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functionalgenomics.pl is a fearless research initiative advancing human genome engineering technology to drive the next generation gene therapy
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The aim of functionalgenomics.pl is to deliver next-generation gene therapies offering lasting solutions for neurodevelopmental and neurodegenerative conditions throughout Somatic Cell Genome Editing (SCGE) program and Targeted Genome Editor Delivery Challenge (TARGETED) including:

 

Gene repair technology development for somatic cell (post mitotic neurons, astrocytes and microglia).

Cell type specific delivery of therapeutic gene editing components using engineered next generation non viral particles.

Application of patient derived 3D human brain model systems  driven by deep learning to support treament decisions in the context of personalized medicine by:

(1) high throughput phenotypic screening;

(2) drug screening and drug reporposing as suplementary strategies.

 

 

 

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