Gene Therapy Drug Articles & Analysis
4 articles found
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), ...
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 ...
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. ...