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Participant’s Diagnosis Halts Gene Therapy Clinical Trial
Participant’s Diagnosis Halts Gene Therapy Clinical Trial
The FDA pauses the research program on a lentivirus-based treatment for a rare neurological condition after a patient developed a bone marrow disorder that could presage leukemia.
The FDA pauses the research program on a lentivirus-based treatment for a rare neurological condition after a patient developed a bone marrow disorder that could presage leukemia.
clinical data, genetics & genomics
Gene Therapy in One Eye Improves Vision in Both Eyes
Abby Olena, PhD | Dec 11, 2020 | 4 min read
It’s not clear why the patients with Leber hereditary optic neuropathy, a mitochondrial disorder that causes blindness, also experienced the modest benefits in their untreated eye.
A New Way to Establish Cause and Effect in Epidemiology?
Rachael Moeller Gorman | Jan 1, 2020 | 7 min read
A technique called Mendelian randomization is overturning the conclusions of observational studies in public health. But researchers question whether the method can overcome its fundamental limitations.
The Open Data Explosion
Viviane Callier | Jan 1, 2019 | 8 min read
Scientists are working to maximize the benefits and minimize the costs of sharing.