Researchers test novel approach for injecting gene therapy vectors into kidney
A mouse-experiment conducted by the researchers to better understand the ways to treat renal diseases has identified that before gene therapy is implemented to treat, targeted delivery of the therapeutic genes to the kidney should be the 1st necessary step.
In a novel approach devised by a group of scientists, 3 different gene delivery vectors were injected intravenously and directly into the kidneys of mice.
As the kidney filters out large compounds from the bloodstream, the researchers chose to study the ability to deliver 3 different sized vectors via an intravenous route: small adeno-associated virus (AAV) vectors (25mm), larger adenovirus vectors (100mm) and lentiviral vectors (120mm).
To bypass this filtering mechanism, they also tested 2 different direct injection routed into the kidney and found these to be superior to intravenous injections.
However, some of the vectors were able to leak out of the kidney, creating the possibility for off-target tissue effects.
The potential for direct injections opens new possibilities for treating kidney diseases with gene therapy, but additional improvements are needed.
The great burden of kidney diseases in the U.S. and Europe has yet to be impacted by gene therapy, said one of the researchers.
The researchers have performed an important head-to-head comparison of currently available gene therapy technology, to identify which may best be used to address this important group of diseases.
In a novel approach devised by a group of scientists, 3 different gene delivery vectors were injected intravenously and directly into the kidneys of mice.
As the kidney filters out large compounds from the bloodstream, the researchers chose to study the ability to deliver 3 different sized vectors via an intravenous route: small adeno-associated virus (AAV) vectors (25mm), larger adenovirus vectors (100mm) and lentiviral vectors (120mm).
To bypass this filtering mechanism, they also tested 2 different direct injection routed into the kidney and found these to be superior to intravenous injections.
However, some of the vectors were able to leak out of the kidney, creating the possibility for off-target tissue effects.
The potential for direct injections opens new possibilities for treating kidney diseases with gene therapy, but additional improvements are needed.
The great burden of kidney diseases in the U.S. and Europe has yet to be impacted by gene therapy, said one of the researchers.
The researchers have performed an important head-to-head comparison of currently available gene therapy technology, to identify which may best be used to address this important group of diseases.