Efficient kidney gene transfer and proximal tubule transduction using self-complementary AAV.cc47 vectors.

ABSTRACT: Gene delivery to critical cell types within the kidney can enable preclinical evaluation of gene therapies for kidney disease. The novel adeno-associated virus AAV.cc47 was discovered after sequential evolution in mice, pigs, and macaques and improved transduction in multiple tissues but without in-depth exploration of the kidney. We observed robust kidney transduction by AAV.cc47 vectors in mice in vivo and in human kidney organoids compared to AAV9, mostly within the proximal tubule (PT) epithelium. We then developed a quantitative analysis method of transgene expression utilizing automated classification of nephron cell types coupled with cellular expression. Despite exhibiting similar biodistribution to AAV9 in renal and extrarenal tissues, AAV.cc47 consistently transduced the k

SUBMITTER: Peek JL 

PROVIDER: S-EPMC12554119 | biostudies-literature | 2025 Dec

REPOSITORIES: biostudies-literature