Gene Therapy has finally entered the clinic. Currently, there are 16 Gene Therapies approved worldwide with thousands more in development. The numerous preclinical studies and clinical trials have made it clear that Gene Therapy provides safe therapeutic benefits for otherwise incurable diseases. This has been achieved by the current generation of biotechnological tools that have permitted the design and implementation of successful single target approaches.
Although the current generation of single target Gene Therapies show great promise, the leading causes of death worldwide are from diseases that have a protracted and complex pathogenesis. We need safe and effective biotechnological tools in place to design next-generation composite gene therapies that can treat these diseases. Therefore we need the tools to treat specific cell types, multiple tissues, or organs and modulate gene circuits when needed.
How can we build on what the gene therapy field has achieved over the last decades and develop effective transformative treatments for severe multifactorial diseases such as ischaemic heart failure, Alzheimer's and other dementias, stroke, chronic obstructive pulmonary disease? With the rapid development of biomedical biotechnology in recent years is the gene therapy field capable of designing therapies that can address the complex nature of these diseases?
The goal of this Research Topic is to assess the current status, advances, and challenges of biotechnological tools needed to develop composite gene therapies to treat multifactorial diseases. Specifically, viral vector design that can accommodate complex approaches, strategies to target multiple genes or cell types, use of inducible gene expression systems, manipulation of endogenous gene expression, and synthetic circuit applications for gene therapy.
The specific themes we would like the contributors to address are:
- Viral Vector Design
- Inducible gene expression systems
- Therapeutic modulation of endogenous gene expression
- Combinatorial manipulation of therapeutic genes
- Synthetic circuits
As this research topic is also meant to address current challenges, the editors also feel that Original Research articles or Brief research report articles that have well-designed and executed studies that had a negative outcome are also welcomed. In the context of this research topic, we feel that such articles will also help to move the field forward.
Gene Therapy has finally entered the clinic. Currently, there are 16 Gene Therapies approved worldwide with thousands more in development. The numerous preclinical studies and clinical trials have made it clear that Gene Therapy provides safe therapeutic benefits for otherwise incurable diseases. This has been achieved by the current generation of biotechnological tools that have permitted the design and implementation of successful single target approaches.
Although the current generation of single target Gene Therapies show great promise, the leading causes of death worldwide are from diseases that have a protracted and complex pathogenesis. We need safe and effective biotechnological tools in place to design next-generation composite gene therapies that can treat these diseases. Therefore we need the tools to treat specific cell types, multiple tissues, or organs and modulate gene circuits when needed.
How can we build on what the gene therapy field has achieved over the last decades and develop effective transformative treatments for severe multifactorial diseases such as ischaemic heart failure, Alzheimer's and other dementias, stroke, chronic obstructive pulmonary disease? With the rapid development of biomedical biotechnology in recent years is the gene therapy field capable of designing therapies that can address the complex nature of these diseases?
The goal of this Research Topic is to assess the current status, advances, and challenges of biotechnological tools needed to develop composite gene therapies to treat multifactorial diseases. Specifically, viral vector design that can accommodate complex approaches, strategies to target multiple genes or cell types, use of inducible gene expression systems, manipulation of endogenous gene expression, and synthetic circuit applications for gene therapy.
The specific themes we would like the contributors to address are:
- Viral Vector Design
- Inducible gene expression systems
- Therapeutic modulation of endogenous gene expression
- Combinatorial manipulation of therapeutic genes
- Synthetic circuits
As this research topic is also meant to address current challenges, the editors also feel that Original Research articles or Brief research report articles that have well-designed and executed studies that had a negative outcome are also welcomed. In the context of this research topic, we feel that such articles will also help to move the field forward.
