Clinical research
Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.
この試験で検証していること
The trial that started clinical gene editing. Years before CRISPR was a tool, patients with HIV had their own immune cells taken out, had a gene edited with zinc finger nucleases, and had them returned. It showed that a permanent, targeted edit could be made in a patient's cells and given back safely.
A Phase 1 study of autologous CD4 T cells with CCR5 disrupted by zinc finger nucleases in people with HIV. Published in the New England Journal of Medicine, it reported that infusion was safe and that modified cells persisted, establishing the clinical feasibility of therapeutic genome editing.
Sources
- New England Journal of Medicine · 2014
Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV ↗
試験結果が意味すること・意味しないこと
初期段階の結果は少人数の参加者と短い追跡期間から得られたものです。参加者が増え時間が経つにつれて変わる可能性があり、募集中・実施中・完了済みの試験であっても、治療効果が確立されたわけではありません。登録基準は厳格で、適格性の判断は試験実施施設が行うものであり、ウェブサイトが行うものではありません。