New assay leads to step toward gene therapy for deaf patients – Science Daily
Science Daily |
New assay leads to step toward gene therapy for deaf patients
Science Daily "We were trying to shorten the gene, to find a truncated form that can be used for gene therapy. There is a size limit in terms of what you can package into the gene delivery vehicle, and otoferlin is too large. That's the holy grail, trying to find a … |
