Article: Blind Patients Hope Landmark Gene-Editing Experiment Will Restore Their Vision
Source: NPR, via Genetic Literacy Project
Published: May 10, 2021
Article: Gene editing clinical trial participant dreams of a future with sight
Source: Oregon Health & Science University
Published: May 10, 2021 (added August 6, 2021)
NPR interviewed two patients with Leber congenital amaurosis (LCA) who participated in a landmark study using CRISPR gene editing. It was the first study to deliver CRISPR for gene editing inside the human body. Specifically, the two patients, Carlene Knight and Michael Kalberer, are affected by a version of LCA caused by a mutation in the CEP290 gene, affecting the photoreceptors of the eye. Both are legally blind with very limited tunnel vision centrally; Knight additionally has nystagmus. LCA was a suitable disease to test in vivo applications of CRISPR gene editing for two reasons: The retina is too fragile a tissue to remove, edit in vitro and then return to the eye. Furthermore, the healthy version of the gene is too large to use with viral vectors in traditional gene therapy. Instead, viral vectors, injected subretinally, were engineered to carry genetic instructions to manufacture the CRISPR gene-editor inside the retina. As a point of scientific interest, the news article also mentions safety precautions such as using the lowest number of viruses, beginning the clinical trials with older patients whose vision was already extensively damaged, and treating only one eye in each patient. At the same time, there was much consideration for the possibility that these patients would lose the little bit of vision they had remaining. The researchers have treated a total of four patients, and hope to add more with a wider age range. At this point in time, the effect of the treatment is yet to be seen. However, as Kalberer says, "But to even have the possibility — it's a gift."
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