{"id":13979,"date":"2025-04-14T22:00:00","date_gmt":"2025-04-14T22:00:00","guid":{"rendered":"https:\/\/modernsciences.org\/staging\/4414\/?p=13979"},"modified":"2025-04-04T08:45:05","modified_gmt":"2025-04-04T08:45:05","slug":"gene-therapy-blindness-curiosity-driven-research-medical-breakthrough-april-2025","status":"publish","type":"post","link":"https:\/\/modernsciences.org\/staging\/4414\/gene-therapy-blindness-curiosity-driven-research-medical-breakthrough-april-2025\/","title":{"rendered":"\u2018Curiosity-driven\u00a0research\u2019 led to a recent major medical breakthrough. But it\u2019s under\u00a0threat"},"content":{"rendered":"\n\n
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\n \n Hakase_420\/Shutterstock<\/a><\/span>\n <\/figcaption>\n <\/figure>\n\n Sean Coakley<\/a>, The University of Queensland<\/a><\/em><\/span>\n\n

Earlier this year news broke about doctors in London curing blindness in children with a rare genetic condition<\/a>. <\/p>\n\n

The genetic condition was a severe, albeit rare, form of retinal dystrophy. It causes severe sight impairment and can be caused by defects in many different genes. <\/p>\n\n

In this case, the four young patients had mutations in the gene encoding AIPL1. This accounts for up to 5% of infants affected by this condition, and has no treatment.<\/p>\n\n

In this study, published in The Lancet<\/a>, a team from the Moorfields Eye Hospital and University College London Institute of Ophthalmology<\/a> injected a new copy of the gene AIPL1 into one eye of each patient to replace the defective one. The four children in the study showed improved functional vision without serious adverse effects. <\/p>\n\n

The story of this incredible breakthrough actually begins 132 years ago. It highlights the importance of research done not for any clear application in the world \u2013 just curiosity. But around the world, this kind of research is under threat<\/a>. <\/p>\n\n

Understanding the world \u2013 just for the sake of it<\/h2>\n\n

Curiosity-driven research is exactly what it sounds like: research driven by the goal of understanding nature without regard for application. It has many aliases. \u201cBlue-sky research\u201d, \u201cdiscovery science\u201d and \u201cbasic science\u201d are all terms commonly used to describe this approach. <\/p>\n\n

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