A gene therapy researcher will describe “Adeno-Associated Virus (AAV) Genome Rescue of NPHS2 in Mice” in the Michelle P. Winn, MD, Endowed Lectureship on Thursday, November 3.
Moin Saleem, MBBS, MBChB, MD, PhD, heads Bristol Renal, a glomerular research group of approximately 45 researchers that encompasses many areas, including cell biology, transgenic models, and population cohorts and genetics. He is also a professor of pediatric renal medicine at the University of Bristol in the United Kingdom.
Dr. Saleem has published extensively about adeno-associated virus gene therapy to prevent progression of kidney diseases in genetic human and mouse models of nephrotic syndrome. Gene therapy targeting the kidney has proven challenging, although AAV has been used successfully for gene therapy targeting other organs, with particular success in targeting monogenic diseases.
The most common cause of genetic nephrotic syndrome in children is a mutation in the NPHS2 gene encoding podocin. Among other approaches, Dr. Saleem and colleagues have tested AAV-mediated gene therapy in a conditional podocin knockout mouse model and in human podocytes with the most common podocin mutation.
Dr. Saleem was the originator of the UK Registry of Rare Kidney Diseases and currently leads the UK National Study of Nephrotic Syndrome (NephroS) and a major industry-academic collaboration, termed NURTuRE (National Unified Renal Translational Research Enterprise). NURTuRE is a national cohort and biobank of patients with chronic kidney disease and nephrotic syndrome who are being studied for stratification and re-definition of diseases, according to molecular phenotypes. This effort is being extended to international cohorts in developing countries. Dr. Saleem's work in genetic stratification of nephrotic syndromes has led to gene discoveries and the establishment of national clinical gene panels that have been integrated into new international testing guidelines.
His laboratory undertakes research from basic cell and animal biology and physiology to studies of international patient cohorts with rare diseases, including genome sequencing and systems biology approaches. The lab has developed the world's gold standard glomerular cell lines. He commenced his gene therapy program in 2014 and is a founder of the field of podocyte biology with a focus on targeting the podocyte to radically change the treatment of kidney diseases. He is the co-founder and chief scientific officer of Purespring Therapeutics, the world's first renal gene therapy biotech company featuring research programs for diseases with no currently effective therapies. Dr. Saleem received a PhD in transplantation immunology from the Institute of Child Health in London. He trained in pediatric nephrology at the Great Ormond Street Hospital in London.