1/13/2024 0 Comments Ag source inc![]() People with one copy of the mutation usually don't have any symptoms of the disease, while people with two copies – one from each parent – can develop severe complications. The disorder is most common in Africa, where the sickle cell gene helped protect against malaria. Scientist Linus Pauling described sickle cell as the first molecular disease in 1949. The finding sparked a rush of companies seeking to leverage that insight to treat various diseases. In 2012, researchers Jennifer Doudna and Emmanuelle Charpentier published their seminal paper on a system for editing genes called CRISPR-Cas9. Food and Drug Administration would mark a scientific milestone about a decade after the discovery of CRISPR and a breakthrough for patients desperate for a better option. It works differently than exa-cel but is administered similarly and is also intended to eliminate pain crises. It's expected to be approved later this month.Īpproval of exa-cel by the U.S. are also reviewing another gene therapy from Bluebird Bio called lovo-cel. approved it under the brand name Casgevy last month. regulators are expected to approve exa-cel for use in sickle cell patients by the end of this week. ![]() The treatment uses Nobel Prize-winning technology called CRISPR to edit a person's DNA and alleviate the symptoms of sickle cell disease. ![]() Tsogbe, now 37, received one of those options, known as exa-cel and co-developed by Vertex Pharmaceuticals and CRISPR Therapeutics, via a clinical trial in 2021. New genetic treatments aim to offer relief while eliminating the need to track down donors. Few treatments are available, and the only cure is a bone marrow transplant where a patient receives healthy blood stem cells from a donor. The disease affects about 100,000 people in the U.S., many of whom are Black. Personal Loans for 670 Credit Score or Lower Personal Loans for 580 Credit Score or Lower Best Debt Consolidation Loans for Bad Credit
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