Advances on gene therapy for inherited kidney diseases in children / 国际儿科学杂志

Gene therapy has enjoyed a renaissance with recent successes in the treatment of many diseases.The further studies had identified the defected gene of inherited kidney diseases,just like Alport syndrome,polycystic kidney and Fabry's disease,and make it possible for gene therapy.However,the clinical application still faces enormous challenges such as safety,effectiveness and ethical issues.In this review,we summarized the development and the treatment of gene therapy for genetic kidney diseases.Finally,we discussed the future development prospects of gene editing therapy.