BIC lysosomal disorders symposium
Date: Tuesday 19 February 2019
Time: 10.00am – 5.00pm followed by drinks and a BBQ
Venue: UC Club/Ilam Homestead, 87 Ilam Road, Chrischurch
In collaboration with Lysosomal Diseases New Zealand (LDNZ), we are holding a symposium that will bring together world-leading researchers into Lysosomal storage diseases (LSDs). This Christchurch-based event follows on from the 2nd Asia-Pacific Lysosomal conference, which is being held in Auckland 14 - 16 February 2019.
LSD's are a group of more than 50 genetically inherited disorders that affect both adults and children. They are characterised by a deficiency of one or more specific lysosomal enzymes. When these degradative enzymes are missing or non-functioning, compounds that should be degraded build up in the lysosomes leading to physical and/or neurological symptoms. These disorders are classified globally as rare. In New Zealand there are about 200 families affected. There is currently no specific cure, but there are a range of treatments available. What is exciting is the broad range of significant and varied research efforts world-wide meaning there have been significant advances in our understanding of these disorders in the last decade, and advances towards new potential treatments.
This full-day symposium is a chance to hear about the research efforts from a range of New Zealand and international speakers at the cutting edge of research into these diseases.
The programme will be led by Professor Antony Fairbanks. Our international speakers include:
- Professor Konrad Sandhoff, LIMES Institute, Bonn, Germany. His major research area is the analysis and pathobiochemistry of LSD's, and the structure and function of lysosomal proteins.
- Professor Hans Aerts, Leiden University, The Netherlands. His research centres on biochemical investigations of glycosphingolipids and their metabolising enzymes, to develop biomarkers and improve diagnosis and therapies for inherited lysosomal disorders and other neurodegenerative diseases and metabolic syndromes.
- Dr Steven Gray, University of Texas Southwestern Medical Centre. His core expertise is in adeno-associated virus (AAV) gene therapy vector engineering and optimising approaches to deliver a gene to the nervous system. He is currently developing novel AAC capsids which can results in wide-spread gene transfer. He is managing a number of pre-clinical studies, as well as a human gene therapy clinical trial for Giant axonal Neuropathy (GAN).
- Associate Professor David Sleat, Rutgers University, His reaserch focuses on proteomic approached to characterise components of the lysosome. These basic insights into the functin of the lysosome and lysosomal proteins has led to the discovery of gene defects in many unsolved human diseases, including LSD's.
- Professor Jonathan Cooper, Washington University School of Medicine. He leads the Pediatric Storage Disorders lab, which focuses on the neuronal ceroid lipofuscinoses (NCLs, or Batten Disease), which is the commonest cause of childhood dementia, with the aim to understand the disease and devise therapies.
Registration is free and we welcome anyone who would like to attend. Lunch, afternoon tea and a BBQ afterwards will be provided. Register for the symposium.