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The top academic journal "Cell" recently published a new study on gene therapy from Professor Bo Chen's team at Icahn School of Medicine at Mount Sinai, which has attracted widespread attention
.
The research provides a new way to the light for the treatment of common blinding diseases such as glaucoma and diabetic retinopathy, and is expected to prevent vision loss
This gene therapy reactivates a key enzyme called CaMKII in retinal ganglion cells (RGC), which can provide strong protection to damaged retinal ganglion cells
.
Retinal ganglion cells are nerve cells that send image information to the brain.
The optic nerve, composed of their axons, extends from the retina to the brain like a cable
.
When the retina is damaged or pathological changes occur, retinal ganglion cells will degenerate
▲The optic nerve composed of axons of retinal ganglion cells transmits visual signals to the brain's visual processing center (picture source: reference material [3]; Credit: National Eye Institute)
"In order to preserve vision, we urgently need strategies that can protect nerves to retain fragile retinal ganglion cells
.
" Professor Bo Chen believes, "We have shown for the first time through evidence that CaMKII is retinal nerve in both normal and diseased retinas.
The full name of CaMKII is calcium/calmodulin-dependent protein kinase II.
This signaling pathway regulates key cellular processes and functions in many types of cells
.
However, what role CaMKII plays for the health of retinal ganglion cells was not very clear in the past
Researchers examined the effects of this enzyme in a variety of animal models of retinal diseases and injuries
.
Experimental results show that when retinal ganglion cells are exposed to toxins (acute injury) or the optic nerve is squeezed (slower injury), CaMKII and its downstream signaling molecule CREB will be inactivated, indicating that CaMKII activity and retinal ganglion cells The survival of there is a correlation
Based on this discovery, the researchers designed a gene therapy that uses adeno-associated virus (AAV) as a vector to bring a modified version of CaMKII with enhanced activity into retinal ganglion cells
.
▲Gene therapy protects damaged retinal ganglion cells by reactivating CaMKII (picture source: reference [1])
In mice receiving gene therapy after acute retinal injury, 77% of retinal ganglion cells still survived 12 months later, compared to only 8% of mice in the control group
.
Similarly, in mice whose optic nerve was squeezed, the survival rate of retinal ganglion cells increased from 7% to 77% 6 months after receiving gene therapy
.
This gene therapy has also been verified in two glaucoma model mice to delay disease progression and prevent vision loss
.
Due to increased intraocular pressure or genetic defects, the retinal ganglion cells of these mice degenerate
Reference materials:
[1] Xinzheng Guo et al.
[2] Gene therapy may preserve vision inretinal disease and serious retinal injury.
