he tumor-associated immunosuppression, as a key barrier, prevents the immunotherapy withstanding tumors. Here an ingenious “nanoconverter” was designed to convert immunosuppression to immunoactivation, which was C6-ceramide (C6) modified tumor cytomembrane...
Purpose
The aim of study was to establish
Rdh12
-associated inherited retinal disease (
Rdh12
-IRD) mouse model and to identify the best timepoint for gene therapy.
Methods
We induced retinal degeneration in
Rdh12
−/−
mice using a bright light. We clarified the establishment of
Rdh12
-IRD mouse model by analyzing the thickness of retinal layers and electroretinography (ERG).
Rdh12
-IRD mice received a subretinal injection of adeno-associated virus 2/8-packaged
Rdh12
cDNA for treatment. We evaluated the visual function and retinal structure in the treated and untreated eyes to identify the best timepoint for gene therapy.
Results
Rdh12
-IRD mice showed significant differences in ERG amplitudes and photoreceptor survival compared to
Rdh12
+/+
mice. Preventive gene therapy not only maintained normal visual function but also prevented photoreceptor loss. Salvage gene therapy could not reverse the retinal degeneration phenotype of
Rdh12
-IRD mice, but it could slow down the loss of visual function.
Conclusion
The light-induced retinal degeneration in our
Rdh12
−/−
mice indicated that a defect in
Rdh12
alone was sufficient to cause visual dysfunction and photoreceptor degeneration, which reproduced the phenotypes observed in
RDH12
-IRD patients. This model is suitable for gene therapy studies. Early treatment of the primary
Rdh12
defect helps to delay the later onset of photoreceptor degeneration and maintains visual function in
Rdh12
-IRD mice.
Herein, the cobalt-involved redox in a magnetic drug-loaded nanocatalyst (PTX/Co-Lips@Fe3O4) was used to convert Fe(III) to Fe(II) for amplification of tumor ferrotherapy for the first time. Moreover, this work highlighted...
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