With gene drives, scientists are trying to supercharge evolution to eradicate malaria and save endangered species from extinction. But is this DARPA-funded tech safe enough to test in the wild? One of its creators isn’t so sure.
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Category: genetics – Page 475
It’s all a question of money. We should come up with enough money for funding this so that we can clone a perfect genetic match of every organ in the body by 2025. It will solve the organ shortage issue, and nip the illegal black market organ industry in the bud.
The FDA said it is looking into “regenerative medicine.”
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On Wednesday—for only the second time—the Food and Drug Administration approved a cutting-edge therapy that genetically modifies a patient’s blood cells in order to attack cancer. This time the therapy, known as CAR T-cell therapy, is designed to treat aggressive non-Hodgkin lymphoma.
In August, the FDA approved the first CAR T-cell therapy, for a drug called Kymriah designed for children and young adults whose leukemia doesn’t respond to standard treatments. The FDA’s approval of Yescarta, manufactured by Kite Pharma, comes just a few months after its first approval—an indication of just how quickly the field of immunotherapy is moving. Several other companies also have CAR-T therapies in the works.
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[p]Patients with junctional epidermolysis bullosa (JEB) carry mutations in genes that encode components of the basement membrane, which ensures the integrity between the epidermis and the dermis, such as laminin-332. These mutations cause blistering of the skin and chronic wounds. Following initial treatment of an adult patient with a limited affected region, Michele De Luca and colleagues reconstruct the full epidermis of a 7-year-old patient with autologous transgenic cells transduced with a virus vector carrying the non-mutated form of laminin-322.
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For the first time ever, scientists have attempted to cure a person’s disease by editing a gene inside the body.
Scientists used an IV to inject a patient with billions of copies of a corrective gene and a genetic tool to cut his DNA in a specific spot. “We cut your DNA, open it up, insert a gene, stitch it back up.”
Scientists have edited people’s genes in the past, but that work involved altering cells inside a lab and then returning them to the body, whereas the latest experiment was performed inside a person’s body.
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He is the first patient to receive an experimental gene therapy as part of a clinical trial. Earlier this week, Sangamo Therapeutics injected Madeux with viruses containing a package of gene-editing material, according to the AP. The hope is that these viruses will enter Madeux’s cells, specifically liver cells, inject the missing gene at the right place in his DNA. Only about 1% of the liver’s cells need to be fixed, and give his liver the ability to produce the enzyme he has been missing all his life.
Brian Madeux’s life hasn’t been easy. So far, he’s had 26 operations to fix problems in everything from hernias to eyes. He has a rare disease called Hunter syndrome, which is caused by the lack of a gene that’s used to produce an enzyme that breaks down certain carbohydrates. As a result, the carbohydrates build up in his body’s cells causing all sorts of problems.
There is no cure. One way to deal with some of the symptoms is to receive regular doses of the missing enzyme, which may cost him in the US between $100,000 and $400,000 per year. Even then, the enzyme won’t reverse the damage made already and it won’t stop further deterioration that happens in the brain.
But Madeux’s life might be about to change. He is the first patient to receive an experimental gene therapy as part of a clinical trial. Earlier this week, Sangamo Therapeutics injected Madeux with viruses containing a package of gene-editing material, according to the AP. The hope is that these viruses will enter Madeux’s cells, specifically liver cells, inject the missing gene at the right place in his DNA. Only about 1% of the liver’s cells need to be fixed, and give his liver the ability to produce the enzyme he has been missing all his life.
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The success of a novel gene therapy for blindness caused by a genetic mutation paves the way for gene therapies which treat other forms of blindness as well as genetic therapies which treat other diseases.
After being treated with a novel gene therapy for blindness, patients who had lost their sight due to a genetic retinal disease could see well enough to navigate a maze, according to research presented today at the 2017 Annual Meeting of the American Academy of Ophthalmology (AAO 2017).
Novel Gene Therapy for Blindness
Patients in the study had a form of blindness called Leber congenital amaurosis (LCA), an inherited degenerative retinal disease characterized by severe loss of vision at birth. This novel gene therapy for blindness is currently under review by the FDA for potential approval. There are no other FDA-approved treatments available for inherited retinal diseases.
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“Scientists are hopeful that the approach could offer new ways to understand – and even one day tackle – certain human genetic diseases by correcting mutations in a patient’s body.”
Fresh DNA base editing breakthrough brings hope of potential treatment for huge number of diseases that arise as a result of a single genetic ‘misspelling’.
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Gene editing and beyond
Posted in bioengineering, biotech/medical, genetics
The CRISPR-Cas9 system has revolutionised gene-editing, but cutting DNA isn’t all it can do. From turning gene expression on and off to fluorescently tagging particular sequences, this animation explores some of the exciting possibilities of CRISPR.
Download a poster on ‘The expanding CRISPR toolbox’ here: https://www.nature.com/posters/crisprtoolbox
Produced with support from Dharmacon: https://www.dharmacon.com
Nature has full responsibility for all editorial content, including Nature Video content. This content is editorially independent of sponsors.
31st October 2017
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