Results 31 to 40 of about 11,459,182 (339)

Human Cytomegalovirus Induces Cellular and Humoral Virus-specific Immune Responses in Humanized BLT Mice

Scientific Reports, 2017
The strict species specificity of Human Cytomegalovirus (HCMV) has impeded our understanding of antiviral adaptive immune responses in the context of a human immune system.
Lindsey B. Crawford, Rebecca Tempel, Daniel N. Streblow, Craig Kreklywich, Patricia Smith, Louis J. Picker, Jay A. Nelson, Patrizia Caposio   +7 more
doaj   +1 more source

MicroRNA-7, synergizes with RORα, negatively controls the pathology of brain tissue inflammation

Journal of Neuroinflammation, 2020
Background Accumulating evidence has documented that microRNA-7 (miR-7) plays an important role in the pathology of various diseases. However, the potential role of miR-7 in brain tissue inflammation (BTI) remains unclear.
Dongxu Yue, Juanjuan Zhao, Huizi Chen, Mengmeng Guo, Chao Chen, Ya Zhou, Lin Xu   +6 more
doaj   +1 more source

Data collection infrastructure for patient outcomes in the UK – opportunities and challenges for cell and gene therapies launching

Journal of Market Access & Health Policy, 2019
Background: Cell and gene therapies are associated with uncertainty around their value claims at launch due to limitations of supporting clinical data; furthermore, their high costs present affordability issues for payers.
Jesper Jørgensen, Laura Mungapen, Panos Kefalas   +2 more
doaj   +1 more source

Impaired regeneration in calpain-3 null muscle is associated with perturbations in mTORC1 signaling and defective mitochondrial biogenesis

Skeletal Muscle, 2017
Background Previous studies in patients with limb-girdle muscular dystrophy type 2A (LGMD2A) have suggested that calpain-3 (CAPN3) mutations result in aberrant regeneration in muscle.
Mehmet E. Yalvac, Jakkrit Amornvit, Cilwyn Braganza, Lei Chen, Syed-Rehan A. Hussain, Kimberly M. Shontz, Chrystal L. Montgomery, Kevin M. Flanigan, Sarah Lewis, Zarife Sahenk   +9 more
doaj   +1 more source

Gene Therapy in Cancer Treatment: Why Go Nano?

Pharmaceutics, 2020
The proposal of gene therapy to tackle cancer development has been instrumental for the development of novel approaches and strategies to fight this disease, but the efficacy of the proposed strategies has still fallen short of delivering the full ...
C. Roma-Rodrigues, L. Rivas-García, P. Baptista, A. R. Fernandes   +3 more
semanticscholar   +1 more source

Functional roles of the membrane-associated AAV protein MAAP

Scientific Reports, 2021
With a limited coding capacity of 4.7 kb, adeno-associated virus (AAV) genome has evolved over-lapping genes to maximise the usage of its genome. An example is the recently found ORF in the cap gene, encoding membrane-associated accessory protein (MAAP),
Lionel Galibert, Amira Hyvönen, Reetta A. E. Eriksson, Salla Mattola, Vesa Aho, Sami Salminen, Justin D. Albers, Sanna K. Peltola, Saija Weman, Tiina Nieminen, Seppo Ylä-Herttuala, Hanna P. Lesch, Maija Vihinen-Ranta, Kari J. Airenne   +13 more
doaj   +1 more source

Alphaviruses in Gene Therapy [PDF]

Viruses, 2009
Alphaviruses are enveloped single stranded RNA viruses, which as gene therapy vectors provide high-level transient gene expression. Semliki Forest virus (SFV), Sindbis virus (SIN) and Venezuelan Equine Encephalitis (VEE) virus have been engineered as efficient replication-deficient and -competent expression vectors.
openaire   +7 more sources

Gene therapy

Biology of Blood and Marrow Transplantation, 1999
For the past two decades, concerted efforts have been made to treat human disease by replacing nonfunctioning genes in cells or by correcting mutations that produce disease. Successful application of these methods could lead to effective therapies for a variety of genetic and acquired diseases, many of which are not treatable today.
openaire   +2 more sources

Clinical development on the frontier: gene therapy for duchenne muscular dystrophy

Expert Opinion on Biological Therapy, 2020
Introduction: The development of adeno-associated virus (AAV) vectors as safe vehicles for in vivo delivery of therapeutic genes has been a major milestone in the advancement of gene therapy, enabling a promising strategy for ameliorating a wide range of
D. Asher, Khampaseuth Thapa, Sachi D. Dharia, N. Khan, R. Potter, L. Rodino-Klapac, J. Mendell   +6 more
semanticscholar   +1 more source

Comparison of two lab-scale protocols for enhanced mRNA-based CAR-T cell generation and functionality

Scientific Reports, 2023
Process development for transferring lab-scale research workflows to automated manufacturing procedures is critical for chimeric antigen receptor (CAR)-T cell therapies.
Nadine von Auw, Robert Serfling, Reni Kitte, Nadja Hilger, Chengkang Zhang, Clara Gebhardt, Anna Duenkel, Paul Franz, Ulrike Koehl, Stephan Fricke, U. Sandy Tretbar   +10 more
doaj   +1 more source