This topic will be focusing on the highly specific tools that are emerging for enabling brain circuit dissection and refinement of gene-based therapeutics in the central nervous system of human and non-human primates. It will assemble experts in the field (academics, clinicians, and from industry) and provide the reader with an overview of the current tools available, their limitations, and a perspective on where these tools are headed in the future. In this context, genetically encoded tools such as those used in optogenetics/chemogenetics and their applications in human therapeutics will be considered.
Delivery tools will focus on viral vectors, mostly adeno-associated viruses, and the major improvements made in the last few years related to cell type and circuit targeting and blood-brain barrier crossing while highlighting the remaining challenges. Cell targeting and mapping strategies such as those elaborated using single-cell transcriptomics and epigenomics will be explored. Such approaches can be viewed as the foundation for defining the parts list (i.e., brain cell types) and as a hook for developing novel tools for targeted access to genetically defined cell types. The various models used in translational research (neurons and brain organoids derived from human iPSC, post-mortem and neurosurgical human brain tissue, various NHP species, etc.) will be described, reviewed, and challenged.
Topics to be covered will include:
- The concepts of targeting specific brain cell types across species and across lifetimes. For instance, how can reliable access to cell types be achieved given that gene expression and enhancer/promoter activity patterns can change throughout development and in normal aging and disease states?
- Opportunities and limitations of gene-based treatments such as optogenetics and chemogenetics
- Since human brain circuit dissection, cell/gene targeting, and therapeutics are at the heart of this issue, ethical perspectives will also be explored. For instance, the ethics of harvesting and experimenting on human brain tissue or cells and organoids of human origin.
Topic Editor Dr. Ting participates in research at the Allen Institute which received financial support from BioMarin, a leader in AAV gene therapy. Dr. Ting also holds patent applications on cell type-specific enhancers, including uses thereof and for Gene Therapy applications. Topic Editor Dr. Paquet declares no competing interests with regard to the Research Topic subject.
This topic will be focusing on the highly specific tools that are emerging for enabling brain circuit dissection and refinement of gene-based therapeutics in the central nervous system of human and non-human primates. It will assemble experts in the field (academics, clinicians, and from industry) and provide the reader with an overview of the current tools available, their limitations, and a perspective on where these tools are headed in the future. In this context, genetically encoded tools such as those used in optogenetics/chemogenetics and their applications in human therapeutics will be considered.
Delivery tools will focus on viral vectors, mostly adeno-associated viruses, and the major improvements made in the last few years related to cell type and circuit targeting and blood-brain barrier crossing while highlighting the remaining challenges. Cell targeting and mapping strategies such as those elaborated using single-cell transcriptomics and epigenomics will be explored. Such approaches can be viewed as the foundation for defining the parts list (i.e., brain cell types) and as a hook for developing novel tools for targeted access to genetically defined cell types. The various models used in translational research (neurons and brain organoids derived from human iPSC, post-mortem and neurosurgical human brain tissue, various NHP species, etc.) will be described, reviewed, and challenged.
Topics to be covered will include:
- The concepts of targeting specific brain cell types across species and across lifetimes. For instance, how can reliable access to cell types be achieved given that gene expression and enhancer/promoter activity patterns can change throughout development and in normal aging and disease states?
- Opportunities and limitations of gene-based treatments such as optogenetics and chemogenetics
- Since human brain circuit dissection, cell/gene targeting, and therapeutics are at the heart of this issue, ethical perspectives will also be explored. For instance, the ethics of harvesting and experimenting on human brain tissue or cells and organoids of human origin.
Topic Editor Dr. Ting participates in research at the Allen Institute which received financial support from BioMarin, a leader in AAV gene therapy. Dr. Ting also holds patent applications on cell type-specific enhancers, including uses thereof and for Gene Therapy applications. Topic Editor Dr. Paquet declares no competing interests with regard to the Research Topic subject.
