Sep. 9, 2024 — Researchers tested a preclinical model for an experimental gene therapy designed to treat multiple sulfatase deficiency (MSD), a disorder that affects the brain, lungs ...
However, there are few theories and little empirical evidence to guide investigations in this area. To advance this emerging area of inquiry, we present a conceptual model that synthesizes evidence, ...
22, 2024 — Geneticists have developed a gene drive-based solution to the widespread ... scientists rely on the mouse as a model organism. Researchers have now developed a new method that is ...
Investigators from New York State Office for People with Developmental Disabilities (OPWDD) and affiliated organizations have reported the discovery and preclinical characterization of a gene therapy ...
demonstrating the use of SPLICER for skipping APP exon 17 in a mouse model of Alzheimer’s disease. In this paper, a CRISPR-based gene addition strategy at the CX3CR1 locus of human hematopoietic ...
and the performance of clinical trials are of particular relevance when focusing on ways to turn cardiovascular gene therapy, stem cell therapy, or both, into reality. These exciting therapeutic ...
With cell and gene therapies growing more and more important ... as they become more numerous in the marketplace. The current model for paying for treatment in the US is buy-and-bill, in which ...
The company, which secured $200 million from investors in a private funding deal last week, learned Tuesday of a serious side effect experienced by one participant on a high dose of its Rett therapy.
Published recently in Nature Communications, their findings introduce a new conceptual model that could deepen our grasp of these unique electrical events. A new conceptual model of cloud top ...