-
Mashup Score: 3Preclinical safety and efficacy of lentiviral-mediated gene therapy for leukocyte adhesion deficiency type I - 1 year(s) ago
Severe LAD-I is a fatal primary immunodeficiency characterized by defective expression of the CD18 β2-integrin in PB leukocytes. To address an urgent medical need for these patients, our preclinical study demonstrates the safety and efficacy of a lentiviral-mediated gene therapy approach to support a new therapy for LAD-I patients.
Categories: Healthcare Professionals, Latest HeadlinesTweet
-
Mashup Score: 1Dasatinib is a potent enhancer for CAR TÂ cell generation by CD3-targeted lentiviral vectors - 1 year(s) ago
This paper describes the discovery of dasatinib as transduction enhancer for gene delivery into T lymphocytes by CD3-targeted lentiviral vectors. Dasatinib acts through preventing CD3:TCR intake, thereby increasing the amount of vector particles bound to the TÂ cell surface. Improved generation of CAR TÂ cells is illustrated as an immediate application.
Categories: Hem/Oncs, Latest HeadlinesTweet
-
Mashup Score: 0Rapid evolution of blood-brain-barrier-penetrating AAV capsids by RNA-driven biopanning - 1 year(s) ago
In this article, Nonnenmacher and colleagues establish TRACER, a cell-specific RNA-driven directed evolution platform for functional biopanning of adeno-associated virus (AAV) capsid libraries in animal tissue. Neuron- and glial-specific selection of an AAV9 library in mouse brain identified several AAV variants with unprecedented brain and spinal cord transduction.
Categories: General Medicine News, Latest HeadlinesTweet
-
Mashup Score: 0Mechanistic model for production of recombinant adeno-associated virus via triple transfection of HEK293 cells - 1 year(s) ago
A mechanistic model for recombinant adeno-associated virus viral vector production is developed to gain insights into the viral synthesis dynamics in non-viral transfection culture. Parameter estimation and validation of the model against experimental data reveal potential bottlenecks of the process, and potential process improvements are proposed.
Categories: General Medicine News, Latest HeadlinesTweet
-
Mashup Score: 2Analytical methods for process and product characterization of recombinant adeno-associated virus-based gene therapies - 1 year(s) ago
This review discusses analytical methods for the characterization of rAAV-based products during their development. With the focus on vector titer, content ratio, and aggregation, the review assesses established and upcoming methods and serves as a guide for the selection of analytical methods for rAAV development.
Categories: General Medicine News, Latest HeadlinesTweet
-
Mashup Score: 1LPA disruption with AAV-CRISPR potently lowers plasma apo(a) in transgenic mouse model: A proof-of-concept study. - 2 year(s) ago
Doerfler and Park et al. show that editing of the LPA gene is a promising therapeutic approach to lower Lipoprotein(a), a circulating lipid particle strongly associated with heart disease. AAV delivery of CRISPR/Cas9 nearly completely removed apolipoprotein(a) levels in a transgenic mouse model with a single treatment.
Categories: Cardiologists, Latest HeadlinesTweet
-
Mashup Score: 0
Animal models are crucial for the development and improvement of adoptive cellular therapies, such as CAR-T cells. Murine xenografts, transgenic mouse models, and non-human primate models have proven essential. Every model system has inherent advantages and disadvantages that must be weighed when considering a given question.
Categories: Hem/Oncs, Latest HeadlinesTweet
-
Mashup Score: 0A chimeric virus-based probe unambiguously detects live circulating tumor cells with high specificity and sensitivity - 2 year(s) ago
A hybrid virus probe is designed that incorporates components from both human papillomavirus (HPV) and SV40, with the former providing epithelium-specific infection for a highly selective entry of the probe into circulating tumor cells (CTCs) and the latter providing amplification for sensitive detection. Most importantly, such a detection method ensures that all the detected CTCs are alive.
Categories: Latest Headlines, Oncologists2Tweet
-
Mashup Score: 0Quantification of cell-free DNA for the analysis of CD19-CAR-T cells during lymphoma treatment - 2 year(s) ago
The study proves the feasibility to quantify cell-free CAR-DNA in lymphoma patients during CAR-T cell treatment by a novel digital-droplet PCR assay. Combined analysis of cell-free CAR-DNA and reference cell-free DNA displays CAR-T cell-mediated lymphoma killing in individual patients.
Categories: Hem/Oncs, Latest HeadlinesTweet
-
Mashup Score: 0Quantification of cell-free DNA for the analysis of CD19-CAR-T cells during lymphoma treatment - 2 year(s) ago
The study proves the feasibility to quantify cell-free CAR-DNA in lymphoma patients during CAR-T cell treatment by a novel digital-droplet PCR assay. Combined analysis of cell-free CAR-DNA and reference cell-free DNA displays CAR-T cell-mediated lymphoma killing in individual patients.
Categories: Hem/Onc News and Journals, Latest HeadlinesTweet
RT @Primary_Immune: A promising gene 🧬therapy for Leukocyte Adhesion Deficiency type I (LAD-I) https://t.co/PorLVhiFQt #openscience