Viral-mediated gene therapy is intended to treat disorders by introducing therapeutic gene(s) or correcting the missense mutation in the patient using viral vectors as a delivery tool. This innovative technique is used not only for treating a wide range of inherited diseases but has the potential to cure other diseases, including cancer, neurodegenerative diseases, and infectious diseases. For example, the use of viral-mediated gene therapy for treating patients of Duchenne muscular dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration due to the absence of dystrophin gene. However, the challenges in such therapy are the immune responses and vector safety concerns. Although there is an increased number of literature on these issues focusing mainly on the advancements in viral vector design and their delivery methods, there is still scope for improvement. The need to develop a next-generation viral vector with improved safety profiles and high transduction efficiency to reduce viral titer which includes the use of adeno-associated viruses (AAVs), lentiviruses, adenovirus and other viral vectors such as cytomegalovirus (CMV). Moreover, optimizing delivery strategies to target specific tissues is a major concern of the field. Additionally, the advancement of gene editing technologies like CRISPR-Cas9 for precise gene modification is an important part of viral gene therapy.
Here, we welcome researchers to submit Original Research articles and up-to-date Reviews of the primary literature that help us to better understand viral-mediated gene therapy, particularly in the advancement of engineered viral vectors and their delivery methods in human diseases.
The scope of this Research Topic is to cover all the viral-mediated gene therapies in various human diseases. The key points of this topic are:
• Engineering the viral vectors to reduce their immunogenicity with high transduction efficiency.
• Enhancing the tissue-specific targeting of viral vectors using various strategies (not only antigen antibody-mediated) to reduce the off-target effect.
• Optimizing the viral vector formulation to improve their stability and cellular uptake, especially for solid tumors and the central nervous system.
• Development of novel vector serotypes to evade the immune system of patients in preclinical and clinical settings.
• Optimizing the gene editing techniques like CRISPR-Cas9 within the context of viral vector-mediated gene therapy
Keywords:
Viral gene therapy, Crispr, Tissue specific, Modified viral vector, AAVs, CMV
Important Note:
All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.
Viral-mediated gene therapy is intended to treat disorders by introducing therapeutic gene(s) or correcting the missense mutation in the patient using viral vectors as a delivery tool. This innovative technique is used not only for treating a wide range of inherited diseases but has the potential to cure other diseases, including cancer, neurodegenerative diseases, and infectious diseases. For example, the use of viral-mediated gene therapy for treating patients of Duchenne muscular dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration due to the absence of dystrophin gene. However, the challenges in such therapy are the immune responses and vector safety concerns. Although there is an increased number of literature on these issues focusing mainly on the advancements in viral vector design and their delivery methods, there is still scope for improvement. The need to develop a next-generation viral vector with improved safety profiles and high transduction efficiency to reduce viral titer which includes the use of adeno-associated viruses (AAVs), lentiviruses, adenovirus and other viral vectors such as cytomegalovirus (CMV). Moreover, optimizing delivery strategies to target specific tissues is a major concern of the field. Additionally, the advancement of gene editing technologies like CRISPR-Cas9 for precise gene modification is an important part of viral gene therapy.
Here, we welcome researchers to submit Original Research articles and up-to-date Reviews of the primary literature that help us to better understand viral-mediated gene therapy, particularly in the advancement of engineered viral vectors and their delivery methods in human diseases.
The scope of this Research Topic is to cover all the viral-mediated gene therapies in various human diseases. The key points of this topic are:
• Engineering the viral vectors to reduce their immunogenicity with high transduction efficiency.
• Enhancing the tissue-specific targeting of viral vectors using various strategies (not only antigen antibody-mediated) to reduce the off-target effect.
• Optimizing the viral vector formulation to improve their stability and cellular uptake, especially for solid tumors and the central nervous system.
• Development of novel vector serotypes to evade the immune system of patients in preclinical and clinical settings.
• Optimizing the gene editing techniques like CRISPR-Cas9 within the context of viral vector-mediated gene therapy
Keywords:
Viral gene therapy, Crispr, Tissue specific, Modified viral vector, AAVs, CMV
Important Note:
All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.