PARM is a deep-learning model trained on data from massively parallel reporter assays to help predict promoter activity in different human cell types, design synthetic promoters and identify key features…
In a multicentre trial of AAV1-hOTOF gene therapy involving 42 participants aged 0.8–32.3 years with autosomal recessive deafness 9, treatment demonstrated safety, tolerability, and improved hearing and speech perception, with…