Neonatal gene therapy has been shown to prevent inner ear dysfunction in mouse models of Usher syndrome type I (USH1), the most common genetic cause of combined deafness-blindness and vestibular dysfunction. However, hearing onset occurs after birth in mice and in utero in humans, making it questionable how to transpose murine gene therapy outcomes to clinical settings. Here, we sought to extend the therapeutic time window in a mouse model for USH1G to periods corresponding to human neonatal stages, more suitable for intervention in patients. Mice with deletion of Ush1g ( Ush1g ^–/– ) were subjected to gene therapy after the hearing onset. The rescue of inner ear hair cell structure was evaluated by confocal imaging and electron microscopy. Hearing and vestibular function were assessed by recordings of the auditory brain stem response and vestibulo-ocular reflex and by locomotor tests. Up to P21, gene therapy significantly restored both the hearing and balance deficits in Ush1g ^–/– mice. However, beyond this age and up to P30, vestibular function was restored but not hearing. Our data show that effective gene therapy is possible in Ush1g ^–/– mice well beyond neonatal stages, implying that the therapeutic window for USH1G may be wide enough to be transposable to newborn humans.

(1) Background: Sequels of facial palsy lead to major psychosocial repercussions, disrupting patients’ quality of life (QoL). Botulinum toxin (BoNT) injections can permit us to treat long-standing facial palsy, improving facial symmetry and functional signs including synkinesis and contractures. (2) Methods: The main aim of this study was to assess the evolution of the QoL for patients with long-standing facial palsy before, at 1 month, and at 4 months after BoNT injections by using three questionnaires (HFS-30, FaCE, and HAD). The other goals were to find clinical factors associated with the improvement in the QoL and to assess the HFS-30 questionnaire for patients with unilateral facial palsy (3) Results: Eighty-eight patients were included in this study. There was a statistically significant improvement in QoL at 1 month after injections, assessed using the three questionnaires. This improvement was sustained at 4 months after the injections, with a statistically significant difference for the HFS-30 and FaCE questionnaires. (4) Conclusions: This study showed that the BoNT injections lead to a significant increase in the QoL of patients with unilateral facial palsy. This improvement is sustained 4 months after the injections.

Objective: The role of antibiotics in ear surgery is still controversial. The aim of this study was to assess their need in cholesteatoma surgery when performing obliteration with S53P4 bioactive glass, a biocompatible material with antibacterial properties.Methods: This retrospective cohort study was conducted in a tertiary referral center between January 2017 and May 2019. Sixty‐nine consecutive patients, who underwent surgery for cholesteatoma removal and/or rehabilitation of canal‐wall‐down mastoidectomy with mastoid and epitympanic obliteration using S53P4 granules were included. Before 2019, antibiotics were routinely used (group “w/AB”). Patients received intravenous antibiotics during surgery, oral treatment was continued for 7 days and topical antibiotics for 1 month. After 2019, no antibiotics were administered (group “w/oAB”). The primary outcome was the occurrence of early surgical site infection. Secondary outcomes were late infection, anatomic and functional results at 3 and 12 months.Results: Twenty‐three patients were included in group “w/oAB” and 46 in group “w/AB”, with no significant differences in demographics, medical history or follow‐up. Five ears (22%) in group “w/oAB” developed an early infection compared with 2 (4%) in group “w/AB” (p = .03). The relative risk was 6.11, 95CI%[1.09;31.96]. Infections were successfully treated with antibiotics, and no patient underwent surgical removal of the granules. No late infections or complications were observed. There was no difference in graft failure or air‐bone gap closure at 1 year.Conclusion: Peri‐/post‐operative antibiotics prevent early infection in obliteration surgery with S53P4 granules. Infections can be treated medically without complications or require removal of the implanted material.Level of evidence: 4.