Mutation profile of BBS genes in patients with Bardet-Biedl syndrome: an Italian study.
Elena ManaraStefano PaolacciFabiana D'EspositoAndi AbeshiLucia ZiccardiBenedetto FalsiniLeonardo ColomboGiancarlo IarossiAlba PilottaLoredana BocconeGiulia GuerriMarica MonicaBalzarini MartaPaolo Enrico MalteseLuca BuzzonettiLuca RossettiMatteo BertelliPublished in: Italian journal of pediatrics (2019)
NGS is a powerful tool that can help understanding BBS patients' phenotype through the identification of mutations that could explain differences in phenotype severity and could provide insights for the development of targeted therapy. Furthermore, our results support the existence of additional BBS loci yet to be identified.