{"id":1992,"date":"2025-06-01T01:49:03","date_gmt":"2025-06-01T01:49:03","guid":{"rendered":"https:\/\/jonathandespres.com\/cryonicsrevival\/?p=1992"},"modified":"2025-06-01T01:49:03","modified_gmt":"2025-06-01T01:49:03","slug":"no-more-death-sentence-100-survival-rate-achieved-in-gene-therapy-trial","status":"publish","type":"post","link":"https:\/\/jonathandespres.com\/cryonicsrevival\/2025\/06\/01\/no-more-death-sentence-100-survival-rate-achieved-in-gene-therapy-trial\/","title":{"rendered":"No more ‘death sentence’: 100% survival rate achieved in gene therapy trial"},"content":{"rendered":"

A gene therapy offered new hope for children suffering from a rare and potentially fatal immune disorder, according to a recent study published in the New England Journal of Medicine. The study evaluated the outcomes after two years of treatment in nine patients aged between 5 months and 9 years, all of whom survived to the two-year follow-up mark and are living with no disease symptoms, indicating a “sustained clinical benefit in all patients.”<\/p>\n

Leukocyte Adhesion Deficiency Type I (LAD-I) is an extremely rare genetic disease, occurring in one case per million, caused by a mutation in the gene ITGB2. This mutation leads to the production of a defective protein called CD18, preventing leukocytes from traveling to infection sites and effectively fighting infections. Without treatment, survival beyond childhood is rare for individuals with severe LAD-I, and the most severe cases are often fatal before the age of two.<\/p>\n

The only available treatment for patients with severe LAD-I was a bone marrow transplant from a compatible stem cell donor. However, finding a suitable donor is challenging, and transplants carry risks, including potentially deadly side effects such as graft-versus-host disease.<\/p>\n","protected":false},"excerpt":{"rendered":"

A gene therapy offered new hope for children suffering from a rare and potentially fatal immune disorder, according to a recent study published in the New England Journal of Medicine. The study evaluated the outcomes after two years of treatment in nine patients aged between 5 months and 9 years, all of whom survived to […]<\/p>\n","protected":false},"author":1,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":[],"categories":[58],"tags":[],"_links":{"self":[{"href":"https:\/\/jonathandespres.com\/cryonicsrevival\/wp-json\/wp\/v2\/posts\/1992"}],"collection":[{"href":"https:\/\/jonathandespres.com\/cryonicsrevival\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/jonathandespres.com\/cryonicsrevival\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/jonathandespres.com\/cryonicsrevival\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/jonathandespres.com\/cryonicsrevival\/wp-json\/wp\/v2\/comments?post=1992"}],"version-history":[{"count":1,"href":"https:\/\/jonathandespres.com\/cryonicsrevival\/wp-json\/wp\/v2\/posts\/1992\/revisions"}],"predecessor-version":[{"id":1993,"href":"https:\/\/jonathandespres.com\/cryonicsrevival\/wp-json\/wp\/v2\/posts\/1992\/revisions\/1993"}],"wp:attachment":[{"href":"https:\/\/jonathandespres.com\/cryonicsrevival\/wp-json\/wp\/v2\/media?parent=1992"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/jonathandespres.com\/cryonicsrevival\/wp-json\/wp\/v2\/categories?post=1992"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/jonathandespres.com\/cryonicsrevival\/wp-json\/wp\/v2\/tags?post=1992"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}