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VIC Stem Cell Network Monthly Seminar Series - March 2015

04:00 PM - Tuesday 03 March, 2015

Join us to hear Dr Sara Howden from Murdoch Childrens Research Institute discuss correcting genetic mutations in induced pluripotent stem cell lines and what this means for patients.

Generation of gene corrected iPS lines from patient-specific fibroblasts

Guest Speaker: 
Dr Sara Howden, Murdoch Children’s Research Institute

4:00PM on Tuesday 3 March 2015 (Refreshments to follow)

Level 5 Seminar Room, Melbourne Brain Centre, Parkville Campus 

BIO:  Sara Howden is a postdoctoral fellow at the Murdoch Childrens Research Institute. She received a BSc from the University of Melbourne and joined the Cell and Gene Therapy Lab at the MCRI to undertake an honors project. She stayed on to complete her PhD in the same lab before moving to Madison, Wisconsin for a postdoctoral position in the laboratory of Dr James Thomson. She has recently returned to complete the Australian component of her Overseas Biomedical research fellowship where she will continue to develop and apply the reprogramming and gene targeting methodologies she acquired during her time in the Thomson lab.

ABSTRACT: Autologous cell therapies based on gene-corrected patient-derived induced pluripotent stem cells hold great promise for the treatment of many inherited and acquired diseases. I will discuss the generation of the first gene-corrected iPS cell lines, isolated from a patient with Gyrate atrophy. I will then describe a recently developed protocol for the simultaneous generation of gene-targeted/corrected iPS lines following a single electroporation of patient-specific fibroblasts using episomal-based reprogramming vectors and the Cas9/CRISPR gene targeting system. Importantly, this procedure allows the generation of gene-targeted iPS lines with only a single clonal event in as little as three weeks and without the need for drug selection, thereby facilitating “seamless” single base pair changes. We anticipate that simultaneous reprogramming and gene-correction of patient-derived cells should facilitate transplantation medicine by enabling cells to be available to patients in a more timely manner whilst minimising risks associated with the culturing of cells in artificial culture conditions.