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Gene Therapy Drug Articles & Analysis
8 articles found
While the reduction was statistically significant, it remained within clinically acceptable limits, suggesting the cartoon superimposed on the pattern stimulus can be suitable for testing young subjects.Pattern VEPs for gene therapy monitoring in young childrenThe ophthalmology field views FDA approval of Luxturna in 2017 and EMA approval in 2018 as landmark ...
Excitingly, recent advancements in gene engineering and drug R&D have led to the development of rare disease gene therapy and small-molecule drugs. Commonly, gene engineering technologies used in gene therapy include CRISPR/Cas9, zinc finger nucleases (ZFNs), ...
Hydrophilic drugs stay in the aqueous core, while hydrophobic drugs integrate into the lipid bilayer. ...
These treatments include antibiotics to treat lung infections, airway clearance techniques to clear mucus from the lungs, nutritional support, and medications that target specific mutations in the CFTR gene. Multiple clinical trials are currently underway to test new therapies for CF, including gene therapy and ...
In many cases, gene therapy requires a vector to deliver the gene therapy drug to the target cell. Viral vectors have been one of the most widely studied vectors due to their outstanding transduction efficiency and other significant advantages. Viral vector-based gene therapy has ...
Critical stages of the viral life cycle are appealing targets for drugs and therapies, making basic research to understand and affect the underlying molecular mechanisms all the more important. ...
However, these drugs have to bind to the active site of target proteins to work. Among the known drug target proteins, only about 20% of the target proteins contain bindable active site, and 80% of the target proteins are non-druggable targets. How to develop drugs by acting on the vast majority of non-druggable targets has become the research ...
and what makes them good candidates for gene therapy? Introduction In the first part of our three part series introducing AAV as a gene therapy vector, we talked about basic AAV vector biology. ...