, Mohamed Osman Eltahir Babiker, Mohammed Al Muqbil, Stéphanie Sanchez, Souad Touati, Nicolas Leboucq, Virginie Kouyoumdjian, Sylvia Sanquer, Denis Mariano‐Goulart, Thomas Roujeau, Agathe Roubertie   +27 more
wiley   +1 more source

Synergy between upregulated small GTPase immunity-associated proteins and lysosomal ATP6V1D in restricting intracellular <i>Toxoplasma</i> growth. [PDF]

Microbiol Spectr
Mia MM, Khan SM, Azam AR, Reilly J, Witola WH.   +4 more
europepmc   +1 more source

Muscle‐Specific Kinase Signaling and Its Therapeutic Potential

Muscle &Nerve, EarlyView.
ABSTRACT The function of the neuromuscular junction (NMJ) is compromised in many neuromuscular diseases (NMDs) such as autoimmune or congenital myasthenia gravis (MG), amyotrophic lateral sclerosis (ALS), spinal muscular atrophy (SMA), and muscular dystrophies.
Stine Marie Jensen, Dana L. E. Vergoossen, Maartje G. Huijbers   +2 more
wiley   +1 more source

Correction to 'Directed evolution of hyperactive integrases for site specific insertion of transgenes'. [PDF]

Nucleic Acids Res
europepmc   +1 more source

Specific pathogen free ten gene-edited donor pigs for xenotransplantation. [PDF]

Protein Cell
Xu K, Zhao H, Jia B, Wang J, Siddig NAMA, Jamal MA, Mao A, Liu K, Cheng W, Yang C, Wei T, Zhu F, Huo X, Jiao D, Guo J, Zhao H, Cheng W, Zhang Y, Zhang X, Jiang L, Zhang Z, Zhang W, Liang T, Zhao HY, Sun BC, Wei HJ.   +25 more
europepmc   +1 more source