More recently, Avexis (acquired by Novartis) developed an AAV gene therapy called Zolgensma, which has enabled children born with spinal muscular atrophy to survive what had been a lethal disease. The first gene therapy to be approved in the United States was an AAV therapy developed by Spark Therapeutics (later acquired by Roche) that delivers a gene into the eye to treat a mutation causing impaired vision. An AAV is a viral vector that delivers episomal DNA (DNA maintained separately from the rest of our genome) to cells. It has been approved by the European Medical Association to treat a mutation that causes an immune disorder known as bubble boy disease.įor in vivo applications of gene therapy, adeno-associated viruses (AAVs) have emerged as the primary means of delivering DNA into cells. Building upon work from Luigi Naldini and others at the San Rafaele Telethon Institute for Gene Therapy, in Milan, GlaxoSmithKline used retroviral vectors to develop Strimvelis, the world’s first ex vivo curative gene therapy (later spun out into Orchard Therapeutics). They have been the workhorses for ex vivo gene therapy, including the use of engineered immune cells to treat cancer, known as CAR-T cell therapies. Retroviral vectors, like the lentiviral vectors derived from HIV, integrate DNA semi-randomly into the genome. Gene therapy can be divided into ex vivo and in vivo approaches. Such mutations typically prevent the gene from functioning as it should. Gene therapy, the more advanced of the two pillars of contemporary genetic medicine, uses engineered viruses to add DNA to cells in order to treat diseases caused by recessive mutations. It can specifically and efficiently direct diverse alterations to the genome, in any type of cell, creating new possibilities for patients with genetic diseases. Gene Writing writes curative therapeutic messages into the genome. The company is pioneering a new category of genome engineering technology called Gene Writing and establishing a new field of genetic medicine. In 2017, Flagship partner Geoffrey von Maltzahn, principal Jacob Rubens, associate Rob Citorik, and others began an exploration inside Flagship Labs to address these limitations by asking, What if nature evolved a better way to alter genomes than cutting DNA? The result is Tessera Therapeutics. But gene therapy and gene editing have significant limitations in the modifications they can make to the genome, the cells they can affect, and how they are manufactured and delivered to cells, leaving today’s genetic medicine powerless against thousands of diseases. Both technologies are enormously promising, and in some cases they have already realized their promise and cured diseases. Today, this area, sometimes called genetic medicine, stands upon two technological pillars: gene therapy and gene editing. This has opened the possibility of creating therapies that not only treat previously untreatable disease but might also cure patients for life with a single treatment. Recently, scientists and doctors have gained the power to add and subtract tiles from our genomic mosaic. Every year the number of human genomes that have been sequenced increases, as does our understanding of how genes provide the instructions to make us healthy or unhealthy human beings. About 60,000 other genetic sites are significantly associated with traits and diseases such as body mass, cardiovascular disease, neurological conditions, metabolic diseases, immune responses, and many cancers. These include the cystic fibrosis transmembrane receptor, which when mutated causes cystic fibrosis, which can severely damage the lungs and other organs to the point of being life-threatening. Development, homeostasis, and reproduction aging, disease, and death-all are driven by the activity of genes.ĭoctors know of about 4,000 genes that directly cause diseases. Specific sequences of base pairs encode genes, which serve every function in our bodies. These represent the code of life and give rise to the complex phenomena that define health and disease. It is composed of base pairs, the nucleobases A, C, T, and G-the building blocks of DNA. Our genome is a mosaic that has evolved over millennia.
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