Researchers have discovered a new way to treat central nervous system diseases with genes

Wuhan, Oct.
26 (ZXN) -- A variant of adeno-associated virus called AAV.
CPP.
16 can effectively pass through the blood-brain barrier of the central nervous system, deliver drugs to the brain and spinal cord, and effectively treat experimental mice
with malignant gliomas.
Recently, a study published in Nature Biomedical Engineering provides a new way
for gene therapy for central nervous system diseases.

Wang Jun, co-first author of the paper and deputy chief physician of the Department of Neurosurgery, Neuropsychiatric Hospital, People's Hospital of Wuhan University, introduced on the 26th that adeno-associated virus (AAV) is a small virus that is currently the main platform
for gene therapy delivery in vivo.
However, the low efficiency of gene vector delivery is an important bottleneck
hindering the development of AAV-mediated gene therapy for the central nervous system.

"The inefficient delivery of gene vectors is related
to the blood-brain barrier.
" Wang Jun explained that the blood-brain barrier is made up of tightly wedged cells that prevent toxins and pathogens that may be present in the blood from entering brain tissue, but it is also a major obstacle
to gene therapy drugs entering the central nervous system.

In the study, the research team screened out adeno-associated virus variants AAV.
CPP.
16
with high blood-brain barrier penetration through design and modification.
By injecting AAV.
CPP.
16 carrying the gene of interest in animal models of different strains of mice, juvenile and adult cynomolgus monkeys, it was confirmed that compared with other AAV variants, AAV.
CPP.
16 crossed the blood-brain barrier with significantly improved transmission efficiency, and the efficient and safe expression
of the gene of interest was also achieved in the central nervous system of primate cynomolgus monkeys.

According to reports, the research results have brought the successful transformation of AAV delivery drugs through the human blood-brain barrier, and also show that AAV can be used as an effective systemic drug delivery tool to fight gliomas or other central nervous system diseases
in need.
(End)