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Intraventricular Transplantation of Engineered Neuronal Precursors for In Vivo Neuroarchitecture Studies

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Published on: May 11, 2019

Dissecting neural function using targeted genome engineering technologies.

Patrick D Hsu¹, Feng Zhang

¹Department of Molecular and Cellular Biology, Harvard University, Cambridge, Massachusetts, USA; Broad Institute of MIT and Harvard, Cambridge, Massachusetts, USA; McGovern Institute for Brain Research, MIT Department of Brain and Cognitive Sciences, Massachusetts Institute of Technology, Cambridge, Massachusetts, USA.

ACS Chemical Neuroscience

|August 17, 2012

Summary

This summary is machine-generated.

Designer DNA-binding proteins like TALEs and ZFPs enable precise genome editing. These engineered nucleases create targeted DNA breaks, facilitating scarless gene modification and diverse applications in synthetic biology and neuroscience.

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Area of Science:

Molecular Biology
Genetics
Biotechnology

Background:

Designer DNA-binding proteins, including transcriptional activator-like effectors (TALEs) and zinc finger proteins (ZFPs), offer modular DNA sequence recognition.
These proteins can be engineered into tools for targeted genome perturbation.

Purpose of the Study:

To review recent advances in designer DNA-binding protein technologies.
To focus on designer nucleases for precise, efficient, and scarless gene modification.
To discuss applications in synthetic biology, disease modeling, and neural function interrogation.

Main Methods:

Engineering of TALE and ZFP nucleases to create targeted double-stranded DNA breaks.
Stimulation of cellular DNA repair pathways for genome modification.
Review of current literature on applications and future perspectives.

Main Results:

TALE and ZF nucleases enable precise, efficient, and scarless modification of the endogenous genome.
These technologies are applicable in synthetic biology and disease modeling.
Novel effector domains allow for genetic and epigenetic regulation.

Conclusions:

Customizable DNA-binding proteins, particularly designer nucleases, are powerful tools for genome engineering.
These proteins have broad applications ranging from basic research to potential therapeutic strategies.
Future directions include interrogating neural function using these versatile protein platforms.