The New England journal of medicine
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Multicenter Study
Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy.
Cerebral adrenoleukodystrophy is a severe form of X-linked adrenoleukodystrophy characterized by white-matter disease, loss of neurologic function, and early death. Elivaldogene autotemcel (eli-cel) gene therapy, which consists of autologous CD34+ cells transduced with Lenti-D lentiviral vector containing ABCD1 complementary DNA, is being tested in persons with cerebral adrenoleukodystrophy. ⋯ At a median follow-up of 6 years after lentiviral gene therapy, most patients with early cerebral adrenoleukodystrophy and MRI abnormalities had no major functional disabilities. However, insertional oncogenesis is an ongoing risk associated with the integration of viral vectors. (Funded by Bluebird Bio; ALD-102 and LTF-304 ClinicalTrials.gov numbers NCT01896102 and NCT02698579, respectively.).
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Letter Randomized Controlled Trial Multicenter Study
Pembrolizumab in HER2-Positive Gastric Cancer.
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Letter Randomized Controlled Trial Comparative Study
Radical Prostatectomy or Watchful Waiting in Early Prostate Cancer.