Results 31 to 40 of about 1,204,526 (212)

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

Systemic γ-sarcoglycan AAV gene transfer results in dose-dependent correction of muscle deficits in the LGMD 2C/R5 mouse model

Molecular Therapy: Methods & Clinical Development, 2023
Limb-girdle muscular dystrophy (LGMD) type 2C/R5 results from mutations in the γ-sarcoglycan (SGCG) gene and is characterized by muscle weakness and progressive wasting.
Young-Eun Seo, Stephen H. Baine, Amber N. Kempton, Oliver C. Rogers, Sarah Lewis, Kaitlin Adegboye, Alex Haile, Danielle A. Griffin, Ellyn L. Peterson, Eric R. Pozsgai, Rachael A. Potter, Louise R. Rodino-Klapac   +11 more
doaj   +1 more source

Application of Raman Spectroscopy and Univariate Modelling As a Process Analytical Technology for Cell Therapy Bioprocessing

Frontiers in Medicine, 2018
Cell therapies offer unquestionable promises for the treatment, and in some cases even the cure, of complex diseases. As we start to see more of these therapies gaining market authorization, attention is turning to the bioprocesses used for their ...
Marc-Olivier Baradez, Daniela Biziato, Enas Hassan, Damian Marshall   +3 more
doaj   +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

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

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

Applications of Ultrasound-Mediated Drug Delivery and Gene Therapy [PDF]

, 2021
Juliana Sitta, Candace M. Howard
openalex   +1 more source

FGF21 gene therapy as treatment for obesity and insulin resistance

EMBO Molecular Medicine, 2018
Prevalence of type 2 diabetes (T2D) and obesity is increasing worldwide. Currently available therapies are not suited for all patients in the heterogeneous obese/T2D population, hence the need for novel treatments.
Veronica Jimenez, Claudia Jambrina, Estefania Casana, Victor Sacristan, Sergio Muñoz, Sara Darriba, Jordi Rodó, Cristina Mallol, Miquel Garcia, Xavier León, Sara Marcó, Albert Ribera, Ivet Elias, Alba Casellas, Ignasi Grass, Gemma Elias, Tura Ferré, Sandra Motas, Sylvie Franckhauser, Francisca Mulero, Marc Navarro, Virginia Haurigot, Jesus Ruberte, Fatima Bosch   +23 more
doaj   +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

ITR-Seq, a next-generation sequencing assay, identifies genome-wide DNA editing sites in vivo following adeno-associated viral vector-mediated genome editing

BMC Genomics, 2020
Background Identifying nuclease-induced double-stranded breaks in DNA on a genome-wide scale is critical for assessing the safety and efficacy of genome editing therapies.
Camilo Breton, Peter M. Clark, Lili Wang, Jenny A. Greig, James M. Wilson   +4 more
doaj   +1 more source