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The biggest challenge faced by the new technology and new target of gene therapy is how to accurately regenerate neurons, and how to make them accurately link with the original neural tissue. Studies have shown that many regenerative neurons appear tortuous in the optic nerve and even regenerate in the opposite direction.

"Now, it is unclear whether these erroneously regenerated neurons can link to the optic nerve and thus produce erroneous visual functions," said a senior scientist at Creative Biolabs, a leading service provider of gene therapy. "Non-viral vector gene therapy with low toxicity but limited efficiency may have a certain therapeutic effect, but this treatment method has not been applied to the treatment of hereditary optic nerve diseases."

Targeted gene therapy for hereditary optic nerve diseases has shown certain therapeutic effects in animal model experiments of optic nerve damage and early human experiments. However, there are still some problems that need to be solved, and scientists worldwide are trying to unveil more secret of genomes.