grant

Customized iPS Cell Therapy for Recessive Monogenic Retinal Degenerative Disease [ 2009 - 2016 ]

Also known as: Stem Cell Gene Therapy for Inherited Diseases

Research Grant

[Cite as https://purl.org/au-research/grants/nhmrc/546489]

Researchers: Dr Sara Howden (Principal investigator)

Brief description The focus of this study is to develop a personalised treatment for certain types of retinal degenerative disease (RDD). Stem cells will be generated from the skin cells obtained from an individual with RDD. Gene therapy will then be applied to correct the underlying disease-causing mutation in the patient cells. The repaired cells will be used to generate retinal cells, which will subsequently be tested in naturally occurring RDD rodent models to determine if they have any beneficial effects.

Funding Amount $AUD 350,714.05

Funding Scheme Early Career Fellowships

Notes CJ Martin Biomedical ECF

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