Principal Investigator Feng Zhang
The mammalian brain expresses around 20,000 genes, and a method to regulate their activity with precise specificity would be of great value as a research tool. It could also lead to new therapies for brain disorders, many of which involve abnormal patterns of gene expression. As a junior fellow at Harvard, Zhang developed a new method for constructing customized DNA-binding proteins. These proteins, known as TAL-effectors, can be produced quickly and cheaply using the new method, and can be targeted to any desired DNA sequence. Zhang plans to use TAL effectors to manipulate brain gene expression, using several different approaches. These proteins can be used to introduce changes into the genome of a cell, for example introducing defined mutations for experimental work or repairing genetic mutations as a potential therapy for certain genetic diseases. TAL effectors can also be designed to influence gene expression in other ways, for example activating or blocking the transcription of the targeted genes.