Nanoparticles have been used to deliver gene therapy to treat age-related macular degeneration (AMD) in mice and rats.
As reported in ScienceDaily, the Johns Hopkins University (JHU) investigators used a uniquely engineered large molecule that facilitated compaction of large bundles of therapeutic DNA to be delivered into the ocular cells.
This approach does not depend on viral vectors, as many of the gene therapies do, to transport the needed material into the cells.
The downside of viral vectors is that an immune response is produced, and dosing cannot be repeated. In addition, the one used most often for ocular gene therapy cannot carry large genes, the investigators pointed out… Continue reading.
Using an exclusively designed large molecule, the researchers could compact huge bundles of therapeutic DNA to be delivered into the cells of the eye.
Reported in the Science Advances journal on July 3rd, 2020, the study offers evidence of the prospective value of nanoparticle-delivered gene therapy for the treatment of wet age-related macular degeneration.
Macular degeneration is an eye disease in which blood vessel growth is abnormal, causing damage to the light-sensitive tissue at the back of the eye, together with rarer, inherited blinding diseases of the retina… Continue reading.
If you dispatch a suicide gene, you want to make sure that it bypasses healthy cells on its way to harmful cells, such as cancer cells. What’s more, you want to make sure that the suicide gene is sent via a delivery system that treads lightly—especially if the suicide gene is meant to treat pediatric patients, who have relatively fragile immune systems. Unfortunately, pediatric patients may have difficulty tolerating the most common gene delivery systems, which are derived from viruses.
To build a targetable and relatively innocuous delivery system, scientists based at the Johns Hopkins University School of Medicine turned to nanotechnology. Basically, they developed a library of poly(beta-amino ester) nanoparticles, or PBAEs. These tiny delivery vehicles consist of biodegradable, cationic polymers, and they self-assemble with nucleic acids… Continue reading.
WASHINGTON, D.C.— The American Institute for Medical and Biological Engineering (AIMBE) has announced the pending induction of Jordan J. Green, Ph.D., Associate Professor, Department of Biomedical Engineering, Johns Hopkins University, to its College of Fellows. Dr. Green was nominated, reviewed, and elected by peers and members of the College of Fellows For outstanding contributions to bioengineering including innovations in nanobiotechnology, biomimetic materials, and cellular engineering.