grant

Correction of Friedreich ataxia induced pluripotent stem cells by non-viral gene therapy [ 2014 - 2016 ]

Also known as: Non-viral gene therapy for Friedreich ataxia

Research Grant

[Cite as https://purl.org/au-research/grants/nhmrc/1055785]

Researchers: Miss Tejal Kulkarni (Principal investigator)

Brief description Friedreich ataxia (FRDA) is an inherited progressive disorder of the nervous system and heart. Stem cell therapy has the potential to repair or replace damaged tissues and restore organ function in FRDA patients. The defect inherent in stem cells obtained from FRDA patients will be corrected by a gene therapy approach that will restore normal FRDA gene expression and addresses major safety concerns for the clinical use of corrected stem cells in transplantation medicine.

Funding Amount $AUD 63,270.50

Funding Scheme Postgraduate Scholarships

Notes Dora Lush Biomedical Postgraduate Scholarship

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Identifiers
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ACN 633 798 857