Soft Magazine - Kindle Edition - September 2019 – Misty Lovelace

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Research into gene editing is advancing as well. In September, Editas Medicine shared preliminary results from the first trial testing a CRISPR gene editing treatment that does its work inside the body. Treatment appeared safe, although the efficacy results were mixed, with several patients experiencing little improvement in vision. The treatment uses CRISPR editing to restore the function of eye cells in people with another form of LCA known as type 10. And as Luxturna keeps working, other drugmakers hope to replicate its success. The eye, in particular, is the focus of many gene therapy developers, as it's easy to access and targeting it doesn't carry as many safety risks as other organs. Novartis, which sells Luxturna in Europe, AbbVie, Biogen and Johnson & Johnson are all exploring gene therapies for the eye. Four years after its approval, Luxturna continues to be sought out by patients. Joachim says she's received messages from people in Spain, South Africa and the U.K. inquiring about Luke and his progress. It’s unknown how long Luxturna’s benefits will last, but Maguire says patients treated up to 11 years ago still have stable vision in the second eye, which received a higher dose than the first. Many of them can now walk without a cane and tell colors apart. Those on the younger end of the 4- to 44-year-old age range report the most benefits, since they’d lost fewer photoreceptor cells: Kids who couldn’t play outdoors after dark, or ride a bike without help, are now able to do those things. Some of the teenagers are eligible for driver’s licenses. They are able to play varsity soccer and join the cheerleading squad. Their social lives blossomed once they could read friends’ faces.

Comander, who was in his residency while Luxturna was tested, said seeing Maguire administer the therapy affirmed his decision to go into the practice. Now, Comander has done close to a dozen surgeries; his youngest patient was 4 years old at the time of treatment and his oldest was in their 30s. While younger patients saw greater improvements, each patient's eyes functioned better in lower light following treatment.Some of those notes compared the design of the Analytical Engine to how weaving machines worked. Weaving machines follow patterns to make a complete design, and Lovelace imagined that the engine could also follow patterns—or codes—not only to calculate numbers, but to form letters, too. This is a very basic explanation of computer programming.

Berrocal believes Luxturna represents the beginning of what genetic medicine can offer to patients with many inherited diseases, not only those of the eye.Dr. Robert A. Sisk of Cincinnati Children’s Hospital Medical Center did find the right problem and he sent a sample of Misty’s blood off to Children’s Hospital of Philadelphia, where she was genetically diagnosed with LCA-RPE65. Their vision isn't perfect, however. Some recipients, Misty included, are still considered legally blind and unable to drive. How long the benefit of gene therapy treatment will last is still unclear, though a recent study co-authored by Maguire and Bennett indicated "improvements were maintained up to 3 to 4 years" after Luxturna.

For Comander, Luxturna was an inspiration, one that he said has helped fuel greater interest in gene therapy. "Many careers have been dedicated to expanding on the success of Luxturna, and it's made a huge difference in the field," he said. Working with the dogs, the scientists modified an adeno-associated virus (a small virus that’s harmless to mammals) so it carried DNA with normal RPE65. Then they injected the virus into one eye of each blind puppy. Within days, the frightened dogs who bumped into objects had turned into active, sighted animals. To the family's disappointment, and as other Luxturna patients have experienced, insurance denied the request and cited the therapy's then "newness" as a reason.There were innumerable obstacles along the way,” said Bennett, 63, curled up in a chair with her coffee. Maguire, 58, handed me a jar of honey from his beehives. He told me his wife deserved credit for frequently staying up working late while he was “snoring in bed.” To understand Misty’s level of vision then, she says to imagine wearing very dark sunglasses and looking through a PVC pipe. With no peripheral vision, she had to move her head, rather than just her eyes, to see up and down. Before Misty’s mom passed away, shortly after her eye surgery, mother and daughter shared a special time.

By 2007, it was time to try the procedure on people. The medical community was still reeling from the 1999 death of teenager Jesse Gelsinger in an unrelated gene therapy study at Penn. Starting this new research was risky. But Gelsinger had been treated for a metabolic liver disease, and the eye had certain advantages: It was easy to access, and only a small area of tissue, not the entire organ, needed to receive the gene. Plus, doctors could try the therapy in one eye before moving on to the second. (They didn’t expect an issue with patients making antibodies to the virus, since eyes are largely shielded from the body’s immune response.) The improvements were almost immediate, however. Lovelace recalls her granddaughter commenting on her wrinkles as soon as the eye patches from the procedure were removed. Misty could make out the fine hairs on the manes of horses, her favorite animal and hobby. Rainbows and stars, though, she found underwhelming. RPE65 is crucial for the visual cycle in mammals. When light hits sensitive pigments in the retina, it launches a series of reactions that make sight possible. Everyone has brief moments when this process falters—for instance, after the eye is overwhelmed by a camera flash. In healthy eyes, these moments are fleeting. But people who have two defective copies of RPE65 don’t react to light properly. Over time, the light-sensing cells—the rods and cones—die off, causing their vision to disappear. She did not take interest in this college and entered the entertainment world. She got a lot of progress and today Misty Lovelace has become a big star. Today, Misty Lovelace has millions of fans all over the world who are longing to meet Her. Proof of which you will find in their social accounts. Here are Misty Lovelace’s professions and social accounts. NameAt some point in the process, however, Luke's file crossed the desk of an anonymous person who was "so moved from Luke's story and from Luke's pictures, he volunteered to pay for Luke's surgery," Joachim said.