Dr Dishant Sharma

Dishant Sharma

Post doctoral Associate

Translational and Clinical Research Institute.
Newcastle University
LocationUnited Kingdom

uk.linkedin.com/in/dishantsharma
ORCiD

Research Interests

Joubert syndrome, Nephronophthisis, Kidney diseases, Anti sense oligonucleotide, Gene Therapy

He is working on a project, “Use of antisense oligonucleotides as personalised therapies for kidney disease”. This is related to Renal ciliopathies: disease mechanisms and therapeutics. Nephronophthisis (NPHP), an autosomal recessive primary ciliopathy, is the most common cause of end-stage renal disease in children and young adults. Our research has revealed a critical role for hedgehog signalling in NPHP such that chemical agonists of smoothened can ameliorate disease phenotypes [Hynes et al.2014. PNAS 111:9893-8; Srivastava et al. 2017. Hum Mol Genet 26:4657-67]. Subsequently, we have demonstrated the therapeutic potential of exon-skipping antisense oligonucleotides (ASO) in patient cells and mouse models in vivo [Ramsbottom et al. 2018. PNAS 115: 12489-94; Molinari et al. 2019. Sci Rep 9:10828]. We now wish to exploit a novel murine model of NPHP and use ASOs to determine the exact therapeutic benefit and tissue delivery and assess off-target effects as a prelude to first-in-man studies.