Catching up but still miles behind-a patient registry for otoferlin.
作者信息
Vona Barbara, Wollnik Bernd, Strenzke Nicola, Moser Tobias
机构信息
Institute of Human Genetics, University Medical Center Göttingen, Göttingen, Germany.
Institute for Auditory Neuroscience and InnerEarLab, University Medical Center Göttingen, Göttingen, Germany.
出版信息
Exp Mol Med. 2024 Jun;56(6):1472-1473. doi: 10.1038/s12276-024-01247-6. Epub 2024 Jun 3.
Abstract
摘要
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AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial.
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