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Split otoferlins reunited

View ORCID ProfileJeffrey R Holt, View ORCID ProfileGwenaelle SG Geleoc
DOI 10.15252/emmm.201809995 | Published online 20.12.2018
EMBO Molecular Medicine (2019) 11, e9995
Jeffrey R Holt
Department of Otolaryngology, Boston Children's Hospital and Harvard Medical School, Boston, MA, USA
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Gwenaelle SG Geleoc
Department of Otolaryngology, Boston Children's Hospital and Harvard Medical School, Boston, MA, USA
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Author Affiliations

  1. Jeffrey R Holt1 and
  2. Gwenaelle SG Geleoc (gwenaelle.geleoc{at}childrens.harvard.edu)1
  1. 1Department of Otolaryngology, Boston Children's Hospital and Harvard Medical School, Boston, MA, USA
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    Figure 1. Dual adeno‐associated viral vectors (AAVs) for gene replacement in the inner ear

    Split AAVs, each containing a fragment of the large otoferlin coding sequence are injected into the inner ears of otoferlin knockout mice. Upon co‐transduction in single cells, the split sequences reassemble full‐length otoferlin, which in sensory hair cells leads to recovery of protein expression, partial recovery of synaptic function and auditory brainstem responses (ABR). Bottom panels illustrate synaptic and ABR recovery after dual‐AAV treatment (orange) relative to Otof−/− mice (blue) and wild‐type mice (black).

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Volume 11, Issue 1
01 January 2019
EMBO Molecular Medicine: 11 (1)
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