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Ishita Roy

How A 2-Year-Old Connecticut Toddler Got His Vision Back With This Breakthrough Gene Therapy

Jace, 2-year-old toddler who got his vision back (credits: Moorfields Hospital)

Science is a lot about experiments, sometimes it goes right, other times it doesn't. One such experimental <span>gene therapy</span> has shown promising results in helping toddlers born with one of the most severe forms of childhood blindness regain significant vision improvements. This trial was conducted by doctors at the Moorfields Eye Hospital in London, the trial focused on children with an aggressive form of Leber Congenital Amaurosis (LCA), a rare genetic disorder that causes rapid vision loss from birth. Before the therapy, the affected children were legally blind, only able to distinguish between light and dark. However, after receiving the treatment, all four toddlers demonstrated remarkable progress—some even beginning to draw and write.

The findings were published in the Lancet medical journal titled Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study. The gene therapy of another form of genetic blindness is also available on the UK National Health Service, since 2020, and this work just builds upon that. 

The findings thus provide hope for future treatments of genetic blindness. 

<h4>How Does This Gene Therapy Work?</h4>

The therapy is innovative in its own way and it involves injecting healthy copies of the AIPL 1 gene into the retina at the back of the eye. The scientists at the University College London developed this method and the procedure was performed by specialists at Great Ormond Street Hospital. The healthy gene copies are delivered using a harmless virus that carries them into the retinal cells, replacing the defective gene. This process also helps the cells function properly, slowing down degeneration and improving vision. 

Unlike the traditional clinical trials, in this trial, the therapy was offered under a special compassionate-use license. This is because no other treatment options had existed for these children and thus each child received the therapy in only one eye to minimize any potential risk. 

<h4>Case Study of Jace From Connecticut</h4>

Jace, who is a toddler from Connecticut, was among the four children who received the treatment, reports the BBC. His parents noticed early signs of visual impairments when he failed to make eye contact or smile when he was as young as just 8 weeks old. After months of medical consultations, he was diagnosed with the ultra-rare genetic condition. When his parents got to know about the London trial, they thought of taking a chance. Jace was then two years old, and underwent the procedure. His mother said that the surgery was quick and minimally invasive, leaving only four tiny scars on his eyes. 

Within a month, the family was able to notice changes in Jace's responses. He now squinted in bright sunlight, which he has not done before, his vision also not improved. Before surgery, he could not track objects near his face, now he can pick up small toys from the floor and navigate his surroundings. Jace may require further treatments in future, but this, so far, has been life-changing, for both him and his parents. 

Prof. James Bainbridge, a retinal surgeon at Moorfields Eye Hospital, emphasized how crucial early intervention is for children with severe sight impairment. “Treatment in infancy with this new genetic medicine can transform the lives of those most severely affected,” he stated.

The trial results are compelling. The vision in the treated eyes improved, while the untreated eyes continued to deteriorate as expected. Consultant eye surgeon Prof. Michel Michaelides described the outcomes as "hugely impressive," showcasing the power of gene therapy in changing lives.

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Science is a lot about experiments, sometimes it goes right, other times it doesn't. One such experimental gene therapy has shown promising results in helping toddlers born with one of the most severe forms of childhood blindness regain significant vision improvements. This trial was conducted by doctors at the Moorfields Eye Hospital in London, the trial focused on children with an aggressive form of Leber Congenital Amaurosis (LCA), a rare genetic disorder that causes rapid vision loss from birth. Before the therapy, the affected children were legally blind, only able to distinguish between light and dark. However, after receiving the treatment, all four toddlers demonstrated remarkable progress—some even beginning to draw and write.The findings were published in the Lancet medical journal titled Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study. The gene therapy of another form of genetic blindness is also available on the UK National Health Service, since 2020, and this work just builds upon that. The findings thus provide hope for future treatments of genetic blindness. How Does This Gene Therapy Work?The therapy is innovative in its own way and it involves injecting healthy copies of the AIPL 1 gene into the retina at the back of the eye. The scientists at the University College London developed this method and the procedure was performed by specialists at Great Ormond Street Hospital. The healthy gene copies are delivered using a harmless virus that carries them into the retinal cells, replacing the defective gene. This process also helps the cells function properly, slowing down degeneration and improving vision. Unlike the traditional clinical trials, in this trial, the therapy was offered under a special compassionate-use license. This is because no other treatment options had existed for these children and thus each child received the therapy in only one eye to minimize any potential risk. Case Study of Jace From ConnecticutJace, who is a toddler from Connecticut, was among the four children who received the treatment, reports the BBC. His parents noticed early signs of visual impairments when he failed to make eye contact or smile when he was as young as just 8 weeks old. After months of medical consultations, he was diagnosed with the ultra-rare genetic condition. When his parents got to know about the London trial, they thought of taking a chance. Jace was then two years old, and underwent the procedure. His mother said that the surgery was quick and minimally invasive, leaving only four tiny scars on his eyes. Within a month, the family was able to notice changes in Jace's responses. He now squinted in bright sunlight, which he has not done before, his vision also not improved. Before surgery, he could not track objects near his face, now he can pick up small toys from the floor and navigate his surroundings. Jace may require further treatments in future, but this, so far, has been life-changing, for both him and his parents. A Future For Children Prof. James Bainbridge, a retinal surgeon at Moorfields Eye Hospital, emphasized how crucial early intervention is for children with severe sight impairment. “Treatment in infancy with this new genetic medicine can transform the lives of those most severely affected,” he stated.The trial results are compelling. The vision in the treated eyes improved, while the untreated eyes continued to deteriorate as expected. Consultant eye surgeon Prof. Michel Michaelides described the outcomes as "hugely impressive," showcasing the power of gene therapy in changing lives.

how a 2-year-old connecticut toddler got his vision back with this breakthrough gene therapy

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