Prenatal stem cell gene therapy for Wilson's Disease using amniotic fluid stem cells. (360G-Wellcome-098539_Z_12_Z)

£318,402

Advances in prenatal diagnosis have led to the development of fetal therapies for inherited disorders. Stem cell and gene transfer techniques have shown thegreatest potential for use in the fetus, but their clinical utility has been limited by poor efficacy and concerns about safety. A novel approach that may address these limitations is stem cell-gene therapy with autologous amniotic fluid stem cells (AFS). These cells could undergo genetic modification in vitro and subsequently injected to the same fetus without the risk of an immune reaction/rejection. The proposed programme of work will investigate thetherapeutic potential of stem cell-gene transfer with AFS in Wilson's disease.This is an autosomal recessive disorder of copper metabolism that can be diagnosed in utero, and is caused by a mutation in the gene coding for the copper transport protein ATP7B. A model of Wilson's disease in ATP7B gene "knockout" mice will be used to test the hypothesis that correction of the genetic defect in autologous AFS, followed by in prenatal administration has long-term salutary effects effect in this setting. Key goals of the fellowshipare to maximise transduction of isolated AFS with functional ATP7B gene, optimise in utero transplantation of transduced AFS, and compare the therapeutic efficacy of AFS cell-gene transfer with other modes of fetal intervention for Wilson's disease.

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Grant Details

Amount Awarded 318402
Applicant Surname Loukogeorgakis
Approval Committee Clinical Interview Committee
Award Date 2012-05-31T00:00:00+00:00
Financial Year 2011/12
Grant Programme: Title Postdoctoral Training Fellowship for Clinicians
Internal ID 098539/Z/12/Z
Lead Applicant Dr Stavros Loukogeorgakis
Partnership Value 318402
Planned Dates: End Date 2015-10-03T00:00:00+00:00
Planned Dates: Start Date 2012-10-04T00:00:00+00:00
Recipient Org: Country United Kingdom
Region Greater London
Sponsor(s) Prof Andrew Copp