Results 31 to 40 of about 89,643 (298)

Emerging Concepts and Challenges in Rheumatoid Arthritis Gene Therapy

Biomedicines, 2020
Rheumatoid arthritis (RA) is a systemic inflammatory joint disease affecting about 1% of the population worldwide. Current treatment approaches do not ensure a cure for every patient.
Andrei A. Deviatkin, Yulia A. Vakulenko, Ludmila V. Akhmadishina, Vadim V. Tarasov, Marina I. Beloukhova, Andrey A. Zamyatnin Jr., Alexander N. Lukashev   +6 more
doaj   +1 more source

The Potential of eCD4-Ig, Delivered by Adeno-Associated Virus (AAV) Vector as a Novel Vaccine for HIV/AIDS Infection

Scripta Score Scientific Medical Journal, 2021
Background: HIV/AIDS has already become one of the world's major health issues taking its toll on millions of lives each year. Developing an HIV vaccine with excellent efficacy has become a global urgency that must be addressed immediately.
Ghea Mangkuliguna
doaj   +1 more source

Wild type AAV, recombinant AAV, and Adenovirus super infection impact on AAV vector mobilization [PDF]

, 2020
Abstract Recombinant Adeno-associated viral vector (rAAV) mobilization is a largely theoretical process in which intact AAV vectors spread or “mobilize” from transduced cells and infect additional cells within, or external, of the initial host. This process can be replication independent (vector alone), or replication-
Song, Liujiang, Samulski, R. Jude, Hirsch, Matthew L   +2 more
openaire   +1 more source

AAV-expressed serum mAb retains full neutralization activity.

, 2018
(A) Standard neutralization regression analyses are shown for ITS01 (upper) and ITS06.02 (lower) from purified mAb (red, open circles) or sera from AAV injected rhesus macaques (blue, open squares) (n = 6).
Rosemarie D. Mason (90621), Yi Zhang (9093), Sandeep Narpala (517528), Lingshu Wang (3204129), Madeleine F. Jennewein (2564638), Cheng Cheng (68821), Galit Alter (91383), Hugh C. Welles (3174969), Alejandro B. Balazs (244312), Jeffrey D. Lifson (140183), John R. Mascola (229761), Mario Roederer (5259), John-Paul Todd (6054251), Adrian B. McDermott (635782)   +13 more
core   +1 more source

AAV-HGFK1 and Ad-p53 cocktail therapy prolongs survival of mice with colon cancer [PDF]

, 2008
This study tried to evaluate the application of a novel cancer gene therapy using recombinant adeno-associated virus (AAV) carrying the kringle 1 domain of human hepatocyte growth factor (AAV-HGFK1) in combination with recombinant adenovirus carrying p53
B. Nie, Hsueh, WD, Huo, LF, M. C.M. Lin, Wong, OGW, Lin, MCM, O. G.-W. Wong, B. Jiang, W. D. Hsueh, Kung, HF, Z. Shen, Nie, B, Wen, JB, H.-F. Kung, Shen, Z, Jiang, B, J.-B. Wen, L.-F. Huo   +17 more
core   +1 more source

Clinical Variability and Genotype Driven Outcomes in an International Cohort of 14 Pediatric FIG4 Patients

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Background Biallelic FIG4 variants classically cause distinct conditions characterized by peripheral neuropathy (Charcot‐Marie‐Tooth disease type 4J) or skeletal dysplasia (Yunis‐Varón syndrome). However, central nervous system involvement is increasingly recognized. The prevalence and specific characteristics of movement disorders within FIG4‐
Andrea Gangfuß, Vykuntaraju K. Gowda, Daniel Natera‐de Benito, Pinki Munot, Maria Vanegas, Alejandra Darling, Constanze Reutlinger, Laura Carrera‐Garcia, Gilbert Wunderlich, Heike Kölbel, Burkhard Stüve, David Muhmann, Lucía Monfort Belenguer, Diana‐Gabriela Barca, Viveka Santhosh Reddy C, Varunvenkat M. Srinivasan, Cristina Molina Anguita, Heinz Jungbluth, Hormos Salimi Dafsari, Thomas Rossor, Johann Böhm, Ulrike Schara‐Schmidt, Andreas Roos   +22 more
wiley   +1 more source

AAV-Tis21 infects DAOY and D283 MB cell lines and decreases Cyclin D1 mRNA expression levels in Tis21 dose-dependent manner.

, 2018
Real-time PCR analysis of mRNA obtained from DAOY (A and B) and D283 (C and D) cells infected with AAV overexpressing Tis21 (AAV-Tis21) or AAV-CBA (control). Increasing MOI employed for infection is indicated in graphs.
Manuela Ceccarelli (4398151), Giuliana Papoff (688457), Simone Samperna (828290), Felice Tirone (256018), Simonetta Santini (828289), Laura Micheli (256012), Cristiana Lalli (687846), Lorena Zentilin (1933051), Dario Presutti (828288), Giovina Ruberti (687850)   +9 more
core   +1 more source

Race and Household Income Are Associated With Disease Outcomes in Pediatric Patients With Antineutrophil Cytoplasmic Antibody–Associated Vasculitis Hospitalized in the United States

Arthritis Care &Research, EarlyView.
Objective Race and household income impact outcomes in patients with rheumatic conditions; however, their role in pediatric antineutrophil cytoplasmic antibody (ANCA)–associated vasculitis (AAV) remains poorly understood. We aimed to evaluate whether race and ethnicity and household income are associated with severe AAV disease and renal outcomes among
Roberto Alejandro Valdovinos, William Daniel Soulsby III, Emily von Scheven   +2 more
wiley   +1 more source

Recombinant human myelin-associated glycoprotein promoter drives selective AAV-mediated transgene expression in oligodendrocytes

Frontiers in Molecular Neuroscience, 2016
Leukodystrophies are hereditary central white matter disorders caused by oligodendrocyte dysfunction. Recent clinical trials for some of these devastating neurological conditions have employed an ex vivo gene therapy approach that showed improved ...
Georg evon Jonquieres, Claudia Bettina Klugmann, Dominik eFröhlich, Xin eWen, Anne Editha Harasta, Roshini eRamkumar, Ziggy HT Spencer, Gary D Housley, Matthias eKlugmann   +8 more
doaj   +1 more source

An Epigenetic Fate Converter Based on Nuclei‐Targeting Lipid Nanoparticles Drives Neuronal Programming of Stem Cells for Spinal Cord Repair

Advanced Functional Materials, EarlyView.
Schematic illustration of LNP‐MPG nuclei‐targeting delivery of HMW‐FGF2 promoting histone acetylation to regulate the fate of DPSCs and treat spinal cord injury. LNPs components include pHMW‐FGF2 plasmid, DSPC, Dlin‐MC3‐DMA, cholesterol, and PEG2000, and are modified with MPG to form HMW‐FGF2@LNP‐MPG (HLM). HLM nuclei‐targets DPSCs to deliver HMW‐FGF2,
Heng Zhou, Qiang Qin, Xiqin Li, Hanyu Ding, Chun Xu, Yan He, Qingsong Ye   +6 more
wiley   +1 more source