{"id":1060,"date":"2023-06-15T19:28:08","date_gmt":"2023-06-15T19:28:08","guid":{"rendered":"https:\/\/hemdifferently-dev-001.azurewebsites.net\/en-gb\/?page_id=1060"},"modified":"2024-02-20T10:11:41","modified_gmt":"2024-02-20T10:11:41","slug":"what-is-gene-therapy","status":"publish","type":"page","link":"https:\/\/haemophilia.biomarin.com\/en-gb\/what-is-gene-therapy\/","title":{"rendered":"What is gene therapy"},"content":{"rendered":"
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What is gene therapy?\n<\/h1>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n\n
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A genetic condition could potentially have a genetic solution, right?<\/h2>\n

This logical thinking gave rise to the first research in gene<\/span><\/strong> therapy more than 50 years ago. Now being evaluated in multiple clinical trials for a range of genetic conditions to determine the possible benefits and risks, gene therapy research has the potential to bring an entirely new option to people with specific genetic conditions and those who support and care for them.<\/p>\n <\/div>\n \t\t<\/div>\n\t<\/div>\n<\/div>\n\n

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GENE THERAPY SEEKS TO ADDRESS GENETIC MUTATIONS<\/h2>\n

Simply put, gene therapy is being researched in clinical studies as a novel method that attempts to use genes to treat or prevent disease.<\/p>\n <\/div>\n \n

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METHODS OF GENE THERAPY BEING EXPLORED<\/h2>\n

A few different approaches to gene therapy are being explored. Gene therapy may involve attempting to repair or replace a mutated gene, disabling a mutated gene that is causing trouble or introducing a functional copy of the gene into the body to help the body produce the affected protein<\/span>.<\/strong><\/p>\n