Results 31 to 40 of about 1,173,119 (313)

Establishing the cost of implementing a performance-based, managed entry agreement for a hypothetical CAR T-cell therapy

Journal of Market Access & Health Policy, 2018
Background: Market access stakeholders consider the adoption of Managed Entry Agreements (MEAs), however a clearly described methodology to quantify their implementation burden is not available in the public domain.
Panos Kefalas, Omar Ali, Jesper Jørgensen, Nick Merryfield, Tim Richardson, Adam Meads, Laura Mungapen, Matthew Durdy   +7 more
doaj   +1 more source

Universal Method for the Purification of Recombinant AAV Vectors of Differing Serotypes

Molecular Therapy: Methods & Clinical Development, 2018
The generation of clinical good manufacturing practices (GMP)-grade adeno-associated virus (AAV) vectors requires purification strategies that support the generation of vectors of high purity, and that exhibit a good safety and efficacy profile. To date,
Shelley A. Nass, Maryellen A. Mattingly, Denise A. Woodcock, Brenda L. Burnham, Jeffrey A. Ardinger, Shayla E. Osmond, Amy M. Frederick, Abraham Scaria, Seng H. Cheng, Catherine R. O’Riordan   +9 more
doaj   +1 more source

Idiotype vaccines produced with a non-cytopathic alphavirus self-amplifying RNA vector induce antitumor responses in a murine model of B-cell lymphoma

Scientific Reports, 2021
A promising therapy for patients with B-cell lymphoma is based on vaccination with idiotype monoclonal antibodies (mAbs). Since idiotypes are different in each tumor, a personalized vaccine has to be produced for each patient.
Erkuden Casales, Eva Martisova, Helena Villanueva, Ascensión López Díaz de Cerio, Susana Inoges, Noelia Silva-Pilipich, María Cristina Ballesteros-Briones, Alejandro Aranda, Jaione Bezunartea, Maurizio Bendandi, Fernando Pastor, Cristian Smerdou   +11 more
doaj   +1 more source

Evolutionary interplay between viruses and R‐loops

FEBS Letters, EarlyView.
Viruses interact with specialized nucleic acid structures called R‐loops to influence host transcription, epigenetic states, latency, and immune evasion. This Perspective examines the roles of R‐loops in viral replication, integration, and silencing, and how viruses co‐opt or avoid these structures.
Zsolt Karányi, Zita Képes, Zoltán Szabó, Eszter Csoma, Lóránt Székvölgyi   +4 more
wiley   +1 more source

Mesenchymal stem cell therapy for liver disease: full of chances and challenges

Cell & Bioscience, 2020
Liver disease is a major health problem that endangers human health worldwide. Currently, whole organ allograft transplantation is the gold standard for the treatment of end-stage liver disease.
Xue Yang, Yan Meng, Zhipeng Han, Fei Ye, Lixin Wei, Chen Zong   +5 more
doaj   +1 more source

Circulating histones as clinical biomarkers in critically ill conditions

FEBS Letters, EarlyView.
Circulating histones are emerging as promising biomarkers in critical illness due to their diagnostic, prognostic, and therapeutic potential. Detection methods such as ELISA and mass spectrometry provide reliable approaches for quantifying histone levels in plasma samples.
José Luis García‐Gimenez, Juan Carlos Ruiz‐Rodríguez, Ricard Ferrer, Raquel Durá, Antonio Artigas, Iván Bajaña, David Bolado López de Andujar, Irene Cánovas‐Cervera, Adrián Ceccato, Luis Chiscano‐Camón, Elena Climent‐Martinez, Georgia García Fernández, Gemma Goma, Verónica Monforte, Beatriz Quevedo‐Sánchez, Adolf Ruiz‐Sanmartín, Antonio Sierra‐Rivera, Nieves Carbonell Monleón   +17 more
wiley   +1 more source

Aurintricarboxylic acid increases yield of HSV-1 vectors

Molecular Therapy: Methods & Clinical Development, 2014
Production of large quantities of viral vectors is crucial for the success of gene therapy in the clinic. There is a need for higher titers of herpes simplex virus-1 (HSV-1) vectors both for therapeutic use as well as in the manufacturing of clinical ...
Peter Pechan, Jeffery Ardinger, Jyothi Ketavarapu, Hillard Rubin, Samuel C Wadsworth, Abraham Scaria   +5 more
doaj   +1 more source

Gene therapy for hemophilia [PDF]

Journal of Thrombosis and Haemostasis, 2013
Hemophilia A and B are X-linked monogenic disorders resulting from deficiencies of factor VIII and FIX, respectively. Purified clotting factor concentrates are currently intravenously administered to treat hemophilia, but this treatment is non-curative.
Thierry VandenDriessche, Thierry VandenDriessche, Hanneke Evens, Marinee Chuah   +3 more
openaire   +3 more sources

Single‐cell insights into the role of T cells in B‐cell malignancies

FEBS Letters, EarlyView.
Single‐cell technologies have transformed our understanding of T cell–tumor cell interactions in B‐cell malignancies, revealing new T‐cell subsets, functional states, and immune evasion mechanisms. This Review synthesizes these findings, highlighting the roles of T cells in pathogenesis, progression, and therapy response, and underscoring their ...
Laura Llaó‐Cid
wiley   +1 more source

Autoimmune dysregulation and purine metabolism in adenosine deaminase (ADA)-deficiency

Frontiers in Immunology, 2012
Genetic defects in the adenosine deaminase (ADA) gene are among the most common causes for severe combined immunodeficiency (SCID). ADA-SCID patients suffer from lymphopenia, severely impaired cellular and humoral immunity, failure to thrive and ...
Aisha Vanessa Sauer, Immacolata eBrigida, Nicola eCarriglio, Nicola eCarriglio, Alessandro eAiuti, Alessandro eAiuti   +5 more
doaj   +1 more source