Gene Therapy Vectors Articles & Analysis
11 articles found
l Complex product formats: Bispecific antibodies, antibody-drug conjugates, and fusion proteins introduce unique purification challenges. l Gene and cell therapies: Large biomolecules like adeno-associated virus (AAV) or lentiviral vectors are difficult to purify at scale. ...
AAV vectors are used as delivery vehicles or "vectors" to administer therapeutic genes into patient's cells. ...
Two types of gene therapy have the potential to treat CF, including non-integrated and integrated gene therapy. ...
Adeno-associated viruses (AAV) have emerged as safe and effective vectors for gene therapy and the advancement of biopharmaceutical research. ...
In many cases, gene therapy requires a vector to deliver the gene therapy drug to the target cell. ...
Why need viral vector characterization? Cell and gene therapies for various rare diseases are currently undergoing clinical trials worldwide. The rapid development in this field has led to an increase in regulatory scrutiny and product characterization requirements, as well as a bottleneck in viral vector supply. The ...
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. ...
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. ...
The growing pipeline of cell and gene therapy therapies has led to an increase in demand for gene delivery vehicles such as plasmid DNA (pDNA), messenger RNA (mRNA) and viral vectors. ...
This post (the first in a three part series on AAV) provides an overview of AAV as a gene therapy vector, focusing primarily on the genetic and protein structure of an AAV capsid. ...
A key hurdle to making adeno-associated virus (AAV) capsid mediated gene therapy broadly beneficial to all patients is overcoming pre-existing and therapy-induced immune responses to these vectors. ...